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

> **NCT01461304** · — · NO_LONGER_AVAILABLE · sponsor: **Jerry Vockley, MD, PhD**

## Conditions studied

- 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

## Key facts

- **NCT ID:** NCT01461304
- **Lead sponsor:** Jerry Vockley, MD, PhD
- **Sponsor class:** OTHER
- **Phase:** —
- **Study type:** EXPANDED_ACCESS
- **Status:** NO_LONGER_AVAILABLE
- **Start date:** —
- **Primary completion:** —
- **Final completion:** —
- **Target enrollment:** — (—)
- **Last updated:** 2021-12-10

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01461304

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01461304, "Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT01461304. Licensed CC0.

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