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

  
3 trials found
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1ChiCTR2100054613
2022-08-012021-12-21Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuriaEffects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria Methylmalonic acidemiaExperimental group:PEG-rhGH;Control Group:None;Zhengzhou Children's HospitalNULLPending03BothExperimental group:30;Control Group:30;Phase 4China
2NCT05154890
(ClinicalTrials.gov)
May 13, 202120/8/2021A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS DeficiencyA 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 ACN00177Homocystinuria Due to Cystathionine Beta-Synthase DeficiencyDrug: PegtarviliaseAeglea BiotherapeuticsNULLRecruiting12 YearsN/AAll36Phase 1/Phase 2United States;Australia;United Kingdom
3NCT03406611
(ClinicalTrials.gov)
January 22, 20195/12/2017Pegtibatinase 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)HomocystinuriaDrug: Pegtibatinase;Drug: PlaceboTravere Therapeutics, Inc.NULLRecruiting12 Years65 YearsAll32Phase 1/Phase 2United States