NCT03301532

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2018

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 29, 2017

Completed
1 month until next milestone

First Posted

Study publicly available on registry

October 4, 2017

Completed
8 months until next milestone

Study Start

First participant enrolled

June 5, 2018

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 18, 2021

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2022

Completed
1.5 years until next milestone

Results Posted

Study results publicly available

January 17, 2024

Completed
Last Updated

January 17, 2024

Status Verified

December 1, 2023

Enrollment Period

3 years

First QC Date

August 29, 2017

Results QC Date

November 9, 2023

Last Update Submit

December 22, 2023

Conditions

GLUT1DS1

Keywords

G1D, glucose, transporter

Outcome Measures

Primary Outcomes (1)

  • Change in Ketosis (Beta-hydroxybutyrate Levels)

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

    Day 1, Day 4

Secondary Outcomes (2)

  • Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate

    Baseline (Day 1) - Day 4

  • Change in Glycemia

    Day 1 - Day 4

Study Arms (1)

Patients on a ketogenic diet

EXPERIMENTAL

This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.

Drug: Triheptanoin

Interventions

TriheptanoinDRUG

Dietary supplementation with triheptanoin

Also known as: C7 oil
Patients on a ketogenic diet

Eligibility Criteria

Age30 Months - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.
  • Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.
  • Males and females 30 months to 35 years and 11 months old inclusive.

You may not qualify if:

  • Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
  • Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.
  • Subjects with a BMI (body mass index) greater than or equal to 30.
  • Subjects currently not on ketogenic diet.
  • Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.
  • Allergy/sensitivity to C7
  • Previous use of triheptanoin less than 1 month prior to study initiation.
  • Treatment with medium chain triglycerides in the last 24 hours.
  • Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.
  • Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.
  • Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17,
  • Addition of a new antiseizure drug in the previous 3 months.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UTexasSouthwestern

Dallas, Texas, 75390, United States

Location

Related Publications (1)

  • Avila A, Malaga I, Sirsi D, Kayani S, Primeaux S, Kathote GA, Jakkamsetti V, Kallem RR, Putnam WC, Park JY, Shinnar S, Pascual JM. Combination of triheptanoin with the ketogenic diet in Glucose transporter type 1 deficiency (G1D). Sci Rep. 2023 Jun 2;13(1):8951. doi: 10.1038/s41598-023-36001-x.

    PMID: 37268656RESULT

MeSH Terms

Interventions

triheptanoin

Results Point of Contact

Title
Dr. Juan Pascual
Organization
University of Texas Southwestern Medical Center
juan.pascual@utsouthwestern.edu
214-648-3550

Study Officials

  • Juan Pascual, M.D.

    Study Principal Investigator

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Medicine

Study Record Dates

First Submitted

August 29, 2017

First Posted

October 4, 2017

Study Start

June 5, 2018

Primary Completion

June 18, 2021

Study Completion

July 31, 2022

Last Updated

January 17, 2024

Results First Posted

January 17, 2024

Record last verified: 2023-12

Locations

US(1)