Grant List
Represents Grant table in the DB
GET /v1/grants?page%5Bnumber%5D=1419&sort=end_date
{ "links": { "first": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1&sort=end_date", "last": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1424&sort=end_date", "next": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1420&sort=end_date", "prev": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1418&sort=end_date" }, "data": [ { "type": "Grant", "id": "15778", "attributes": { "award_id": "1R50CA305057-01", "title": "A System to Support Development and Success of NCI-Sponsored Trials for Rare Diseases", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Cancer Institute (NCI)" ], "program_reference_codes": [], "program_officials": [ { "id": 32853, "first_name": "SONYA", "last_name": "ROBERSON", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-08-11", "end_date": "2030-07-31", "award_amount": 122610, "principal_investigator": { "id": 32854, "first_name": "Kim A", "last_name": "Reiss Binder", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2627, "ror": "", "name": "UNIVERSITY OF PENNSYLVANIA", "address": "", "city": "", "state": "PA", "zip": "", "country": "United States", "approved": true }, "abstract": "R50 Abstract – Reiss Gastrointestinal maligancies remain lethal and difficult-to-treat, with poor outcomes for many patients. In spite of this, we are in a moment of a scientific and clinical revolution with the development of biomarker-driven therapies for selected patients. With these advances come multiple new questions for the field, many of which the NCTN infrastructure is optimally suited to address. To date, I have demonstrated a strong commitment to clinical and translational research for populations of patients with rare subsets or biomarkers, including via the development and implementation of NCI-trials at my own institution: I am the international study chair and lead accruer for EA2192 as well as the study champion and lead national accruer for SWOG-2001. On the larger scale, I hold leadership roles within the Abramson Cancer Center (ACC) at the University of Pennsylvania and at the NCTN. I serve as the co-chair of the ECOG- ACRIN GI Committee, I am the co-leader of the ACC Cancer Therapeutics Program and I am the co-leader of the ACC Pancreatic Clinical Trial Program. These leadership positions are optimal platforms upon which to spearhead programs that (1) formally and longitudinally assist junior and mid-career oncology faculty in their quests to develop investigator-initiated studies and (2) develop a program at the NCTN that focuses on developing realistic trials for rare populations and then on accruing them successfully. To date, I have demonstrated a persistent and strong commitment to the NCI research enterprise. I have been involved with NCI-related research since my early career, initially attending meetings and later developing my own protocol, EA2192. Based on my steady engagement and input, I was appointed as the co-chair of the GI Committee in 2022 alongside Jordan Berlin (see LOS). Together, we have made a commitment to improving the process of clinical trial development, a mission that will be critical in order for the NCTN to remain competitive in an ever-changing landscape. Over the past three years, we have employed multiple initiatives including boosting the education of our investigators about the NCTN process, leaning on the excellent Working groups chairs to fine-tune concepts prior to Committee Presentation and providing substantial assistance during the submission process. With the support of the R50 Research Specialist award, I will build further on this approach in two ways: At the ACC, I will develop a sustainable program for early and mid-career investigators to provide longitudinal support in the development of investigator-initiated trials, with a particular focus on rare disease studies that can be best executed via the ECOG-ACRIN system. Within the NCTN, I will employ a novel program assisting investigators in the development and successful enrollment of trials for rare disease populations, a subset of studies that have lost ground since the COVID pandemic. Specifically, I will create a living resource for NCTN investigators that focuses on methods to design practical, feasible studies and will provide longitudinal support to those across the NCTN who are developing studies in this space. My ultimate goal is to develop and implement pragmatic clinical trials that address key questions in the field of gastrointestinal malignancies, particularly for patients with rare subsets of disease.", "keywords": [ "Abramson Cancer Center at the University of Pennsylvania", "Address", "American College of Radiology Imaging Network", "Award", "Berlin", "Biological Markers", "COVID-19 pandemic", "Cancer Biology", "Cancer Center", "Clinical", "Clinical Research", "Clinical Trials", "Development", "Disease", "Eastern Cooperative Oncology Group", "Education", "Faculty", "Feasibility Studies", "Genomics", "Goals", "Infrastructure", "Institution", "International", "Jordan", "Knowledge", "Lead", "Leadership", "Malignant Neoplasms", "Malignant neoplasm of gastrointestinal tract", "Methods", "Mission", "Oncology", "Outcome", "Pancreas", "Patient Selection", "Patients", "Persons", "Population", "Positioning Attribute", "Pragmatic clinical trial", "Process", "Program Sustainability", "Protocols documentation", "Rare Diseases", "Research", "Research Personnel", "Resources", "Role", "Selection for Treatments", "Southwest Oncology Group", "Specialist", "System", "Therapeutic", "Thinness", "Translational Research", "biomarker development", "biomarker driven", "cancer subtypes", "career", "design", "gastrointestinal", "improved outcome", "investigator-initiated trial", "meetings", "novel", "patient population", "process improvement", "programs", "success", "trial enrollment", "working group" ], "approved": true } }, { "type": "Grant", "id": "15781", "attributes": { "award_id": "1R01AI183979-01A1", "title": "Expanding access and impact of tuberculosis preventive therapy: Community-friendly delivery and monitoring of TPT to improve uptake and reduce TB transmission", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32562, "first_name": "KAREN A", "last_name": "LACOURCIERE", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-08-19", "end_date": "2030-07-31", "award_amount": 690538, "principal_investigator": { "id": 28321, "first_name": "ADRIENNE