1 enrolling by invitation. 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 1 | Enrolling by invitation 1 |
Follow Homocystinuria: new trials by email Literature Review of Homocystinuria trials Research Report on Homocystinuria
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 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) Pegtibatinase - Homocystinuria | Phase 3 | Enrolling by invitation | Travere Therapeutics, Inc. | 100 | 2024-04-30 | 2026-05-28 |
Amino Acid Metabolism, Inborn Errors (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Central Nervous System Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Connective Tissue Diseases (1) · Genetic Diseases, Inborn (1) · Hyperhomocysteinemia (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1) · Nutritional and Metabolic Diseases (1) · Skin and Connective Tissue Diseases (1)
Travere Therapeutics, Inc. (1)
Australia (1) · Belgium (1) · France (1) · Germany (1) · Ireland (1) · Italy (1) · Poland (1) · Portugal (1) · Qatar (1) · Saudi Arabia (1) · Spain (1) · Turkey (Türkiye) (1) · United Kingdom (1) · United States (1)