Ignite Creation Date:
2025-12-24 @ 8:03 PM
Ignite Modification Date:
2026-05-29 @ 12:36 PM
Study NCT ID:
NCT01461304
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2021-12-10
First Post:
2011-10-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
Sponsor:
Jerry Vockley, MD, PhD
Organization:
Conditions & Keywords Data
Conditions:
Conditions
| Condition Brief | Condition Text | View |
|---|---|---|
| None | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | View |
| None | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | View |
| None | Mitochondrial Trifunctional Protein Deficiency | View |
| None | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | View |
| None | Glycogen Storage Disorders | View |
| None | Pyruvate Carboxylase Deficiency Disease | View |
| None | ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of | View |
| None | Barth Syndrome | View |
Keywords:
Keywords
| Keyword Brief | Keyword Text | View |
|---|---|---|
| None | fatty acid oxidation | View |
| None | triheptanoin | View |
| None | long-chain fatty acid oxidation disorders | View |
| None | LCHADD | View |
| None | VLCADD | View |
| None | CPT1 | View |
| None | CPT2 | View |
| None | TFP | View |
| None | Glycogen Storage Disorders | View |
| None | GSD | View |
| None | Pyruvate Carboxylase | View |
| None | PC | View |
| None | ACAD9 | View |
| None | Barth | View |