E", "last_name": "SHAPIRO", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2645, "ror": "", "name": "UNIVERSITY OF WASHINGTON", "address": "", "city": "", "state": "WA", "zip": "", "country": "United States", "approved": true }, "abstract": "Enter the text here that is the new abstract information for your application. International and Kenyan guidelines recommend TB preventive therapy (TPT) for people with HIV (PWH) and other people at high risk for TB, including close contacts of people with TB. Despite the evidence for reduced morbidity and mortality for people with HIV (PWH) who receive TPT, and guidelines recommending use, there remains a substantial gap between people recommended to receive and people who actually receive and complete a course of TPT. The 2022 WHO Global TB Report highlighted the growing gap in access and provision of TPT, which has been aggravated by the COVID-19 pandemic. Bridging this gap is a Kenyan and global priority. With the recent availability and evidence for newer, shorter regimens of TPT, a transformation of HIV care delivery models (in part forced by the COVID-19 pandemic) and evolving national guidelines for TPT, it is increasingly urgent to explore new person-friendly models of TPT delivery to inform programmatic guidance that results in greater uptake, adherence, and completion of TPT. HIV care transformed to adopt “differentiated service delivery” (DSD) models, which encourage community-delivered care, infrequent clinic/facility visits, multi- month dispensing, limited laboratory monitoring, and task-shifted treatment models to deliver comprehensive HIV care to stable adults in community settings. These successful models for differentiated HIV care delivery may be able to be adapted to include TPT. The availability of safe, effective, short-course TPT (i.e. 3HP, 3HR) with limited monitoring requirements suggests that similar community-based and multi-month dispensing models may be adapted to scale essential TPT to populations who most need it, including PWH, young child contacts, and all household contacts of people with TB. We will explore two approaches of adapting HIV differentiated services to TB prevention. We hypothesize that people who receive differentiated TPT delivery have higher rates of completion of a course of TPT than people who receive standard-of-care clinic-based TPT. We will 1) conduct a randomized controlled trial of DSD care (multi-month dispensing) vs. clinic standard-of-care TPT delivery in two priority populations for TPT in Kenya: household contacts of people with TB and PWH, 2) investigate the impact of DSD TPT on household and community TB transmission with follow-up testing and mathematical modeling, and 3) examine preferences, barriers and facilitators of TPT completion and TPT implementation using qualitative research. Together, this research will establish the foundation for implementation studies of optimized patient and community-friendly, differentiated TPT delivery approaches to increase TPT uptake and completion in Kenya and ultimately decrease morbidity, transmission, and mortality from TB.", "keywords": [ "Adherence", "Adopted", "Adult", "Adverse effects", "Biological Assay", "COVID-19 pandemic", "Caring", "Cessation of life", "Child", "Client", "Clinic", "Clinical", "Communicable Diseases", "Communities", "Dose", "Drug resistance in tuberculosis", "Eligibility Determination", "Focus Groups", "Foundations", "Friends", "Goals", "Guidelines", "HIV", "HIV/TB", "Health system", "Home", "Household", "Incidence", "Individual", "Interferon Type II", "International", "Interview", "Kenya", "Laboratories", "Measures", "Methodology", "Methods", "Modeling", "Monitor", "Morbidity - disease rate", "Patients", "Persons", "Pharmaceutical Preparations", "Population", "Prevention", "Preventive therapy", "Qualitative Research", "Randomized", "Randomized Controlled Trials", "Recommendation", "Regimen", "Reporting", "Research", "Schedule", "Service delivery model", "Services", "Testing", "Text", "Time", "Toxic effect", "Travel", "Treatment Protocols", "Tuberculosis", "Tuberculosis diagnosis", "Visit", "World Health Organization", "care delivery", "community setting", "cost", "experience", "follow-up", "gaps in access", "health care delivery", "high risk", "immunoreactivity", "implementation strategy", "implementation study", "improved", "informant", "innovation", "isoniazid", "mathematical model", "mortality", "person centered", "pill", "preference", "prevent", "preventable death", "rifapentine", "scale up", "screening", "service delivery", "standard of care", "supply chain", "transmission process", "treatment as usual", "trial comparing", "uptake" ], "approved": true } }, { "type": "Grant", "id": "15782", "attributes": { "award_id": "1R01AI189398-01", "title": "Structural and mechanistic study of bat NLRP6 inflammasome in detecting RNA viruses", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32860, "first_name": "KENTNER L", "last_name": "SINGLETON", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-08-01", "end_date": "2030-07-31", "award_amount": 451836, "principal_investigator": { "id": 32861, "first_name": "Chen", "last_name": "Shen", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2646, "ror": "", "name": "WASHINGTON UNIVERSITY", "address": "", "city": "", "state": "MO", "zip": "", "country": "United States", "approved": true }, "abstract": "Bats harbor the unique ability to host a wide array of emerging viruses, such as Ebola virus, Nipah virus, Hendra virus, and severe acute respiratory syndrome coronavirus (SARS-CoV). These RNA viruses are highly pathogenic and often lethal to humans and animals. Intriguingly, bats develop no/minimal signs of diseases in both natural and experimental infections. Significant progress has been made to suggest the altered immunological networks and dampened inflammatory signaling in bats. However, the direct viral sensing mechanisms in bats and the unique immunological features that distinguish bats from other mammals remain poorly studied. Inflammasomes are multi-protein signaling platforms that form in epithelial cells and myeloid cells upon stimulation by pathogen and damage signals. Their primary function is to active the inflammatory caspases such as caspase-1. Canonical inflammasome sensors consist mainly of nucleotide-binding domain (NBD), leucine-rich repeat (LRR)-containing (NLR) family proteins. Among these NLR proteins, NLRP6 is a unique pattern recognition receptor that is predominantly expressed in intestinal and