Treatment Trials

2 Clinical Trials for Various Conditions

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      ACTIVE_NOT_RECRUITING
      Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients
      Description

      The purpose of this study is to gather information on the possible symptoms that patients with atypical neuronal ceroid lipofuscinosis type 2 (also known as aTPP1 or atypical tripeptidyl peptidase deficiency) have and how they change over time.

      Conditions
      Neuronal Ceroid-LipofuscinosesNeuronal Ceroid Lipofuscinosis CLN2Spinocerebellar Ataxia, Autosomal Recessive 7
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      WITHDRAWN
      Study of CAD-1883 for Spinocerebellar Ataxia
      Description

      This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA). This study offers the opportunity to understand the safety, tolerability, and efficacy of CAD-1883 in the SCA patient population.

      Conditions
      Spinocerebellar AtaxiasSpinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2Spinocerebellar Ataxia Type 3Spinocerebellar Ataxia Type 6Spinocerebellar Ataxia Type 7Spinocerebellar Ataxia Type 8Spinocerebellar Ataxia Type 10Spinocerebellar Ataxia Type 17ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1
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