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Triheptanoin for Diabetes

Phase 1
Waitlist Available
Research Sponsored by Juan Pascual
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 medication taken daily for 7 days.
Awards & highlights

Study Summary

This trial is testing how much of a certain oil people with a certain genetic disorder can tolerate.

Eligible Conditions
  • Diabetes
  • Epilepsy
  • Glucose Transporter Deficiency Syndrome
  • Glut1 Deficiency Syndrome
  • Glucose Transport Defect

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~medication taken daily for 7 days.
This trial's timeline: 3 weeks for screening, Varies for treatment, and medication taken daily for 7 days. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated dose trial

Side effects data

From 2019 Phase 2 trial • 20 Patients • NCT02036853
83%
Diarrhea
33%
Fever
33%
Headache
33%
Stomach Virus
17%
Skin Scale Hands
17%
Rash-Right Leg
17%
Decreased Appetite
17%
Convulsive Seizures
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: TriheptanoinExperimental Treatment1 Intervention
Dose 1 C7 administered as 40% daily caloric intake. Dose 2. C7 administered as 45% daily caloric intake.
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?

Juan PascualLead Sponsor
4 Previous Clinical Trials
19 Total Patients Enrolled
National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,339 Previous Clinical Trials
649,378 Total Patients Enrolled
5 Trials studying Diabetes
8,696 Patients Enrolled for Diabetes

Frequently Asked Questions

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

Who else is applying?

What state do they live in?
Missouri
How old are they?
< 18
What site did they apply to?
UT Southwestern Medical Center
What portion of applicants met pre-screening criteria?
Did not meet criteria
Recent research and studies
Journal of Cerebral Blood Flow & MetabolismJournal
Heptanoate as a Neural Fuel: Energetic and Neurotransmitter Precursors in Normal and Glucose Transporter I-deficient (G1D) Brain
Marin-Valencia, Isaac, Levi B Good, Qian Ma, Craig R Malloy, and Juan M Pascual. 2012. “Heptanoate as a Neural Fuel: Energetic and Neurotransmitter Precursors in Normal and Glucose Transporter I-deficient (G1D) Brain”. Journal of Cerebral Blood Flow & Metabolism. SAGE Publications. doi:10.1038/jcbfm.2012.151.
Pediatric NeurologyJournal
Glucose Transporter Type I Deficiency (G1D) at 25 (1990-2015): Presumptions, Facts, and the Lives of Persons with This Rare Disease
Pascual, Juan M., and Gabriel M. Ronen. 2015. “Glucose Transporter Type I Deficiency (G1D) at 25 (1990-2015): Presumptions, Facts, and the Lives of Persons with This Rare Disease”. Pediatric Neurology. Elsevier BV. doi:10.1016/j.pediatrneurol.2015.08.001.
JAMA NeurologyJournal
Triheptanoin for Glucose Transporter Type I Deficiency (G1D)
Pascual, Juan M., Peiying Liu, Deng Mao, Dorothy I. Kelly, Ana Hernandez, Min Sheng, Levi B. Good, et al.. 2014. “Triheptanoin for Glucose Transporter Type I Deficiency (G1D)”. JAMA Neurology. American Medical Association (AMA). doi:10.1001/jamaneurol.2014.1584.
clinicaltrials.govStudy
Treatment Development of Triheptanoin (G1D)
Juan Pascual 2017. "Treatment Development of Triheptanoin (G1D)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03041363.
~1 spots leftby Apr 2025
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