liver system. The inflammasome function of NLRP6 has been reported to directly detect the RNA viruses (rotavirus and mouse hepatitis virus) that infect the gastrointestinal (GI) tract. On the other hand, the excessive activation of NLRP6 inflammasome may exacerbate the tissue damage and cause the autoinflammatory diseases. In bats, the GI tract represents one major organ for viral infection, while infections rarely cause symptoms. The long-term goal of our project is to understand the specific inflammasome sensing mechanisms in detecting RNA viruses in the intestinal epithelium of bats and gain the insights of how bats protect themselves from the pathogenesis of inflammation-induced intestinal barrier dysfunction. In this application, we propose to pursue the following specific aims: 1) Determine the cryo- EM structures of bat NLRP6 monomer, elucidate the biochemical foundation of bat NLRP6- dsRNA interaction, determine the cryo-EM structures of bat NLRP6 with viral dsRNA and compare the structural mechanisms of dsRNA sensing and inflammasome signaling among bat, mouse and human NLRP6; 2) Elucidate the RNA virus-induced bat NLRP6 inflammasome signaling in reconstituted intestinal epithelial cells (IECs), analyze the bat inflammasome signaling in Eonycteris spelaea (Es) in response to bat-borne RNA viruses, study the genetic role of bat NLRP6 in regulating inflammasome signaling in bat primary IECs/bat intestinal organoids. The proposed studies will guide the development of therapeutics to target GI inflammatory disorders in human based on the molecular details of bat NLRP6 inflammasome.", "keywords": [ "Address", "Affinity", "Animals", "Attention", "Binding", "Biochemical", "Biochemistry", "Biology", "Biophysics", "Blood", "Body Size", "CASP1 gene", "Caspase", "Chiroptera", "Coronavirus", "Cryoelectron Microscopy", "Data", "Development", "Diarrhea", "Disease", "Double-Stranded RNA", "Ebola virus", "Electrophoretic Mobility Shift Assay", "Enteritis", "Epithelial Cells", "Exhibits", "Flying body movement", "Foundations", "Functional disorder", "Gastrointestinal tract structure", "Genetic", "Genetic study", "Goals", "Hendra Virus", "Human", "Immune system", "Immunologics", "Immunology", "In Vitro", "Infection", "Inflammasome", "Inflammation", "Inflammatory", "Inflammatory Bowel Diseases", "Inflammatory Response", "Innate Immune System", "Interdisciplinary Study", "Intestines", "Learning", "Leucine-Rich Repeat", "Liver", "Mammals", "Maps", "Mediating", "Middle East Respiratory Syndrome", "Middle East Respiratory Syndrome Coronavirus", "Molecular", "Murine hepatitis virus", "Mus", "Myeloid Cells", "Nipah Virus", "Nucleotides", "Organ", "Organoids", "Outcome", "Pathogenesis", "Pathogenicity", "Pattern", "Pattern recognition receptor", "Play", "Protein Family", "Proteins", "RNA Virus Infections", "RNA Viruses", "Recombinants", "Reporting", "Resolution", "Role", "Rotavirus", "SARS coronavirus", "Signal Transduction", "Signaling Protein", "Structure", "Symptoms", "Syndrome", "System", "Therapeutic", "Tissues", "Viral", "Viral Load result", "Virus", "Virus Diseases", "Work", "autoinflammatory", "autoinflammatory diseases", "bat-borne", "emerging virus", "experimental study", "gastrointestinal", "gastrointestinal system", "insight", "intestinal barrier", "intestinal epithelium", "life span", "metabolic rate", "monomer", "novel therapeutics", "pathogen", "prevent", "reconstitution", "response", "restraint", "sensor", "therapeutic development", "viral RNA" ], "approved": true } }, { "type": "Grant", "id": "15783", "attributes": { "award_id": "1R01AI188944-01A1", "title": "PRECISE: accessible sample-to-answer RT-PCR detection of hepatitis C infection from whole blood", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32845, "first_name": "JULIE", "last_name": "DYALL", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-08-08", "end_date": "2030-07-31", "award_amount": 575356, "principal_investigator": { "id": 32862, "first_name": "Samuel K", "last_name": "Sia", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2647, "ror": "", "name": "COLUMBIA UNIVERSITY HEALTH SCIENCES", "address": "", "city": "", "state": "NY", "zip": "", "country": "United States", "approved": true }, "abstract": "This proposal is in response to NOSI NOT-AI-23-001, which calls for new point-of-care HCV diagnostics. Most of global HCV burden is concentrated in low- and middle-income countries (LMICs), but the U.S. has witnessed a steady increase in infection rates in the past decade, with an estimated 140,000 new cases annually. Whereas direct-acting antivirals (DAAs) can over 95% of HCV-infected individuals, and are becoming more accessible, about 40% of infected people are unaware of their status. The current two-step diagnostic process requires an initial antibody screen followed by an expensive and time-consuming RNA test to confirm active infection. This cumbersome workflow requires multiple office visits for patients, resulting in delays in treatment initiation and significant patient follow-up loss. Our lab has been developing plasmonic-based thermocycling into a clinically useful method for fast multiplexed RT-PCR at the POC. In this approach, heating is achieved not externally via the Peltier effect, but rather internally via infrared excitation of nanoparticles; as a result, the heating is rapid, and powered with low- power robust optical components. We have gathered substantial preliminary data to validate the premise of this approach to work on clinical specimens (saliva and nasal swabs); the results (published in Nature Nanotechnology, 2022) showed the ability, within 25 minutes from sample to result, to detect SARS-CoV-2 with high sensitivity and specificity. Moreover, even in the presence of the plasmonic nanoparticles, we showed that real-time qPCR could be performed with accurate quantitative determinations of cycle threshold (Ct) values. This capability for sample-to-result analysis is faster than traditional PCR approaches using Peltier heating, and the instrumentation using readily available optics renders the approach suitable for community testing sites where rapid and accurate results are sought. This proposal integrates plasmonic PCR with plasma filtration (starting with whole blood) and magnetic bead-based nucleic-acid extraction (a technique we recently published called PRECISE, Lab Chip, 2024). The proposed combined method, PRECISE plasmonic PCR, takes advantage of special nanoparticles to facilitate a seamless sample-to-result