9 completed, 7 recruiting, 4 terminated, 1 active not recruiting. 7 Phase 2, 5 Phase 1, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 2, Recruiting 2, Terminated 2, Withdrawn 1 |
| Phase 1 | 5 | Recruiting 3, Completed 1, Terminated 1 |
| Not applicable | 2 | Completed 1, Not yet recruiting 1 |
| Phase 3 | 1 | Completed 1 |
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 |
|---|---|---|---|---|---|---|
| AAVrh10-PCCA Gene Therapy for Propionic Acidemia AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose - Propionic Acidemia | Phase 1 | Recruiting | Mayo Clinic | 9 | 2026-07-20 | 2026-08-21 |
| 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 Retrospective Study to Characterize Participants With Propionic Acidemia Propionic Acidemia | Recruiting | ModernaTX, Inc. | 60 | 2023-06-16 | 2026-02-05 | |
| 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 | |
| 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 | |
| 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 | |
| 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 |
| 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 | |
| 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 |
| 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 |
| 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 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| "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 | |
| Review of Charts From Amish/Mennonite Variant PA Patients Propionic Acidemia | Completed | University of Pittsburgh | 38 | 2017-04-06 | 2026-01-29 | |
| Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia Metabolic Disease, Propionic Acidemia | Recruiting | National Human Genome Research Institute (NHGRI) | 1045 | 2016-11-29 | 2026-10-29 | |
| 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 |
| 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 |
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Methylmalonic acidemia (15) · Metabolism, Inborn Errors (8) · Carbamoyl-Phosphate Synthase I Deficiency Disease (5) · Metabolic Diseases (5) · Genetic Diseases, Inborn (4) · Hyperammonemia (4) · Ornithine Carbamoyltransferase Deficiency Disease (4) · Urea Cycle Disorders, Inborn (4) · Argininosuccinic Aciduria (3) · Citrullinemia (3) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (2) · Acid-Base Imbalance (2) · Acidemia, isovaleric (2) · Acidosis (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Nutritional and Metabolic Diseases (23) · Amino Acid Metabolism, Inborn Errors (21) · Genetic Diseases, Inborn (19) · Metabolic Diseases (18) · Metabolism, Inborn Errors (15) · Nervous System Diseases (8) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Brain Diseases (6) · Pathologic Processes (6) · Pathological Conditions, Signs and Symptoms (6) · Mitochondrial Diseases (5) · Genetic Diseases, X-Linked (4)
carglumic acid (7) · N-carbamylglutamate (2) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Glutamic Acid (1) · Glutamine (1) · Hypothermia, Induced (1) · Registries (1) · Renal Dialysis (1) · Sodium Citrate (1) · Standard of Care (1) · ornithine alpha-ketoglutarate (1)
ModernaTX, Inc. (4) · HemoShear Therapeutics (2) · Mendel Tuchman (2) · RECORDATI GROUP (2) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital of Philadelphia (1) · CoA Therapeutics, Inc., a BridgeBio company (1) · Emma Marie Caroline Slack (1) · King Abdullah International Medical Research Center (1) · Mayo Clinic (1) · National Human Genome Research Institute (NHGRI) (1) · National Taiwan University Hospital (1) · Nicola Longo (1) · Oregon Health and Science University (1) · RTI International (1)
United States (16) · United Kingdom (6) · France (5) · Spain (5) · Canada (4) · Saudi Arabia (4) · Japan (3) · Netherlands (3) · Australia (2) · Italy (2) · Belgium (1) · Germany (1) · Norway (1) · Sweden (1) · Switzerland (1)
Mendel Tuchman, MD (2) · Charles P Venditti, M.D. (1) · Curt Scharfe, MD, PhD (1) · David R. Deyle, MD (1) · Dershung Yang, PhD (1) · Laurent Servais (1) · Lina Ghaloul-Gonzalez, MD (1) · Majid A. Alfadhel, MD (1) · Marc Yudkoff, MD (1) · Ni-Chung Lee, M.D., Ph.D (1) · Nicholas Ah Mew, MD (1) · Nicola Longo, MD, PhD (1) · Patrick Horn, MD PhD (1) · Sufin Yap, MD (1)
PMID 18414145, PMID 19660971, PMID 20338795, PMID 20566609, PMID 28712602, PMID 31196016, PMID 31249402, PMID 31315600, PMID 33006765, PMID 34007002, PMID 35142618, PMID 36577040, PMID 37875310, PMID 39787887, PMID 42839282
R01HD058567 (1), R21DK077415 (1), R44HD107766 (1)