337. ホモシスチン尿症 Homocystinuria Clinical trials / Disease details
臨床試験数 : 16 / 薬物数 : 19 - (DrugBank : 7) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 23
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03406611 (ClinicalTrials.gov) | January 22, 2019 | 5/12/2017 | Pegtibatinase as an Enzyme Therapy for Patients With Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) | A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058), Administered Subcutaneously in Patients With Cystathionine Beta-Synthase Deficient Homocystinuria (COMPOSE) | Homocystinuria | Drug: Pegtibatinase;Drug: Placebo | Travere Therapeutics, Inc. | NULL | Recruiting | 12 Years | 65 Years | All | 32 | Phase 1/Phase 2 | United States |