workflow for the end user, suitable for the target community health settings to facilitate HCV diagnosis in a single patient visit to enable on-the-spot treatment. The target metrics would be the best in class among HCV nucleic-acid tests, meeting all of the criteria in the target product profile recommended by the NOSI for an “optimal” test except for the 15-minute turnaround time (our target is 30 minutes), and surpassing all “minimal” targets. The target metrics include cost considerations.", "keywords": [ "Accountability", "Anti-viral Agents", "Antibodies", "Benchmarking", "Biological Assay", "Blood", "Blood specimen", "COVID-19 detection", "Caring", "Clinic", "Clinical", "Collection", "Communities", "Community Health", "Consumption", "Data", "Decentralization", "Detection", "Devices", "Diagnosis", "Diagnostic", "Filtration", "HCV infection", "Health Care Surveys", "Health Personnel", "Heating", "Hepatitis C virus", "India", "Individual", "Infection", "Liver", "Magnetism", "Malignant neoplasm of liver", "Methods", "Microfluidics", "Nanotechnology", "Nature", "New Jersey", "Nucleic Acid Amplification Tests", "Nucleic Acids", "Office Visits", "Optics", "Patients", "Persons", "Phase", "Plasma", "Preparation", "Primary Care", "Process", "Publishing", "RNA", "Recommendation", "Research Institute", "Reverse Transcriptase Polymerase Chain Reaction", "Saliva", "Sampling", "Sensitivity and Specificity", "Site", "Specimen", "Spottings", "Techniques", "Technology", "Temperature", "Testing", "Time", "Training", "Tube", "United States", "Vendor", "Visit", "Whole Blood", "Work", "cost", "cost effective", "detection limit", "detection platform", "follow-up", "high risk", "improved", "infection rate", "innovation", "instrumentation", "low and middle-income countries", "magnetic beads", "meetings", "mortality", "nanoGold", "nanoparticle", "nanoshell", "nasal swab", "novel", "operation", "plasmonics", "point of care", "point of care testing", "response", "screening uptake", "skills", "usability", "user-friendly", "viral detection" ], "approved": true } }, { "type": "Grant", "id": "15824", "attributes": { "award_id": "1U01AI191995-01", "title": "Phase 1 study of a SARS-CoV-2 vaccine adjuvanted via controlled biodistribution of mRNA encoding spike protein and IL-12p70", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32597, "first_name": "BROOKE ALLISON", "last_name": "BOZICK", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-25", "end_date": "2030-07-31", "award_amount": 1673641, "principal_investigator": { "id": 44236, "first_name": "Lindsey Robert", "last_name": "Baden", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 44237, "first_name": "David James", "last_name": "Dowling", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 3368, "ror": "", "name": "BOSTON CHILDREN'S HOSPITAL", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "mRNA-lipid nanoparticle (LNP)-based vaccines are an exciting platform that made advances in combating the pandemic viruses. However, currently licensed mRNA-LNP vaccines approaches are limited in their ability to induce long lived antibody durability, degree of protection varies by age, and levels of cell mediated immunity (CMI) are suboptimal. mRNA vaccine self-adjuvantation is lower in aged individuals, with impaired immune activation upon exposure, inducing insufficient immunity with consequential impaired protection. To try to advance solutions, our published and unpublished work focus on two major novel mRNA vaccine innovations. One is a multi-organ protection (MOP) mRNA sequence homologous to tissue-specific micro-RNAs and enable tissue- specific mRNA translation, constraining expression to the site of injection. Secondly, a biological mRNA-encoded molecular adjuvant precisely guides immunity through inducing the potent and Th1-polarizing analyte, IL-12p70. Based on promising SARS-CoV-2 spike-trimer specific humoral and CMI, in young and aged murine models, and protection from viral challenge in non-human primates (NHPs), the goal of this U01 IICT application is to evaluate a mRNA encoding molecular adjuvant, delivered alongside MOP technology, in the context of a SARS-CoV-2 vaccine. Spike-MOP (CTx892) and IL-12-MOP (CTx672) are first-in-human evaluations, so to assess safety and tolerability, we would first perform dose escalations for CTx892 (stage 1A) and CTx 672 (stage 1B). Based on tolerability, doses less than maximal tolerated CTx892 are chosen to expand sample size for subsequent immunogenicity and mechanism studies. In stage 2, two different doses of CTx 892 will be paired with an escalating CTx672 (IL-12-MOP, e.g., 0.1, 1 µg) dose to assess adjuvanticity along with local and systemic safety and tolerability. Additionally, humoral immunity will be quantified with Spike-specific total IgG (titer and pre-immunization fold- change), IgG1 and IgG3 (Th1 markers), and IgG4 (Th2 marker), with surrogate virus, pseudo- virus, and true viral neutralization. Cellular immunity will include innate assessment in the days after, and adaptive CD4, CD8, and B cell immunity in the weeks, post-immunization. Further readouts include dried blood spots in the hours post-injection to assess proteomic kinetics. In Stage 3, biopsies of lymph node at ~14 days post-immunization and bone marrow at 6 months post-immunization will enable in-depth immunophenotyping and quantification of long-lived plasma cell (LLPC) responses. Durability of humoral and cellular immunity will be probed with a 6-month follow-up sampling of peripheral blood, IL-12-sustained humoral and cellular immunity.", "keywords": [ "2019-nCoV", "Adjuvant", "Adjuvanticity", "Adult", "Age", "Antibodies", "Antigens", "B-Lymphocyte Epitopes", "B-Lymphocytes", "Biodistribution", "Biological", "Blood", "Bone Marrow", "CD8B1 gene", "COVID-19 vaccine", "Cellular Immunity", "Communicable Diseases", "Data", "Dose", "Dryness", "Encapsulated", "Enrollment", "Epitopes", "Evaluation", "Future", "Gene Silencing", "Goals", "Health", "Hour", "Human", "Humoral Immunities", "IgG1", "IgG3", "IgG4", "Immune", "Immune response", "Immunity", "Immunization", "Immunologics", "Immunophenotyping", "Impairment", "Injections", "Interleukin-12", "Interleukins", "Investigation", "Kinetics", "Licensing", "Malaise", "Maximum Tolerated Dose", "Measurement", "Measures", "Messenger RNA", "MicroRNAs", "Molecular", "Myocarditis", "Older Population", "Organ", "Participant", "Pathway interactions", "Pfizer-BioNTech COVID-19 vaccine", "Phase I Clinical