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

NCT01461304 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2021-12-10

No results posted yet for this study

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.

Conditions

  • Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
  • Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)
  • Mitochondrial Trifunctional Protein Deficiency
  • Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency
  • Glycogen Storage Disorders
  • Pyruvate Carboxylase Deficiency Disease
  • ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
  • Barth Syndrome

Interventions

DRUG

triheptanoin

subjects will receive a modified diet containing triheptanoin (up to 2 grams/kg/24 hours; subjects who experience cardiomyopathy may receive doses up to 4 grams/kg/24 hours), or continued on their previously established triheptanoin dose; not to exceed RDA for fat, substituted for their MCT oil and/or natural fat. Study subjects will continue the triheptanoin-supplemented diet for a period of 12 months and then be able to continue into an indefinite extension phase in this compassionate use study. Laboratory evaluations will take place at two, six, and twelve months as well as every 12 months in the extension phase.

Sponsors & Collaborators

Principal Investigators

  • Jerry Vockley, MD, PhD · Children's Hospital of Pittsburgh, University of Pittsburgh

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01461304 on ClinicalTrials.gov