NCT03665636 is a EARLY_PHASE1 clinical trial of Triheptanoin in Glycogen Storage Disease Type I, sponsored by Areeg El-Gharbawy.

Who is sponsoring NCT03665636?

NCT03665636 is sponsored by Areeg El-Gharbawy.

What drugs are being tested in NCT03665636?

Interventions in NCT03665636: Triheptanoin.

What condition does NCT03665636 study?

NCT03665636 studies Glycogen Storage Disease Type I.

Is NCT03665636 still recruiting?

NCT03665636 is currently completed. Last updated 15 December 2021.

Last reviewed 2021-12-15 · How we verify

NCT03665636

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Completed EARLY_PHASE1 Last updated 15 December 2021

What this trial tests

EARLY_PHASE1 trial testing Triheptanoin in Glycogen Storage Disease Type I in 4 participants. Completed in 21 October 2021.

Timeline

16 October 2020

Primary endpoint
21 October 2021

21 October 2021

Quick facts

Lead sponsor	Areeg El-Gharbawy
Phase	EARLY_PHASE1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	16 October 2020
Primary completion	21 October 2021
Estimated completion	21 October 2021
Sites	1 location across United States

Drugs / interventions tested

Triheptanoin — full drug profile →

Conditions studied

Glycogen Storage Disease Type I — all drugs for Glycogen Storage Disease Type I →

Sponsor

Areeg El-Gharbawy

Who can join

Adults 1 Month to 65, any sex, with Glycogen Storage Disease Type I. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study will be an open-label, prospective, interventional feasibility pilot project to study the efficacy, safety, and tolerability of UX007 (triheptanoin) on reducing hypoglycemic events in patients with GSD I. Subjects will serve as their own control. Five (5) subjects who are treatment naïve to UX007 (triheptanoin) and are already on standard dietary therapy for GSDI will be enrolled. The primary objective is to evaluate the efficacy, safety, and tolerability of UX007 (triheptanoin) in patients with GSD I. The secondary objectives include evaluating the effect of UX007 (triheptanoin) on maintaining the duration of normoglycemia between meals based on glucose monitoring (Preventing and reducing the frequency of hypoglycemia); reduction/stabilization of the dose of cornstarch; and the prevention of increased liver steatosis based on ultrasound with elastography.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

A retrospective in-depth analysis of continuous glucose monitoring datasets for patients with hepatic glycogen storage disease: Recommended outcome parameters for glucose management.
Peeks F, Hoogeveen IJ, Feldbrugge RL, Burghard R, et al · · 2021 · cited 31× · PMID 33834518 · DOI 10.1002/jimd.12383
Dynamic Methods for Childhood Hypoglycemia Phenotyping: A Narrative Review.
Rossi A, Rutten MGS, van Dijk TH, Bakker BM, et al · · 2022 · cited 6× · PMID 35789807 · DOI 10.3389/fendo.2022.858832
Endocrine involvement in hepatic glycogen storage diseases: pathophysiology and implications for care.
Rossi A, Simeoli C, Pivonello R, Salerno M, et al · · 2024 · cited 3× · PMID 38556561 · DOI 10.1007/s11154-024-09880-2

Verify or expand the search:

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
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
NCT03301532 — Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D · Phase 2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03665636 (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 Areeg El-Gharbawy
Last refreshed: 15 December 2021

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/NCT03665636.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing