3 active not recruiting. 3 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Early Phase 1 | 3 | Active not recruiting 3 |
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 |
|---|---|---|---|---|---|---|
| A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease LY-M001 - Gaucher Disease | Early Phase 1 | Active not recruiting | Shanghai Jiao Tong University School of Medicine | 3 | 2024-08-01 | 2026-08-10 |
| Safety and Efficacy Study of NGGT002 in PKU Adult Subjects NGGT002 Injection - Phenylketonurias | Early Phase 1 | Active not recruiting | The First Affiliated Hospital of Bengbu Medical University | 15 | 2023-03-30 | 2026-08-06 |
| Cholesterol Disruption in Combination With the Standard of Care in Patients With Advanced Pancreatic Adenocarcinoma Cholesterol metabolism disruption - Pancreatic Ductal Adenocarcinoma, Pancreatic Cancer | Early Phase 1 | Active not recruiting | CHU de Quebec-Universite Laval | 3 | 2021-10-04 | 2026-03-19 |
Gaucher Disease (1) · Hyperlipoproteinemia Type II (1) · Neoplasm Metastasis (1) · Neoplasms (1) · Pancreatic Neoplasms (1) · Phenylketonurias (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Amino Acid Metabolism, Inborn Errors (1) · Digestive System Diseases (1) · Digestive System Neoplasms (1)
Atorvastatin (1) · Ezetimibe (1) · evolocumab (1)
CHU de Quebec-Universite Laval (1) · Shanghai Jiao Tong University School of Medicine (1) · The First Affiliated Hospital of Bengbu Medical University (1)
China (2) · Canada (1)
Anne Gangloff, MD PhD FRCPC (1) · Anne-Marie Carreau, MD FRCPC (1) · Francine Aubin, MD FRCPC (1) · Nabil G. Seidah, PhD (1) · Olivier Dumas, MD FRCPC (1) · Vincent Castonguay, MD FRCPC (1)