Trials", "Plasma", "Plasma Cells", "Proteins", "Proteomics", "Publishing", "RNA vaccine", "Randomized", "Risk", "SARS-CoV-2 spike protein", "Safety", "Sample Size", "Sampling", "Seasons", "Sequence Homologs", "Site", "Spottings", "System", "Technology", "Tissues", "Transcript", "Translations", "Vaccine Adjuvant", "Vaccines", "Viral", "Virus", "Virus Diseases", "aged", "aging population", "clinically relevant", "design", "first-in-human", "follow-up", "immune activation", "immunogenicity", "improved", "innovation", "life span", "lipid nanoparticle", "lymph node biopsy", "lymph nodes", "mRNA Translation", "mRNA lipid nano particle vaccine", "mouse model", "next generation", "nonhuman primate", "novel", "older adult", "pandemic virus", "peripheral blood", "phase 1 study", "primary outcome", "research clinical testing", "respiratory", "response", "safety assessment", "safety testing", "transcriptomics", "unpublished works", "vaccination strategy", "vaccine candidate", "vaccine strategy", "young adult" ], "approved": true } }, { "type": "Grant", "id": "15860", "attributes": { "award_id": "1R35GM156197-01", "title": "Pathological Autoantibodies Contribute to Immune Suppression in Sepsis", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of General Medical Sciences (NIGMS)" ], "program_reference_codes": [], "program_officials": [ { "id": 44285, "first_name": "XIAOLI", "last_name": "ZHAO", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-01", "end_date": "2030-07-31", "award_amount": 410000, "principal_investigator": { "id": 44286, "first_name": "Gerard J", "last_name": "Nau", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 3383, "ror": "", "name": "RHODE ISLAND HOSPITAL", "address": "", "city": "", "state": "RI", "zip": "", "country": "United States", "approved": true }, "abstract": "Sepsis is a severe illness associated with high morbidity and mortality in the US and globally. Complications of multi-organ dysfunction, acute respiratory distress syndrome, and infections have a significant impact on late survival. With limited therapeutic interventions, it is essential to understand the fundamental biology of sepsis. Immune dysfunction and immunosuppression are characteristic of sepsis, especially with late complications where lymphopenia predicts a poor prognosis. Gaps remain, however, in the understanding of the mechanisms underlying prolonged immune suppression. Recently, there has been a greater appreciation of autoimmune antibodies that lead to pathological immune consequences in the setting of critical illness. For example, sepsis due to COVID-19 leads to anti-cytokine autoantibodies (ACAAs) that correlate with greater disease severity. In our preliminary data we have also defined ACAAs post-COVID along with ACAAs in ICU patients with sepsis. We have also made the novel discovery of anti-Fas autoantibodies (FasAAs) induced during sepsis, representing a novel mechanism for lymphopenia associated with critical illness. Our overarching hypothesis is that severe conditions like sepsis induce pathological autoimmunity that alters immune function and increases infections. Our hypothesis predicts that sepsis patients with higher levels of autoantibodies will have more immune dysregulation and secondary infectious complications. The objective of this proposal is to determine the prevalence of ACAAs, define mechanism(s) by which they contribute to immunosuppression, and identify sites where their activity could be disrupted for therapeutic purposes. We propose using complementary immune and computational approaches to identify autoantibodies from the blood of sepsis patients and how they correlate with clinical status. We will investigate the biological function of these antibodies on human cytokines and human cells in vitro, along with developing strategies to interfere with deleterious autoantibodies. To address these issues, we have obtained over 350 serum specimens for this study to discriminate between the effects of preexisting autoantibodies and those with a net increase during sepsis. In addition, we have access to deep RNA-seq data from over 100 sepsis patients for computational studies. The long-term objective is to apply this information to assess prognosis of sepsis patients and create precision therapeutics to sustain and restore immune function.", "keywords": [ "Acute Respiratory Distress Syndrome", "Address", "Antibodies", "Autoantibodies", "Autoimmunity", "Biological Process", "Biology", "Blood", "COVID-19", "Cells", "Characteristics", "Clinical", "Critical Illness", "Data", "Functional disorder", "Human", "Immune", "Immune System Diseases", "Immune system", "Immunosuppression", "In Vitro", "Infection", "Lymphopenia", "Modality", "Morbidity - disease rate", "Organ", "Pathologic", "Patients", "Precision therapeutics", "Prevalence", "Prognosis", "Proteins", "Research", "Sepsis", "Serum", "Severity of illness", "Site", "Specimen", "Therapeutic", "Therapeutic Intervention", "computer studies", "cytokine", "immune function", "infection risk", "mortality", "novel", "novel therapeutics", "post sepsis", "post-COVID-19", "preservation", "septic patients", "transcriptome sequencing" ], "approved": true } }, { "type": "Grant", "id": "15872", "attributes": { "award_id": "1U01AI189332-01A1", "title": "Molecular determinants of vaccine-induced antibody durability", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 44300, "first_name": "AMY COLLEEN", "last_name": "PALIN", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-20", "end_date": "2030-07-31", "award_amount": 777495, "principal_investigator": { "id": 44301, "first_name": "George K", "last_name": "Lewis", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 24795, "first_name": "Mohammad Mohseni", "last_name": "Sajadi", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 1532, "ror": "https://ror.org/036a0e562", "name": "Baltimore VA Medical Center", "address": "", "city": "", "state": "MD", "zip": "", "country": "United States", "approved": true } ] } ], "awardee_organization": { "id": 793, "ror": "", "name": "UNIVERSITY OF MARYLAND BALTIMORE", "address": "", "city": "", "state": "MD", "zip": "", "country": "United States", "approved": true }, "abstract": "This research proposal aims to define the molecular determinants of durable versus non-durable antibody responses in vaccinated individuals. Given the critical role of antibodies in combating infectious diseases, it is essential to understand why some antibody responses are long-lasting while others are