NCT03301532

Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

Completed Phase 2 Results posted Last updated 17 January 2024

What this trial tests

Phase 2 trial testing Triheptanoin in GLUT1DS1 in 10 participants. Completed in 31 July 2022.

Timeline

5 June 2018

Primary endpoint
18 June 2021

31 July 2022

Quick facts

Lead sponsor	University of Texas Southwestern Medical Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	5 June 2018
Primary completion	18 June 2021
Estimated completion	31 July 2022
Sites	1 location across United States

Drugs / interventions tested

• Triheptanoin — full drug profile →

Conditions studied

• GLUT1DS1 — all drugs for GLUT1DS1 →

Sponsor

University of Texas Southwestern Medical Center

Who can join

Adults 30 Months to 35, any sex, with GLUT1DS1. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change in Ketosis (Beta-hydroxybutyrate Levels) Primary · Day 1, Day 4

Change in Ketosis as measured by changes in blood beta-hydroxybutyric acid levels.

Group	Value	95% CI
Ketosis	3.82	± 1.9

Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate Secondary · Baseline (Day 1) - Day 4

The percent change in observable seizure rate is measured as the change in observable seizure numbers from baseline to Day 4. Seizure rate is defined as count of seizures per patient per day.

Group	Value	95% CI
Fraction of Subjects With a Change in Seizure Rate	3

Change in Glycemia Secondary · Day 1 - Day 4

Change in Glycemia is measured as changes in blood glucose levels.

Group	Value	95% CI
Glycemia	81.2	± 13.6

Adverse events — posted to ClinicalTrials.gov

Time frame: Days 1 - 4. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ketosis

Serious: 0/10 (0%)

Deaths: 0/10

Other adverse events (1 terms — click to expand)

Reaction	System	Ketosis
Seizures	Nervous system disorders	—

Data from ClinicalTrials.gov NCT03301532 adverse events section.

Sponsor's own description

To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.

Publications & conference data

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

1. 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
2. Triheptanoin: First Approval.
   Shirley M. · · 2020 · cited 26× · PMID 32897506 · DOI 10.1007/s40265-020-01399-5
3. 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
4. Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D).
   Avila A, Málaga I, Sirsi D, Kayani S, et al · · 2023 · cited 2× · PMID 37268656 · DOI 10.1038/s41598-023-36001-x