Propionic Acidemia clinical trials
The registry lists 22 studies for Propionic Acidemia across more than 10 countries. 8 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-22.
Status
- Completed
- 8
- Recruiting
- 7
- Terminated
- 4
- Active, not recruiting
- 1
- Not yet recruiting
- 1
- Withdrawn
- 1
Phase
- Phase 2
- 6
- Phase 1
- 5
- Phase 3
- 1
Where
- United States
- 15
- United Kingdom
- 6
- Spain
- 5
- France
- 5
- Saudi Arabia
- 4
- Canada
- 4
Who runs them
ModernaTX, Inc. (4) · Mendel Tuchman (2) · Recordati Rare Diseases (2) · HemoShear Therapeutics (2) · Children's Hospital of Philadelphia (1) · National Human Genome Research Institute (NHGRI) (1) · Nicola Longo (1) · National Taiwan University Hospital (1)
Studies
Recruiting first, then most recently updated. Showing 22 of 22 — search for more.
- Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic AcidemiaRecruitingNCT028903421 country
- AAVrh10-PCCA Gene Therapy for Propionic AcidemiaRecruitingNCT07643844Phase 11 country
- A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 StudyRecruitingNCT05130437Phase 1/28 countries
- A Retrospective Study to Characterize Participants With Propionic AcidemiaRecruitingNCT057696219 countries
- Open-Label Study of mRNA-3927 in Participants With Propionic AcidemiaRecruitingNCT04159103Phase 1/28 countries
- An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and PediatricsRecruitingNCT050401781 country
- MyRareDiet A Novel Diet Tracking ToolNot yet recruitingNCT06664840Not applicable
- Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachRecruitingNCT041765237 countries
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Review of Charts From Amish/Mennonite Variant PA PatientsCompletedNCT031590261 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Long-term Efficacy of Carglumic Acid in Organic Acidemia.CompletedNCT04284917Not applicable1 country
- Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)CompletedNCT053300391 country
- Study of HST5040 in Subjects With Propionic or Methylmalonic AcidemiaTerminatedNCT04732429Phase 23 countries
- Natural History Study of Patients With Methylmalonic Acidemia and Propionic AcidemiaTerminatedNCT054384853 countries
- A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic AcidemiaTerminatedNCT04836494Phase 11 country
- "The MaP Study": Mapping the Patient Journey in MMA and PACompletedNCT034847674 countries
- Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute HyperammonemiaCompletedNCT01599286Phase 21 country
- Carglumic Acid in Methylmalonic Acidemia and Propionic AcidemiaCompletedNCT02426775Phase 31 country
- Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic AcidemiaTerminatedNCT01597440Phase 21 country
- Anaplerotic Therapy in Propionic AcidemiaCompletedNCT00645879Phase 11 country
- Increasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateWithdrawnNCT01341379Phase 2