transient. This distinction is pivotal for improving vaccine development and enhancing protection against pathogens such as HIV-1, Influenza, and SARS-CoV-2, where current vaccines either fail to induce long-term immunity or require frequent re-administration. The primary objective is to investigate the cellular and molecular mechanisms underpinning the longevity of antibody responses post-vaccination. The study will focus on the dynamics of antibody-secreting cells (ASCs) including plasmablasts, short-lived, and long-lived plasma cells, which are believed to be central to maintaining long-term antibody levels. We propose a benchmark prospective human study to characterize ASC populations and their antibody production over time in response to vaccines that induce varying durations of antibody responses. This involves a clinical trial with vaccine-naïve volunteers using the licensed HPV vaccine, GARDASIL 9, for long-term responses, and an investigational HIV vaccine, IHV01, for short-lived responses. The study will span all zones of antibody dynamics post-vaccination, with comprehensive sampling from peripheral blood, lymph nodes, and bone marrow. Aim 1- A clinical trial will be carried out by vaccinating healthy volunteers intramuscularly with the HPV vaccine on the right thigh and IHV01 on the left thigh, three times at 0, 1, and 6 months with scheduled collections of peripheral blood, lymph node fine needle aspirations (FNA), and bone marrow for 42 months to generate specimens spanning all phases of circulating antibody dynamics. Aim 2- The goal of Aim 2 is to quantify and characterize B cell responses the HPV capsid protein and gp120 epitopes in IHV01 over the course of the clinical trial described above in the following specimens: 1) circulating antibodies; 2) circulating and LN memory B cells; 3) follicular and germinal center B cells; 4) circulating and LN plasmablasts; and 5) bone marrow plasma cell subsets. The specimens/populations will be queried with state-of-the-art methods including cellular phenotyping, transcriptomics, VH/VL lineage analyses, antigen-binding cell frequencies. Aim 3- The goal of Aim 3 is to develop mathematical models relating the molecular and cellular signatures from Aim 2 to the dynamics of the durable and non-durable antibody responses generated to the HPV capsid and the HIV-1 gp120 protein, respectively. The anticipated outcomes include a predictive model of antibody response durability, which will inform future vaccine design and the development of dose-predictive strategies. Successful completion of this study will also provide a valuable resource to the scientific community by archiving and sharing the biological specimens collected, facilitating further research into vaccine-induced immunity.", "keywords": [ "2019-nCoV", "Antibodies", "Antibody Formation", "Antibody Response", "Antibody-mediated protection", "Antigens", "Archives", "B-Lymphocytes", "Benchmarking", "Biological", "Blood", "Bone Marrow", "Capsid", "Capsid Proteins", "Cells", "Characteristics", "Clinical Trials", "Collection", "Communicable Diseases", "Communities", "Data", "Development", "Dose", "Epitopes", "Fine needle aspiration biopsy", "Frequencies", "Future", "Goals", "HIV", "HIV Envelope Protein gp120", "HIV vaccine", "HIV-1", "HIV-1 vaccine", "Human", "Immunity", "Immunization", "Immunize", "Immunoglobulin-Secreting Cells", "Infectious Agent", "Influenza", "Influenza vaccination", "Intramuscular", "Left", "Licensing", "Longevity", "Memory B-Lymphocyte", "Methods", "Molecular", "Mus", "Outcome", "Phase", "Phenotype", "Plasma", "Plasma Cells", "Plasmablast", "Play", "Population", "Prospective Studies", "Proteins", "Reaction", "Research", "Research Design", "Research Proposals", "Resources", "Role", "SARS coronavirus", "Sampling", "Schedule", "Serology", "Specimen", "Structure of germinal center of lymph node", "Thigh structure", "Time", "Vaccinated", "Vaccination", "Vaccine Design", "Vaccinee", "Vaccines", "Work", "antigen binding", "comparative", "design", "draining lymph node", "healthy volunteer", "human study", "improved", "in silico", "lymph nodes", "mathematical model", "pathogen", "peripheral blood", "predictive modeling", "prospective", "response", "time interval", "transcriptomics", "vaccine development", "vaccine response", "vaccine-induced antibodies", "vaccine-induced immunity", "volunteer" ], "approved": true } }, { "type": "Grant", "id": "15891", "attributes": { "award_id": "1R01DA062431-01A1", "title": "The impact of peer support in treatment engagement and adverse events for Medicaid beneficiaries with substance use disorder: a mixed-methods study", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute on Drug Abuse (NIDA)" ], "program_reference_codes": [], "program_officials": [ { "id": 44216, "first_name": "JULIA BETH", "last_name": "ZUR", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-15", "end_date": "2030-07-31", "award_amount": 870378, "principal_investigator": { "id": 24389, "first_name": "Emma Elizabeth", "last_name": "McGinty", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 31780, "first_name": "Jiani", "last_name": "Yu", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 825, "ror": "", "name": "WEILL MEDICAL COLL OF CORNELL UNIV", "address": "", "city": "", "state": "NY", "zip": "", "country": "United States", "approved": true }, "abstract": "Substance use disorder (SUD) due to drugs other than alcohol, which affects 9% of adults in the United States, is a significant public health concern contributing to substantial morbidity and mortality, resulting in over 100,000 overdoses in 2022 alone. While effective SUD treatment exists, fewer than 25% of people with SUD receive treatment. Peer support services (PSS) offer a potential avenue for enhancing SUD treatment and outcomes, with the goal of helping people with SUD initiate and remain engaged in the recovery process. These services, delivered by trained and credentialed peer specialists who have firsthand experience with SUD, encompass a wide range of support services, including recovery planning, community linkages, and social and emotional support services. PSS are unique to other types of care navigation in that they emphasize improving clients' psychosocial skills, including resilience, coping, and empowerment. PSS, which use a recovery coaching model, are also distinct from the peer support provided by uncredentialed sponsors in 12- step facilitation. Growing adoption of PSS has outpaced the evidence on the