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Found 4 clinical trials
A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD

The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).

  • 20 Sep, 2023
  • 4 locations
Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) Online Disease Monitoring Program (DMP)

The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or caregivers) to self-report information.

  • 10 Oct, 2022
  • 1 location
Systemic Biomarkers of Brain Injury From Hyperammonemia

Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxic brain edema and vascular compromise leading to intellectual and developmental disabilities. The following aims are proposed: Aim 1 of this study will …

  • 08 Nov, 2021
  • 1 location
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.

  • 20 Sep, 2023
  • 8 locations