drugset / Trial / NCT01461304
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
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.
Timeline
- Start
- —
- Primary completion
- —
- Completion
- —
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Triheptanoin | Other / unclassified | 2 g/kg | Oral |
| Subject | Triheptanoin | Other / unclassified | 4 g/kg | Oral |
Indications
Barth syndrome
acyl-CoA dehydrogenase 9 deficiency
carnitine palmitoyl transferase deficiency
disorder of glycogen metabolism
long chain 3-hydroxyacyl-CoA dehydrogenase deficiency
mitochondrial trifunctional protein deficiency
pyruvate carboxylase deficiency disease
very long chain acyl-CoA dehydrogenase deficiency