Clinical Trials

Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 40
  • Start Date: January 1, 2022
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy
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Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 1
  • Intervention Type: Drug
  • Participants: 10
  • Start Date: May 1, 2021
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Drug
  • Participants: 12
  • Start Date: May 1, 2021
Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe Disease
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Biological
  • Participants: 20
  • Start Date: January 19, 2021
Endogenous Glucose Production in Subjects With Glycogen Storage Disease Type Ia Estimated by a Single Oral Dose of Stable Isotopes: an Investigator-initiated Human Pilot Study
Clinical Trial
  • Status: Not yet recruiting
  • Intervention Type: Other
  • Participants: 100
  • Start Date: January 10, 2021
Ketogenic Diet Survey in GSDV
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 20
  • Start Date: November 23, 2020
Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1
  • Intervention Type: Drug
  • Participants: 45
  • Start Date: October 16, 2020
A Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia Disorders
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