Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
Public ClinicalTrials.gov record NCT01461304. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Dietary Therapy for Inherited Disorders of Energy Metabolism
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT01461304
- Recruitment status
- No Longer Available
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
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
Eligibility (public fields only)
- Age range
- 1 Month and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Dec 9, 2021
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Pittsburgh Division of Medical Genetics, Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01461304, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 9, 2021 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01461304 live on ClinicalTrials.gov.