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ClinicalTrials.gov public records Last synced May 21, 2026, 5:33 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–14 of 14 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Organic Acidemias Clear all
Withdrawn Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Methylmalonic Acidemia (MMA)
Interventions
SEL-302
Drug
Lead sponsor
Selecta Biosciences, Inc.
Industry
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced May 21, 2026, 5:33 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Registry
Other
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Dec 26, 2023 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Metabolic Disease, Propionic Acidemia, Organic Acidemia
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
1,045 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2036
U.S. locations
2
States / cities
Bethesda, Maryland • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced May 21, 2026, 5:33 PM EDT
Enrolling by invitation Not applicable Interventional
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
Interventions
Ammonia Study Device
Diagnostic Test
Lead sponsor
Sequitur Health Corp.
Industry
Eligibility
12 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 5, 2025 · Synced May 21, 2026, 5:33 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 31, 2015 · Synced May 21, 2026, 5:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Organic Acidemia, Methylmalonic Acidemia, Inborn Errors of Metabolism
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
2,275 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2004
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 21, 2026, 5:33 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Not listed
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Stanford, California • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2021 · Synced May 21, 2026, 5:33 PM EDT
Terminated Phase 2 Interventional
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
HST5040, Placebo
Drug
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
14
States / cities
San Diego, California • New Haven, Connecticut • Washington D.C., District of Columbia + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 4, 2024 · Synced May 21, 2026, 5:33 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Inborn Errors of Metabolism, Pregnancy, Acidemias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
14 Years to 50 Years · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced May 21, 2026, 5:33 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Propionic Acidemia
Interventions
mRNA-3927
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
15
States / cities
Los Angeles, California • Stanford, California • Miami, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced May 21, 2026, 5:33 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
hLB-001
Biological
Lead sponsor
LogicBio Therapeutics, Inc
Industry
Eligibility
6 Months to 12 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
4
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2024 · Synced May 21, 2026, 5:33 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Propionic Acidemia
Interventions
mRNA-3927
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
1 Year and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2031
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Ann Arbor, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced May 21, 2026, 5:33 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
Interventions
BBP-671, Placebo
Drug
Lead sponsor
CoA Therapeutics, Inc., a BridgeBio company
Industry
Eligibility
15 Years to 55 Years
Enrollment
79 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
3
States / cities
Topeka, Indiana • Pittsburgh, Pennsylvania • Austin, Texas
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced May 21, 2026, 5:33 PM EDT