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Inborn Errors Metabolism clinical trials recruiting in the U.S.
12 studies for Inborn Errors Metabolism are recruiting participants at sites across the United States.
Cities with the most studies
- Philadelphia, PA4 studies
- Los Angeles, CA4 studies
- New York City, NY4 studies
- Washington, DC3 studies
- Mountain View, CA3 studies
By state
Recently updated studies
Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
RecruitingObservational studyHealthy volunteers welcome
1 Month – 100 Years · All sexes · Updated Sep 28, 2026 · NCT02890342A Long-Term Observational Study of Patients With Fucosidosis
RecruitingObservational study
All ages · All sexes · Updated Sep 17, 2026 · NCT07615400Clinical and Laboratory Study of Methylmalonic Acidemia
RecruitingObservational studyHealthy volunteers welcome
1 Month – 115 Years · All sexes · Updated Sep 10, 2026 · NCT00078078Study of Alkaptonuria
RecruitingObservational study
Ages 2 to 115 · All sexes · Updated Sep 1, 2026 · NCT00005909Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
RecruitingObservational study
7 Months – 15 Months · All sexes · Updated Aug 31, 2026 · NCT06805695An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
RecruitingPhase 3
24 Hours – 7 Months · Men · Updated Aug 12, 2026 · NCT06255782Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
RecruitingPhase 1/2
1 Month – 25 Years · All sexes · Updated Apr 15, 2026 · NCT06839456Registry of Patients Diagnosed With Lysosomal Storage Diseases
RecruitingObservational study
Up to age 64 · All sexes · Updated Apr 8, 2026 · NCT05619900A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
RecruitingPhase 1/2
Ages 1 and older · All sexes · Updated Mar 23, 2026 · NCT05130437Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
RecruitingEarly phase 1
Up to age 30 · All sexes · Updated Feb 20, 2026 · NCT02356653Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
RecruitingPhase 1/2
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
RecruitingPhase 1/2
Ages 1 and older · All sexes · Updated Dec 23, 2025 · NCT05295433From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.