Clinical Trials

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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2/Phase 3
  • Intervention Type: Other, Drug
  • Participants: 60
  • Start Date: September 28, 2020
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Other
  • Participants: 380
  • Start Date: April 3, 2017
Precision Exercise Program in Children With Malignant Hemopathies Undergoing Therapy and/or Hematopoietic Stem Cell Transplantation: The Sport Therapy Project
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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 50
  • Start Date: January 2015
A Phase I/II Clinical Trial of Lentiviral Hematopoietic Stem Cell Gene Therapy for Treatment of Developed Metachromatic Leukodystrophy and Adrenoleukodystrophy
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1
  • Intervention Type: Biological
  • Participants: 12
  • Start Date: September 2014
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug, Biological
  • Participants: 100
  • Start Date: July 10, 2014
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Clinical Trial
  • Status: Recruiting
  • Participants: 50
  • Start Date: January 2014
Natural History Study of Alpers Huttenlocher Syndrome
Clinical Trial
  • Status: Active, not recruiting
  • Phase: Phase 1
  • Intervention Type: Drug
  • Participants: 43
  • Start Date: April 2013
A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Biological, Other
  • Participants: 1500
  • Start Date: January 11, 2012
Longitudinal Study of Neurodegenerative Disorders
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