Treatment Trials

3 Clinical Trials for Various Conditions

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      RECRUITING
      The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
      Description

      This is a multi-center natural history study of Mitochondrial NeurogastroIntestinal Encephalopathy (MNGIE). Patients will be followed over time to assess clinical symptoms. The investigators hope to learn more about the disease of MNGIE as well as develop useful measures of disease status for use in future clinical trials. Additional clinical centers will be listed as they become available.

      Conditions
      Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
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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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      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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