49 terminated. 49 Phase 1, 26 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 49 | Terminated 49 |
| Phase 2 | 26 | Terminated 26 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
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 |
|---|---|---|---|---|---|---|
| Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma ADI PEG20, Ifosfamide, Radiotherapy - Soft Tissue Sarcoma, Sts | Phase 1 | Terminated | Washington University School of Medicine | 6 | 2024-03-14 | 2026-09-03 |
| A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency HMI-103 - Phenylketonurias, PAH Deficiency | Phase 1 | Terminated | Homology Medicines, Inc | 3 | 2022-06-03 | 2023-10-10 |
| A Study to Evaluate BBI-001 in Hereditary Haemochromatosis (HH) Patients and Iron Deficient Volunteers BBI-001, Original Fibre Metamucil - Hereditary Hemochromatosis | Phase 1 | Terminated | Bond Biosciences | 24 | 2022-03-28 | 2023-08-02 |
| A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout ALN-XDH, Placebo - Gout | Phase 1 / Phase 2 | Terminated | Alnylam Pharmaceuticals | 44 | 2022-02-25 | 2024-03-29 |
| Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) UX053, Placebo, Antipyretic - Glycogen Storage Disease Type III | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 9 | 2021-10-18 | 2024-04-16 |
| 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 Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency Pegtarviliase IV, Pegtarviliase SC - Homocystinuria Due to Cystathionine Beta-Synthase Deficiency | Phase 1 / Phase 2 | Terminated | Aeglea Biotherapeutics | 13 | 2021-05-13 | 2023-07-27 |
| A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis LYS-GM101 - GM1 Gangliosidosis | Phase 1 / Phase 2 | Terminated | LYSOGENE | 5 | 2021-05-11 | 2023-06-09 |
| 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 |
| A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose - Tay-Sachs Disease, Sandhoff Disease | Phase 1 | Terminated | Terence Flotte | 9 | 2021-01-15 | 2025-09-02 |
| A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2020-09-08 | 2026-09-03 |
| Safety and Tolerability of RTX-134 in Adults With Phenylketonuria RTX-134 - Phenylketonurias | Phase 1 | Terminated | Rubius Therapeutics | 1 | 2020-01-29 | 2022-12-08 |
| Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 VTS-270 - Niemann-Pick Disease, Type C1 | Phase 1 / Phase 2 | Terminated | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 | 2020-01-06 | 2022-08-30 |
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease ABO-102 - MPS IIIA, Sanfilippo Syndrome | Phase 1 / Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 5 | 2019-09-18 | 2023-07-25 |
| A Fabry Disease Gene Therapy Study FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2019-07-08 | 2026-09-03 |
| Gene Therapy Clinical Study in Adult PKU HMI-102 - Phenylketonurias, PAH Deficiency | Phase 1 / Phase 2 | Terminated | Homology Medicines, Inc | 10 | 2019-06-10 | 2023-08-29 |
| Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease AVR-RD-02 - Gaucher Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 8 | 2019-05-30 | 2024-01-18 |
| Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I) TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning - Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma | Phase 1 | Terminated | University of Florida | 11 | 2018-07-17 | 2025-10-29 |
Showing 20 of 49. Sign in to see all of them.
Lysosomal Storage Diseases (10) · Metabolic Diseases (5) · Mucopolysaccharidosis III (5) · Phenylketonurias (5) · Fabry Disease (4) · Mucopolysaccharidosis I (4) · Ornithine Carbamoyltransferase Deficiency Disease (4) · Urea Cycle Disorders, Inborn (4) · Genetic Diseases, Inborn (3) · Methylmalonic acidemia (3) · Mucopolysaccharidoses (3) · Rare Diseases (3) · Adrenoleukodystrophy (2) · Crigler-Najjar Syndrome (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Nutritional and Metabolic Diseases (49) · Genetic Diseases, Inborn (46) · Metabolic Diseases (44) · Nervous System Diseases (29) · Central Nervous System Diseases (28) · Brain Diseases (26) · Brain Diseases, Metabolic (24) · Brain Diseases, Metabolic, Inborn (24) · Lipid Metabolism Disorders (16) · Lipid Metabolism, Inborn Errors (16) · Carbohydrate Metabolism, Inborn Errors (14) · Lysosomal Storage Diseases, Nervous System (14) · Skin and Connective Tissue Diseases (14)
Iduronidase (2) · ADI PEG20 (1) · AT2101 (1) · Abiraterone Acetate (1) · Acetaminophen (1) · Acetylcysteine (1) · Antipyretics (1) · Cetirizine (1) · Cyclophosphamide (1) · Diphtheria-Tetanus Vaccine (1) · Erythromycin (1) · Famotidine (1) · GAA protein, human (1) · GALNS protein, human (1) · Granulocyte-Macrophage Colony-Stimulating Factor (1)
AVROBIO (2) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (2) · Genzyme, a Sanofi Company (2) · Homology Medicines, Inc (2) · Ira Fox (2) · Patricia I. Dickson, M.D. (2) · Sangamo Therapeutics (2) · Spur Therapeutics (2) · Ultragenyx Pharmaceutical Inc (2) · University of Florida (2) · Abeona Therapeutics, Inc (1) · Aeglea Biotherapeutics (1) · Alexion Pharmaceuticals, Inc. (1) · Alnylam Pharmaceuticals (1) · Amicus Therapeutics (1)
United States (39) · United Kingdom (13) · Canada (5) · Spain (5) · Australia (4) · France (4) · Netherlands (4) · Finland (3) · Germany (3) · Italy (3) · Austria (2) · Brazil (2) · Norway (2) · Argentina (1) · Belgium (1)
Ira J Fox, MD (2) · Barry J. Byrne, MD, PhD (1) · Celeste Decker, MD (1) · Cortney Caudill (1) · Curtis Scribner, MD (1) · David E Nix, PharmD (1) · Elias Sayour, MD, PhD (1) · Eugene Schneider, MD (1) · Forbes D Porter, M.D. (1) · Hal Landy, MD (1) · Inderpal Panesar, MRPharmS (1)
PMID 36299240, PMID 37624739, PMID 22547151, PMID 22789058, PMID 25421091, PMID 29526614, PMID 32278382, PMID 32884151, PMID 34600820, PMID 36631545, PMID 37875310, PMID 40817303, PMID 41388232, PMID 41912673
2015 (1), 2018 (1), 2019 (1), P01HL059407 (1), U01HD083493 (1)