Clinical Trials

Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Biological
  • Participants: 36
  • Start Date: April 9, 2021
A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia
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Clinical Trial
  • Status: Not yet recruiting
  • Participants: 62
  • Start Date: March 1, 2021
Noninvasive Biomarkers of Hepatic Fibrosis in Urea Cycle Disorders
Clinical Trial
  • Status: Recruiting
  • Participants: 24
  • Start Date: July 9, 2020
Systemic Biomarkers of Brain Injury From Hyperammonemia
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
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 24
  • Start Date: September 17, 2019
A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of Care
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Dietary Supplement
  • Participants: 6
  • Start Date: July 19, 2019
Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Dietary Supplement
  • Participants: 16
  • Start Date: September 15, 2017
Effect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD)
Clinical Trial
  • Status: Recruiting
  • Participants: 1045
  • Start Date: November 29, 2016
The Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Behavioral, Procedure
  • Participants: 56
  • Start Date: August 2016
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
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