1 enrolling by invitation, 1 recruiting. 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Enrolling by invitation 1, Recruiting 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 |
| Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment Pegtibatinase, Placebo - Homocystinuria | Phase 3 | Recruiting | Travere Therapeutics, Inc. | 70 | 2023-12-28 | 2026-10-08 |
Amino Acid Metabolism, Inborn Errors (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Genetic Diseases, Inborn (2) · Hyperhomocysteinemia (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Nutritional and Metabolic Diseases (2) · Skin and Connective Tissue Diseases (2)
Travere Therapeutics, Inc. (2)
Australia (2) · Belgium (2) · France (2) · Germany (2) · Ireland (2) · Italy (2) · Poland (2) · Portugal (2) · Qatar (2) · Saudi Arabia (2) · Spain (2) · Turkey (Türkiye) (2) · United Kingdom (2) · United States (2)