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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.

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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 · NCT02890342

A Long-Term Observational Study of Patients With Fucosidosis

RecruitingObservational study
All ages · All sexes · Updated Sep 17, 2026 · NCT07615400

Clinical and Laboratory Study of Methylmalonic Acidemia

RecruitingObservational studyHealthy volunteers welcome
1 Month – 115 Years · All sexes · Updated Sep 10, 2026 · NCT00078078

Study of Alkaptonuria

RecruitingObservational study
Ages 2 to 115 · All sexes · Updated Sep 1, 2026 · NCT00005909

Long-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 · NCT06805695

An 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 · NCT06255782

Phase 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 · NCT06839456

Registry of Patients Diagnosed With Lysosomal Storage Diseases

RecruitingObservational study
Up to age 64 · All sexes · Updated Apr 8, 2026 · NCT05619900

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

RecruitingEarly phase 1
Up to age 30 · All sexes · Updated Feb 20, 2026 · NCT02356653

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

RecruitingPhase 1/2
All ages · All sexes · Updated Jan 22, 2026 · NCT04159103

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.