Propionic Acidemia clinical trials
The registry lists 23 studies for Propionic Acidemia across more than 10 countries. 8 are recruiting or about to start. 12 of them are mainly about Propionic Acidemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.
Get an email when these change. Save the Propionic Acidemia studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Who runs them
ModernaTX, Inc. (4) · HemoShear Therapeutics (2) · Mendel Tuchman (2) · Recordati Rare Diseases (2) · King Abdullah International Medical Research Center (1) · Centre Hospitalier Universitaire de Liege (1) · National Human Genome Research Institute (NHGRI) (1) · Oregon Health and Science University (1)
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Aug 2027Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia · ModernaTX, Inc.
Who these studies take
Of the 7 Propionic Acidemia studies recruiting now, 4 (57%) set no upper age limit and 1 (14%) stop at 75 or younger. 7 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 6 (86%) | 6 (86%) |
| 18 | 6 (86%) | 6 (86%) |
| 30 | 6 (86%) | 6 (86%) |
| 40 | 6 (86%) | 6 (86%) |
| 50 | 6 (86%) | 6 (86%) |
| 65 | 6 (86%) | 6 (86%) |
| 75 | 6 (86%) | 6 (86%) |
| 85 | 6 (86%) | 6 (86%) |
Across all 23 studies, recruiting or not: 48% stop at 75 or younger and 43% at 65; 100% take children; 0% are for women only and 0% for men only; 30% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 23 of 23. search them.
- 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
- Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young InfantsCompletedNCT01624311Phase 21 country
- Anaplerotic Therapy in Propionic AcidemiaCompletedNCT00645879Phase 11 country
- Increasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateWithdrawnNCT01341379Phase 2