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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1NCT05154890
(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