Treatment Trials

4 Clinical Trials for Various Conditions

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      SUSPENDED
      Natural History Study of MNGIE
      Description

      Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)

      Conditions
      Mitochondrial Neurogastrointestinal Encephalomyopathy
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      TERMINATED
      A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
      Description

      A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study RP103-MITO-001 (NCT02023866).

      Conditions
      Mitochondrial Diseases
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      RECRUITING
      North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
      Description

      The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.

      Conditions
      Mitochondrial DisordersMitochondrial Genetic DisordersMitochondrial DiseasesDisorder of Mitochondrial Respiratory Chain ComplexesDeletion and Duplication of Mitochondrial DNA
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      COMPLETED
      Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
      Description

      To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.

      Conditions
      Inherited Mitochondrial Disease, Including Leigh Syndrome
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