effectiveness of PSS, which is limited and mixed. Nowhere is this more evident than with Medicaid, the largest payer of SUD treatment services in the U.S. Medicaid is an innovator in coverage of PSS. In 2024, 41 state Medicaid programs covered PSS for adults with SUD, increasing from only 26 states in 2017. Medicaid's substantial expansion of PSS coverage and the urgent need for SUD treatment services within the Medicaid population creates an opportunity to rigorously evaluate the impact of receiving PSS on SUD treatment and SUD-related adverse outcomes. Furthermore, the COVID-era Medicaid policy changes that expanded telehealth coverage provide an avenue to compare the effectiveness of telehealth PSS relative to in-person delivered PSS. The overall goals of this study are to provide robust evidence on whether and how PSS impact SUD treatment engagement and outcomes and to inform the design of PSS coverage policy in Medicaid and other insurance programs. We will focus on the following specific aims to achieve these objectives: (1) Assess the impact of Medicaid-covered PSS on SUD treatment initiation, engagement, and retention among Medicaid beneficiaries with SUD, (2) Assess the impact of Medicaid PSS on adverse events including SUD-related emergency department visits, unplanned inpatient readmissions, and drug overdose among Medicaid beneficiaries with SUD; and (3) Characterize how Medicaid PSS coverage policies can influence PSS implementation for Medicaid beneficiaries with SUD. This proposed mixed-methods study will use a concurrent-embedded approach, such that quantitative results (Aims 1-2) will inform qualitative analyses (Aim 3) and vice versa at various points in the study. Study findings will build evidence on the impact of PSS on SUD treatment and overdose and inform the rapidly evolving landscape of PSS policy and implementation.", "keywords": [ "Adopted", "Adoption", "Adult", "Adverse event", "Affect", "Age", "Area", "COVID-19 pandemic", "Caring", "Cessation of life", "Client", "Communication", "Communities", "Coupling", "Credentialing", "Data", "Effectiveness", "Emergency department visit", "Emotional", "Exhibits", "Goals", "Individual", "Inpatients", "Insurance Carriers", "Insurance Coverage", "Internet", "Interview", "Link", "Medicaid", "Mentors", "Methods", "Modeling", "Morbidity - disease rate", "National Institute of Drug Abuse", "Outcome", "Overdose", "Patients", "Persons", "Pharmaceutical Preparations", "Policies", "Population", "Process", "Public Health", "Recovery", "Research", "Role", "Sampling", "Services", "Shapes", "Specialist", "Substance Use Disorder", "Supervision", "Telemedicine", "Testing", "Time", "Training", "Transportation", "Treatment outcome", "United States", "adverse outcome", "alcohol and other drug", "beneficiary", "compare effectiveness", "coping", "design", "empowerment", "evidence base", "experience", "flexibility", "health application", "hospital readmission", "implementation process", "implementation strategy", "improved", "indexing", "insurance program", "marginalization", "mortality", "overdose death", "patient population", "peer", "peer influence", "peer support", "programs", "psychosocial", "resilience", "skills", "social", "social stigma", "telehealth", "treatment and outcome", "treatment services" ], "approved": true } }, { "type": "Grant", "id": "15437", "attributes": { "award_id": "5U24HL166784-02", "title": "2/2: PREcision VENTilation to attenuate Ventilation-Induced Lung Injury (PREVENT VILI)", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Heart Lung and Blood Institute (NHLBI)" ], "program_reference_codes": [], "program_officials": [ { "id": 22589, "first_name": "CHRISTIAN RENE", "last_name": "Gomez", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2023-09-01", "end_date": "2030-08-31", "award_amount": 661149, "principal_investigator": { "id": 27913, "first_name": "Elias", "last_name": "Baedorf Kassis", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 27914, "first_name": "TIMOTHY T", "last_name": "HOULE", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 771, "ror": "https://ror.org/04drvxt59", "name": "Beth Israel Deaconess Medical Center", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "/ ABSTRACT Acute respiratory distress syndrome (ARDS) occurs in up to one-quarter of all critically ill adults receiving mechanical ventilation and is associated with high risk of morbidity and mortality, which is compounded by ventilation-induced lung injury (VILI). Current ventilation strategies and standard care attempt to lessen the effects of VILI, but have not been uniformly successful. The need for improved ventilation strategies have become more compelling and urgent in the context of the COVID-19 pandemic. Precise ventilator titration of tidal volumes to maintain driving pressure ≤ 12 cm H2O may improve overdistension injury and application of positive end expiratory pressure (PEEP) with titration to transpulmonary pressure of 0±2cmH2O may prevent injury from lung collapse. This U24 grant application will establish a Data Coordinating Center (DCC) that supports the PREcision VENTilation to attenuate VILI Clinical Coordinating Center (CCC) in conducting a multicenter, prospective phase III randomized trial to test the hypothesis that this precision ventilation strategy will improve 60-day mortality compared to guided usual care in patients with moderate or severe ARDS. The DCC will provide the infrastructure, support, oversight and quality control necessary to conduct this trial guided by the following aims: Aim 1 - To collaborate in the protocol development. The DCC will assist the CCC in defining the overarching plan of the trial, and ensure the inclusion of essential sections within the protocol document. Aim 2 - To design and execute an appropriate, pre-determined, innovative statistical plan including analysis of study data, assessment of safety, investigation of mechanism of benefit, and facilitation of manuscript preparation. Aim 3 – To facilitate a patient centered study, with community engagement, by providing video-assisted consent materials, real-time reporting of accrual rates, with emphasis on ensuring a diverse participant population and ensuring patient safety by providing clear instructions on identification and timing of adverse event reporting and creation of the Data and Safety Monitoring Board. Aim 4 - To support an advanced physiological core laboratory to integrate physiological expertise into trial conduct and data