2 completed, 2 recruiting, 2 terminated, 1 withdrawn. 7 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 2, Recruiting 2, Terminated 2, Withdrawn 1 |
| Phase 1 | 2 | Recruiting 2 |
Follow Propionic Acidemia: new trials by email Literature Review of Propionic Acidemia trials Research Report on Propionic 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 Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study mRNA-3927 - Propionic Acidemia | Phase 1 / Phase 2 | Recruiting | ModernaTX, Inc. | 50 | 2021-11-09 | 2026-03-23 |
| Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia mRNA-3927 - Propionic Acidemia | Phase 1 / Phase 2 | Recruiting | ModernaTX, Inc. | 77 | 2021-04-15 | 2026-01-22 |
| 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 |
| Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Carbaglu, Placebo - Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia | Phase 2 | Completed | Mendel Tuchman | 35 | 2012-09-01 | 2021-02-15 |
| Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia N-carbamylglutamate, Standard of Care - Propionic Acidemia, Methylmalonic Acidemia | Phase 2 | Terminated | Mendel Tuchman | 1 | 2012-09-01 | 2017-04-26 |
| Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate N-carbamylglutamate - Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism | Phase 2 | Withdrawn | Children's Hospital of Philadelphia | 2010-12-01 | 2014-05-29 | |
| Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants Therapeutic Hypothermia, Standard of Care Therapy - Urea Cycle Disorders, Organic Acidemias | Phase 2 | Completed | Uta Lichter-Konecki | 5 | 2007-08-01 | 2015-06-01 |
Methylmalonic acidemia (5) · Metabolism, Inborn Errors (4) · Carbamoyl-Phosphate Synthase I Deficiency Disease (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Acid-Base Imbalance (2) · Acidosis (2) · Amino Acid Metabolism, Inborn Errors (2) · Hyperammonemia (2) · Ornithine Carbamoyltransferase Deficiency Disease (2) · Urea Cycle Disorders, Inborn (2) · Argininosuccinic Aciduria (1) · Brain Diseases (1) · Brain Edema (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Nutritional and Metabolic Diseases (7) · Amino Acid Metabolism, Inborn Errors (5) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Metabolism, Inborn Errors (3) · Mitochondrial Diseases (3) · Nervous System Diseases (3) · Brain Diseases (2) · Genetic Diseases, X-Linked (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2)
carglumic acid (3) · N-carbamylglutamate (2) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Hypothermia, Induced (1) · Renal Dialysis (1) · Standard of Care (1)
Mendel Tuchman (2) · ModernaTX, Inc. (2) · Children's Hospital of Philadelphia (1) · HemoShear Therapeutics (1) · Uta Lichter-Konecki (1)
United States (6) · Saudi Arabia (3) · Canada (2) · France (2) · Japan (2) · Netherlands (2) · Spain (2) · United Kingdom (2) · Australia (1)
Mendel Tuchman, MD (2) · Marc Yudkoff, MD (1) · Patrick Horn, MD PhD (1) · Uta Lichter-Konecki, MD, PhD (1)
PMID 18414145, PMID 19660971, PMID 20338795, PMID 20566609, PMID 33006765, PMID 36577040
R01HD058567 (1)