Clinical Trials

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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 12
  • Start Date: March 15, 2021
A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Biological
  • Participants: 8
  • Start Date: February 15, 2021
A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
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Clinical Trial
  • Status: Enrolling by invitation
  • Phase: N/A
  • Intervention Type: Drug
  • Participants: 5
  • Start Date: November 28, 2019
Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Drug
  • Participants: 75
  • Start Date: January 15, 2019
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Clinical Trial
  • Status: Active, not recruiting
  • Participants: 97
  • Start Date: March 20, 2018
A Longitudinal, Exploratory, Natural History Study to Further Characterize and Describe the Signs and Symptoms of Patients With Organic Acidemias
Clinical Trial
  • Status: Recruiting
  • Participants: 2275
  • Start Date: June 7, 2004
Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) and Related Disorders
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