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 | ChiCTR2100054613 | 2022-08-01 | 2021-12-21 | Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria | Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria | Methylmalonic acidemia | Experimental group:PEG-rhGH;Control Group:None; | Zhengzhou Children's Hospital | NULL | Pending | 0 | 3 | Both | Experimental group:30;Control Group:30; | Phase 4 | China |
| 2 | NCT05154890 (ClinicalTrials.gov) | May 13, 2021 | 20/8/2021 | A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency | A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 | Homocystinuria Due to Cystathionine Beta-Synthase Deficiency | Drug: Pegtarviliase | Aeglea Biotherapeutics | NULL | Recruiting | 12 Years | N/A | All | 36 | Phase 1/Phase 2 | United States;Australia;United Kingdom |
| 3 | 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 |