management and to ensure rigor and reproducibility of respiratory waveform interpretation used for protocol-directed ventilator management in the intervention arm. The DCC will review all physiological waveforms submitted by sites and provide feedback regarding quality and interpretation. Aim 5 - To provide data administration and monitor trial activities. The DCC will build the eCRFs on a state-of-the-art electronic data capture system. During enrollment, the DCC will ensure high-quality data collection and measure and improve protocol compliance. To foster site communication and identity, the DCC will develop a trial website and quarterly newsletter, and assist the CCC in organizing meetings and conference calls. Aim 6 - To ensure completeness of the study and post-trial activities. This includes meeting milestones and end-of-trial support for participating sites and providing the funding agency with a finalized, deidentified, and locked data set for public use.", "keywords": [], "approved": true } }, { "type": "Grant", "id": "15818", "attributes": { "award_id": "1U54HD121579-01", "title": "Project 3- Access Study", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)" ], "program_reference_codes": [], "program_officials": [], "start_date": "2025-09-15", "end_date": "2030-08-31", "award_amount": 204764, "principal_investigator": { "id": 44226, "first_name": "Kimberly Ann", "last_name": "Chapman", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2610, "ror": "", "name": "BAYLOR COLLEGE OF MEDICINE", "address": "", "city": "", "state": "TX", "zip": "", "country": "United States", "approved": true }, "abstract": "7. ABSTRACT – PROJECT 3 (TELEHEALTH VS VIRTUAL VISITS IN ORGANIC ACIDEMIAS) In a recent survey from the National Organization for Rare Disorders (NORD), nearly 40% of patients/caregivers with rare disorders reported traveling more than 60 miles for medical care. The same survey also found that 70% of respondents would like the option of telehealth for medical appointments and that during the SARS-CoV-2 pandemic, 88% of those offered a video appointment accepted. Of those accepting a video visit, 92% said it was a positive experience. In addition to the access to care issues, the NORD survey also found that 62% of patients with rare diseases were not able to attend work due to their appointments, and that 26% of children missed school regularly for appointments. Consequently, NORD is strongly advocating robust access to telehealth services for patients with rare diseases. Access to care for rare organic acidemias is an even greater challenge. In a recent survey by the Organic Acidemia Association and Propionic Acidemia Foundation, access to care was a top concern for individuals and families. According to the American Board of Medical Genetics and Genomics website, ten US states have no provider certified in either clinical or medical biochemical genetics demonstrating a significant gap in access to local specialists with expertise in organic acidemias. Although telemedicine has been shown to be effective for genetic counseling and genetic patient evaluations, care for individuals with organic acidemias involves more than counseling and diagnosis. Treatment for organic acidemias is complex requiring a combination of low protein diets, medications/supplements, strict adherence to treatment regimens, and the implementation of sick-day protocols in collaboration with the healthcare team. Frequent monitoring of growth, nutrition, and laboratory values and ongoing education are critical to successfully managing organic acidemias. We hypothesize that telehealth is one strategy for increasing access to high-quality care for organic acidemias. To test this hypothesis, we will perform the first large, multi-center study evaluating the efficacy of telehealth for organic acidemias with the following aims: 1) Assess whether virtual clinic visits are equivalent to in-person visits at achieving treatment goals, 2) Compare attendance at clinic visits and knowledge of treatment regimens for those receiving care at virtual and in-person visits, and 3) Assess patient and parent satisfaction and patient-care team relationships with virtual clinic visits compared to in-person visits. Overall, this multi-center, longitudinal study of virtual vs. in-person medical visits will demonstrate whether virtual visits are effective at providing high quality care for individuals with these disorders. As virtual visits may be a strategy for increasing access to clinical trials and meeting enrollment goals in trials for organic acidemias, the results of this study are important for both clinical trial readiness and improving patient access to quality care.", "keywords": [ "Acute", "Adherence", "Advocate", "American", "Appointment", "Area", "Biochemical", "Biochemical Genetics", "COVID-19 pandemic", "Caregivers", "Caring", "Certification", "Child", "Client satisfaction", "Clinic Visits", "Clinical", "Clinical Trials", "Collaborations", "Collection", "Complex", "Counseling", "Diagnosis", "Diet", "Disease", "Education", "Emergency department visit", "Enrollment", "Evaluation", "Event", "Family", "Foundations", "Genetic", "Genetic Counseling", "Genomics", "Goals", "Growth", "Health Services", "Health Services Accessibility", "Hospitalization", "Illness Days", "Improve Access", "Inborn Errors of Metabolism", "Individual", "Knowledge", "Laboratories", "Logistics", "Longitudinal Studies", "Measures", "Medical", "Medical Care Team", "Medical Genetics", "Metabolic", "Monitor", "Multicenter Studies", "Nutrition", "Outcome", "Outcome Assessment", "Outpatients", "Parents", "Participant", "Patient Care Team", "Patients", "Persons", "Pharmaceutical Preparations", "Protein-Restricted Diet", "Protocols documentation", "Provider", "Quality of Care", "Randomized", "Rare Diseases", "Regimen", "Reporting", "Research", "Respondent", "Schools", "Specialist", "Structure", "Surveys", "Telemedicine", "Testing", "Travel", "Treatment Protocols", "US State", "Visit", "Work", "acute care", "care providers", "clinical trial readiness", "dietary", "efficacy evaluation", "experience", "gaps in access", "genetics clinic", "improved", "improved outcome", "ketotic hyperglycinemia", "medical appointment", "meetings", "metropolitan", "patient advocacy group", "satisfaction", "telehealth", "therapeutically effective", "video visit", "virtual", "virtual visit", "web site" ], "approved": true } } ], "meta": { "pagination": { "page": 1419, "pages": 1424, "count": 14236 } } }