Treatment Trials

2 Clinical Trials for Various Conditions

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      RECRUITING
      Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
      Description

      The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices

      Conditions
      Homocystinuria Due to CBS Deficiency
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      TERMINATED
      A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency
      Description

      The purpose of this study is to evaluate the safety and tolerability of pegtarviliase in approximately 36 subjects with homocystinuria due to CBS deficiency.

      Conditions
      Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
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