NO_LONGER_AVAILABLENot Applicable

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

United States

Plain-language summary

This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.

Who can participate

Age range1 Month

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Ages 1 month and up * Diagnosis of disorder in long chain fatty acid oxidation, glycogen storage disease, pyruvate carboxylase deficiency, or Barth Syndrome * Currently receiving triheptanoin as result of participation in previous study will be eligible if they have one of the included diagnoses * Prefer 2 of following 3: acylcarnitine profile, fibroblast acylcarnitine profile or positive medical genetic test Exclusion Criteria: * Pregnant females * MCAD deficiency * disorder of short and medium chain fatty acid oxidation or ketone body metabolism

Trial details

NCT IDNCT01461304

SponsorJerry Vockley, MD, PhD

Sponsor typeOTHER

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency trials