1 terminated, 1 unknown. 2 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Terminated 1, Unknown 1 |
| Phase 2 | 1 | Unknown 1 |
Follow Inborn Errors of Metabolism: new trials by email Literature Review of Inborn Errors of Metabolism trials Research Report on Inborn Errors of Metabolism
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 |
|---|---|---|---|---|---|---|
| N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia N-carbamylglutamate - Inborn Errors of Metabolism | Phase 2 / Phase 3 | Unknown | Mendel Tuchman | 52 | 2008-08-01 | 2020-02-17 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
Metabolism, Inborn Errors (2) · Adrenoleukodystrophy (1) · Canavan Disease (1) · Fucosidosis (1) · Gangliosidosis, GM1 (1) · Hyperammonemia (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Lysosomal Storage Diseases (1) · Mucolipidoses (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis VI (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Nutritional and Metabolic Diseases (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Adrenal Gland Diseases (1) · Adrenal Insufficiency (1) · Bone Diseases (1) · Bone Diseases, Metabolic (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Central Nervous System Diseases (1)
N-carbamylglutamate (1) · carglumic acid (1)
Aldagen (1) · Mendel Tuchman (1)
United States (2)
James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Mendel Tuchman, MD (1)
R01HD058567 (1)