Methylmalonic acidemia clinical trials
The registry lists 26 studies for Methylmalonic acidemia across more than 10 countries. 7 are recruiting or about to start. 14 of them are mainly about Methylmalonic acidemia (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-29.
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Who runs them
ModernaTX, Inc. (4) · Recordati Rare Diseases (2) · Mendel Tuchman (2) · HemoShear Therapeutics (2) · Children's Hospital of Philadelphia (1) · National Human Genome Research Institute (NHGRI) (1) · CoA Therapeutics, Inc., a BridgeBio company (1) · King Abdullah International Medical Research Center (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.
Who these studies take
Of the 5 Methylmalonic acidemia studies recruiting now, 2 (40%) set no upper age limit and 1 (20%) stop at 75 or younger. 5 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (20%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (100%) | 5 (100%) |
| 18 | 4 (80%) | 4 (80%) |
| 30 | 4 (80%) | 4 (80%) |
| 40 | 4 (80%) | 4 (80%) |
| 50 | 4 (80%) | 4 (80%) |
| 65 | 4 (80%) | 4 (80%) |
| 75 | 4 (80%) | 4 (80%) |
| 85 | 4 (80%) | 4 (80%) |
Across all 26 studies, recruiting or not: 50% stop at 75 or younger and 46% at 65; 96% take children; 0% are for women only and 0% for men only; 27% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 26 of 26. search them.
- Clinical and Laboratory Study of Methylmalonic AcidemiaRecruitingNCT000780781 country
- A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT GeneRecruitingNCT074328804 countries
- An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705RecruitingNCT05295433Phase 1/26 countries
- A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type)Not yet recruitingNCT07163364Phase 3
- 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
- A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic AcidemiaTerminatedNCT04899310Phase 1/26 countries
- Long-term Follow-up Study of Patients Who Received hLB-001 Gene TherapyActive, not recruitingNCT055062541 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Long-term Efficacy of Carglumic Acid in Organic Acidemia.CompletedNCT04284917Not applicable1 country
- A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMAWithdrawnNCT05778877Phase 1/21 country
- Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)CompletedNCT053300391 country
- Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic AcidemiaTerminatedNCT04581785Phase 1/21 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
- Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic AcidemiaWithdrawnNCT03810690Phase 1/2
- Patient and Observer Reported Outcome Measurements in Inborn Errors of MetabolismCompletedNCT042480621 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
- Increasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateWithdrawnNCT01341379Phase 2
- Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome StudyStatus unknownNCT012891581 country