Status | Study |
Completed |
Study Name: A Long-Term Extension Study of RP103-MITO-001 (NCT02023866) to Assess RP103 in Children With Inherited Mitochondrial Disease Condition: Mitochondrial Diseases Date: 2015-06-10 Interventions: Drug: Cysteamine Bitartrate Delayed-release Capsules Treatment with experimental drug as detailed in arm |
Recruiting |
Study Name: MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study Condition: Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) Date: 2015-02-10 Interventions: Biological: Hematopoietic Allogeneic Stem Cells HLA 10/10 matched allogeneic bone marrow cells will be i |
Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Hunter Syn Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |
Recruiting |
Study Name: The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) Condition: Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) Date: 2012-09-25 |
Recruiting |
Study Name: North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) Condition: Mitochondrial Disorders Mitochondrial Genetic Disorders Mi Date: 2012-09-25 |
Enrolling by invitation |
Study Name: Tissue Sample Study for Mitochondrial Disorders Condition: Mitochondrial Disorders Mitochondrial Disease Melas Date: 2012-08-31 |