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Triheptanoin for Glut1 Deficiency Syndrome

Phase 2
Waitlist Available
Led By Juan Pascual, MD
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
Diagnosis of glucose transporter type I deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory or by PET scan
Males and females 24 months to 35 years old, inclusive
Must not have
Subjects with a BMI (body mass index) greater than or equal to 30
Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17
Timeline
Screening 3 weeks
Treatment Varies
Follow Up medication taken daily for 6 months.
Awards & highlights
All Individual Drugs Already Approved
No Placebo-Only Group

Summary

This trial will study the effects of C7 supplementation on 45 subjects with G1D who are not receiving any dietary therapy. EEG activity, IQ, language, working memory, processing speed, emotional and behavioral functioning, ataxia, and other neuropsychological and neurological performance indices will be evaluated.

Who is the study for?
This trial is for children and adults aged 2 to 35 with Glucose Transporter Type 1 Deficiency (G1D) confirmed by genotyping or PET scan. They should be on a stable diet without dietary therapy for at least a month. Those overweight, pregnant, breastfeeding, using drugs/alcohol, or with certain medical conditions are excluded.
What is being tested?
The study tests the effects of Triheptanoin (C7 oil) added to regular diets on brain activity and various cognitive functions in G1D patients. It aims to see if this supplement can improve EEG activity, IQ, memory, language skills, emotional state and other neurological functions.
What are the potential side effects?
Potential side effects of Triheptanoin may include stomach pain and diarrhea due to its impact on digestion. Since it's an oil used as a dietary supplement, individual reactions can vary based on tolerance to such nutritional therapies.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with G1D confirmed by genetic testing or a PET scan.
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I am between 2 and 35 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My BMI is 30 or higher.
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I am willing and able to give my consent, or my guardian can consent for me.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~medication taken daily for 6 months.
This trial's timeline: 3 weeks for screening, Varies for treatment, and medication taken daily for 6 months. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Neuropsychological attention scores
Secondary study objectives
Ataxia scores
EEG changes: spike-wave activity duration in EEG (electroencephalogram) tracings
Global impression scale

Side effects data

From 2019 Phase 2 trial • 20 Patients • NCT02036853
83%
Diarrhea
33%
Headache
33%
Fever
33%
Stomach Virus
17%
Rash-Right Leg
17%
Decreased Appetite
17%
Hand Pain
17%
Convulsive Seizures
17%
Lethargy
17%
Skin Scale Hands
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

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: TriheptanoinExperimental Treatment1 Intervention
This is a single arm study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Triheptanoin
FDA approved

Find a Location

Who is running the clinical trial?

National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,378 Previous Clinical Trials
652,147 Total Patients Enrolled
University of Texas Southwestern Medical CenterLead Sponsor
1,083 Previous Clinical Trials
1,058,144 Total Patients Enrolled
Juan Pascual, MDPrincipal InvestigatorStudy Principal Investigator

Media Library

Triheptanoin (Other) Clinical Trial Eligibility Overview. Trial Name: NCT03181399 — Phase 2
Glut1 Deficiency Syndrome Research Study Groups: Triheptanoin
Glut1 Deficiency Syndrome Clinical Trial 2023: Triheptanoin Highlights & Side Effects. Trial Name: NCT03181399 — Phase 2
Triheptanoin (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03181399 — Phase 2
~6 spots leftby Nov 2025
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