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NCT03041363
Treatment Development of Triheptanoin (G1D)
Phase 1 trial testing Triheptanoin in Epilepsy in 12 participants. Completed in 22 December 2017.
22 December 2017
Quick facts
| Lead sponsor | Juan Pascual |
|---|---|
| Phase | Phase 1 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 12 |
| Start date | 29 March 2017 |
| Primary completion | 22 December 2017 |
| Estimated completion | 22 December 2017 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Triheptanoin — full drug profile →
Conditions studied
- Epilepsy — all drugs for Epilepsy →
- GLUT1DS1 — all drugs for GLUT1DS1 →
- Glut1 Deficiency Syndrome 1, Autosomal Recessive — all drugs for Glut1 Deficiency Syndrome 1, Autosomal Recessive →
- Glucose Metabolism Disorders — all drugs for Glucose Metabolism Disorders →
Sponsor
Juan Pascual
Who can join
Adults 2 to 35, any sex, with Epilepsy or GLUT1DS1. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
To determine the maximum tolerated dose (MTD), as a percentage of calories consumed, of triheptanoin (C7 oil; C7) in a pediatric and adult patient population genetically diagnosed with glucose transporter type 1 deficiency disorder (G1D).
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Glut1 Deficiency Syndrome (Glut1DS): State of the art in 2020 and recommendations of the international Glut1DS study group.
Klepper J, Akman C, Armeno M, Auvin S, et al · · 2020 · cited 186× · PMID 32913944 · DOI 10.1002/epi4.12414 -
Development and validation of a LC-MS/MS method for quantitation of 3-hydroxypentanoic acid and 3-oxopentanoic acid in human plasma and its application to a clinical study of glucose transporter type I deficiency (G1D) syndrome.
Kallem RR, Primeaux S, Avila A, Pascual JM, et al · · 2021 · cited 3× · PMID 34482182 · DOI 10.1016/j.jpba.2021.114335 -
Maximum dose, safety, tolerability and ketonemia after triheptanoin in glucose transporter type 1 deficiency (G1D).
Málaga I, Avila A, Primeaux S, Kallem RR, et al · · 2023 · cited 1× · PMID 36859467 · DOI 10.1038/s41598-023-30578-z
Verify or expand the search:
- PubMed search for NCT03041363
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Triheptanoin
Trials testing the same drug.
- NCT06340685 — Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency · Phase 1 · recruiting
- NCT04513002 — Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis · Phase 2 · completed
- NCT03665636 — Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I · EARLY_PHASE1 · completed
- NCT03642860 — The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses · Phase 2 · completed
- NCT03506425 — A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS) · Phase 1, PHASE2 · completed
Other recruiting trials for Epilepsy
Currently open trials in the same condition.
- NCT07095933 — The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy · EARLY_PHASE1 · recruiting
- NCT07224191 — Hippocampal Oscillations During Exploration · NA · recruiting
- NCT07219407 — A Long-term Study of the Safety and Effectiveness of RAP-219 in Adults With Focal Onset Seizures · Phase 2 · recruiting
- NCT07417280 — LIFUS For Neurological Disorders · NA · recruiting
- NCT07490769 — Levetiracetam Three Times Daily in Epilepsy · Phase 3 · recruiting
Other Juan Pascual trials
Trials by the same sponsor.
- NCT02018302 — Post Study Continuation of C7 for G1D · no longer available
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT03041363 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Juan Pascual
- Last refreshed: 22 December 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03041363.
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