8 completed, 6 terminated, 5 recruiting, 3 withdrawn. 10 Phase 2, 6 Phase 1, 2 Not applicable, 2 Phase 3.
Follow Methylmalonic acidemia: new trials by email Literature Review of Methylmalonic acidemia trials Research Report on Methylmalonic acidemia
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 |
| MyRareDiet A Novel Diet Tracking Tool MDR diet assessment, 24 hour recall - Urea Cycle Disorder, Propionic Aciduria | Not applicable | Not yet recruiting | Oregon Health and Science University | 60 | 2024-11-15 | 2024-10-30 |
| 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Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA SEL-302 - Methylmalonic Acidemia (MMA) | Phase 1 / Phase 2 | Withdrawn | Selecta Biosciences, Inc. | 2022-12-19 | 2024-09-24 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy hLB-001 - Methylmalonic Acidemia | Active not recruiting | Alexion Pharmaceuticals, Inc. | 4 | 2022-07-15 | 2026-02-05 | |
| An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics Carglumic Acid - Hyperammonemia, Methylmalonic Acidemia | Recruiting | RECORDATI GROUP | 20 | 2022-06-30 | 2025-04-15 | |
| 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-3705 mRNA-3705 - Methylmalonic Acidemia | Phase 1 / Phase 2 | Recruiting | ModernaTX, Inc. | 56 | 2022-03-08 | 2025-12-23 |
| Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) Inborn Errors of Metabolism, Propionic Aciduria | Completed | Emma Marie Caroline Slack | 22 | 2021-11-30 | 2024-03-01 | |
| Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia Registry - Methylmalonic Acidemia, Propionic Acidemia | Terminated | HemoShear Therapeutics | 11 | 2021-08-27 | 2023-12-27 | |
| A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia mRNA-3705, Placebo - Methylmalonic Acidemia | Phase 1 / Phase 2 | Terminated | ModernaTX, Inc. | 18 | 2021-08-06 | 2026-05-06 |
| Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia hLB-001 - Methylmalonic Acidemia | Phase 1 / Phase 2 | Terminated | LogicBio Therapeutics, Inc | 4 | 2021-05-29 | 2024-02-23 |
| 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 Acidemia BBP-671, Placebo - Healthy Volunteers, Propionic Acidemia | Phase 1 | Terminated | CoA Therapeutics, Inc., a BridgeBio company | 79 | 2021-03-25 | 2023-12-21 |
| Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia HST5040, Placebo - Methylmalonic Acidemia, Propionic Acidemia | Phase 2 | Terminated | HemoShear Therapeutics | 26 | 2021-03-15 | 2024-01-05 |
| Long-term Efficacy of Carglumic Acid in Organic Acidemia. Carglumic Acid (Carbaglu®) - Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) | Not applicable | Completed | National Taiwan University Hospital | 3 | 2019-11-28 | 2025-03-20 |
| Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism Survey - Inborn Errors of Metabolism, Urea Cycle Disorder | Completed | University Children's Hospital, Zurich | 69 | 2019-08-01 | 2020-11-04 | |
| 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 | |
| Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach Carglumic Acid - Methylmalonic Acidemia, Propionic Acidemia | Recruiting | RECORDATI GROUP | 95 | 2019-01-15 | 2024-02-05 | |
| "The MaP Study": Mapping the Patient Journey in MMA and PA Methylmalonic Acidemia, Propionic Acidemia | Completed | ModernaTX, Inc. | 97 | 2018-03-20 | 2021-08-02 | |
| Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Carglumic Acid - Propionic Acidemia, Methylmalonic Acidemia | Phase 3 | Completed | King Abdullah International Medical Research Center | 33 | 2015-11-01 | 2019-04-25 |
Showing 20 of 26. Sign in to see all of them.
Propionic Acidemia (15) · Metabolism, Inborn Errors (11) · Urea Cycle Disorders, Inborn (5) · Carbamoyl-Phosphate Synthase I Deficiency Disease (4) · Genetic Diseases, Inborn (4) · Ornithine Carbamoyltransferase Deficiency Disease (4) · Hyperammonemia (3) · Maple Syrup Urine Disease (3) · Metabolic Diseases (3) · Argininosuccinic Aciduria (2) · Citrullinemia (2) · Malonic aciduria (2) · Phenylketonurias (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Nutritional and Metabolic Diseases (21) · Metabolic Diseases (18) · Genetic Diseases, Inborn (17) · Amino Acid Metabolism, Inborn Errors (16) · Metabolism, Inborn Errors (10) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Nervous System Diseases (7) · Brain Diseases (6) · Genetic Diseases, X-Linked (4) · Mitochondrial Diseases (4) · Pathologic Processes (4) · Pathological Conditions, Signs and Symptoms (4)
carglumic acid (7) · Methylmalonyl-CoA Mutase (2) · N-carbamylglutamate (2) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Hypothermia, Induced (1) · Registries (1) · Renal Dialysis (1) · Standard of Care (1) · Surveys and Questionnaires (1)
ModernaTX, Inc. (4) · HemoShear Therapeutics (2) · Mendel Tuchman (2) · RECORDATI GROUP (2) · Alexion Pharmaceuticals, Inc. (1) · CSPC ZhongQi Pharmaceutical Technology Co., Ltd. (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital of Philadelphia (1) · CoA Therapeutics, Inc., a BridgeBio company (1) · Emma Marie Caroline Slack (1) · Genespire Srl (1) · King Abdullah International Medical Research Center (1) · LogicBio Therapeutics, Inc (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1) · National Human Genome Research Institute (NHGRI) (1)
United States (15) · United Kingdom (6) · Spain (5) · Canada (4) · France (4) · Italy (2) · Netherlands (2) · Saudi Arabia (2) · Switzerland (2) · Australia (1) · Belgium (1) · Germany (1) · Norway (1) · Sweden (1) · Taiwan (1)
Mendel Tuchman, MD (2) · Charles P Venditti, M.D. (1) · Dershung Yang, PhD (1) · Laurent Servais (1) · Majid A. Alfadhel, MD (1) · Marc Yudkoff, MD (1) · Martina Huemer (1) · Ni-Chung Lee, M.D., Ph.D (1) · Nicholas Ah Mew, MD (1) · Patrick Horn, MD PhD (1) · Sufin Yap, MD (1) · Uta Lichter-Konecki, MD, PhD (1) · William Ludlum, MD (1)
PMID 18414145, PMID 19660971, PMID 20338795, PMID 20566609, PMID 21048060, PMID 22614770, PMID 23898205, PMID 25190203, PMID 25567501, PMID 25985870, PMID 26672496, PMID 26790480, PMID 31196016, PMID 33006765, PMID 37243446, PMID 37875310, PMID 41912673, PMID 42839282
R01HD058567 (1), R44HD107766 (1)