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Triheptanoin + Ketogenic Diet for Glucose Transporter Deficiency Syndrome

Phase 2
Waitlist Available
Led By Juan Pascual, M.D.
Research Sponsored by University of Texas Southwestern Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1, day 4
Awards & highlights

Study Summary

This trial is testing whether a ketogenic diet is compatible with triheptanoin oil, by looking at EEG readings, seizure rate, blood sugar, and ketosis levels in patients who have a condition that causes seizures.

Who is the study for?
This trial is for people aged 2.5 to almost 36 years with Glucose Transporter Deficiency Syndrome (G1D) confirmed by genetic testing, who have been on a stable ketogenic diet. It excludes those with other metabolic/genetic diseases, gastrointestinal disorders, obesity (BMI ≥30), not on a ketogenic diet, pregnant or breastfeeding women, and those unwilling to prevent pregnancy.Check my eligibility
What is being tested?
The study tests how well triheptanoin oil (C7) works with the ketogenic diet in G1D patients by monitoring brain activity through EEGs, seizure frequency, blood sugar levels and ketosis state without changing their current diet ratios.See study design
What are the potential side effects?
Potential side effects of C7 are not detailed here but may include digestive issues like diarrhea or stomach pain based on exclusion criteria related to gastrointestinal conditions.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1, day 4
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1, day 4 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Ketosis (Beta-hydroxybutyrate Levels)
Secondary outcome measures
Change in Glycemia
Fraction of Subjects (of a Total of 10 Studied) Who Exhibit a Change in Observable Seizure Rate

Side effects data

From 2019 Phase 2 trial • 20 Patients • NCT02036853
83%
Diarrhea
33%
Fever
33%
Headache
33%
Stomach Virus
17%
Convulsive Seizures
17%
Skin Scale Hands
17%
Rash-Right Leg
17%
Decreased Appetite
17%
Lethargy
17%
Hand Pain
17%
Tongue Pain
17%
Gastric Reflux
17%
Urinary Incontinence
17%
Amenorrhea
17%
Otalgia
17%
Hirsutism
17%
Abdominal Discomfort
17%
Constipation
17%
Emesis
17%
Nausea
17%
Nasal Fracture
17%
Right Arm Fracture
17%
Decreased Hematocrit
17%
Weight Gain
17%
Seizure
17%
Insomnia
17%
Moodiness
17%
Upper Respiratory Infection
17%
Impetigo
17%
Onychomycosis
17%
Rhinovirus
100%
80%
60%
40%
20%
0%
Study treatment Arm
Schedule A
Schedule B

Trial Design

1Treatment groups
Experimental Treatment
Group I: Patients on a ketogenic dietExperimental Treatment1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Triheptanoin
2020
Completed Phase 2
~200

Find a Location

Who is running the clinical trial?

National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,339 Previous Clinical Trials
649,296 Total Patients Enrolled
University of Texas Southwestern Medical CenterLead Sponsor
1,047 Previous Clinical Trials
1,053,789 Total Patients Enrolled
Juan Pascual, M.D.Principal Investigator - Study Principal Investigator
University of Texas Southwestern Medical Center

Media Library

Triheptanoin (Other) Clinical Trial Eligibility Overview. Trial Name: NCT03301532 — Phase 2
Glucose Transporter Deficiency Syndrome Research Study Groups: Patients on a ketogenic diet
Glucose Transporter Deficiency Syndrome Clinical Trial 2023: Triheptanoin Highlights & Side Effects. Trial Name: NCT03301532 — Phase 2
Triheptanoin (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03301532 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the age requirement for this research permit individuals over forty to participate?

"This medical trial seeks participants between the age of 30 months and 35 years old."

Answered by AI

Has the FDA sanctioned Triheptanoin for therapeutic use?

"Triheptanoin was evaluated as a 2 on the safety scale due to its status as a Phase 2 trial, which exhibits evidence of some protective properties yet fails to provide any proof of efficacy."

Answered by AI

Are there any openings for qualification in this research project?

"This trail has concluded its recruitment period. It was first posted on June 5th 2018 and the last update to the information occurred on December 29th 2021. If you are seeking alternative medical trials, there are 2 studies recruiting for Glut1DS1 patients and 3 clinical trials actively looking for participants with Triheptanoin related conditions."

Answered by AI

Am I eligible to take part in this investigation?

"This trial is recruiting 10 individuals in the age range of 30 months to 35 years and 11 months with glut1ds1. The chief requisites are that participants must be boys or girls, have been on a ketogenic diet at a 2.5:1 to 4:1 ratio for two months without any changes prior to joining this research study."

Answered by AI

How many participants are taking part in this medical experiment?

"This clinical trial has ceased its recruitment efforts. It was initially posted on June 5th, 2018, and the latest update to the listing occurred December 29th 2021. For those still looking for trials related to Glut1DS1 or Triheptanoin there are presently 2 and 3 studies respectively currently recruiting patients."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
Juan Pascual 2018. "Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03301532.
~1 spots leftby Apr 2025
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