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

  
1 trial found
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enrollment
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agemin
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agemax
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PhaseCountries
1NCT03406611
(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