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

NCT01461304 · clinicaltrials.gov ↗

NO_LONGER_AVAILABLE

Status

OTHER

Sponsor class

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

Sponsor

Jerry Vockley, MD, PhD

Collaborators

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