2 withdrawn, 1 not yet recruiting, 1 recruiting. 2 Phase 1, 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Withdrawn 2 |
| Phase 2 | 2 | Withdrawn 2 |
| Phase 3 | 1 | Not yet recruiting 1 |
Follow Methylmalonic Acidemia (MMA): new trials by email Literature Review of Methylmalonic Acidemia (MMA) trials Research Report on Methylmalonic Acidemia (MMA)
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene Methylmalonic Acidemia (MMA) | Recruiting | Genespire Srl | 30 | 2026-08-13 | 2026-08-17 | |
| A Study to Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type) Hydroxocobalamin Chloride Injection - Methylmalonic Acidemia (MMA) | Phase 3 | Not yet recruiting | CSPC ZhongQi Pharmaceutical Technology Co., Ltd. | 20 | 2025-08-31 | 2025-09-09 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA SEL-302 - Methylmalonic Acidemia (MMA) | Phase 1 / Phase 2 | Withdrawn | Selecta Biosciences, Inc. | 2022-12-19 | 2024-09-24 | |
| Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia mRNA-3704 - Methylmalonic Acidemia (MMA), Metabolism, Inborn Errors | Phase 1 / Phase 2 | Withdrawn | ModernaTX, Inc. | 2019-05-28 | 2020-11-13 |
Methylmalonic acidemia (4) · Metabolism, Inborn Errors (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Metabolic Diseases (1) · Nutritional and Metabolic Diseases (1)
CSPC ZhongQi Pharmaceutical Technology Co., Ltd. (1) · Genespire Srl (1) · ModernaTX, Inc. (1) · Selecta Biosciences, Inc. (1)
United States (2) · Italy (1) · Spain (1) · United Kingdom (1)