What side effects are associated with this type of intervention?

Common treatments for Glucose Transporter Deficiency Syndrome (G1D) include ketogenic diets and medium-chain triglyceride (MCT) supplementation, such as Triheptanoin (C7 oil). Known side effects of these treatments can include gastrointestinal issues like diarrhea, abdominal pain, and nausea. Additionally, ketogenic diets may lead to complications such as hypoglycemia, hyperlipidemia, and nutrient deficiencies if not properly managed. It is important for patients to be closely monitored by healthcare professionals to mitigate these risks.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Glucose Transporter Deficiency Syndrome (G1D) that function in the same way as Triheptanoin (C7 oil). Triheptanoin is being studied for its potential to improve brain energy metabolism by providing medium-chain triglycerides that can be converted into ketone bodies, offering an alternative energy source to the brain. Current management of G1D often involves dietary modifications, such as a ketogenic diet, to increase ketone body production and support brain energy metabolism.

What other types of research exist?

Currently, no specific novel alternatives to Triheptanoin (C7 oil) for Glucose Transporter Deficiency Syndrome (G1D) are mentioned in the provided context. Patients should consult with their healthcare provider to explore potential treatments that may include other forms of medium-chain triglycerides (MCTs) or ketogenic diet modifications that can similarly improve brain energy metabolism.

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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

No Placebo-Only Group

Summary

This trial is testing whether a special oil called triheptanoin can be safely added to a high-fat diet for patients with a specific condition. These patients already follow a high-fat diet to help manage their condition. The study will monitor brain activity and seizure rates to see if the oil helps improve their treatment. Triheptanoin has been used for the treatment of certain metabolic conditions and has shown potential in treating other metabolic and neurological diseases.

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.

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.

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1, day 4

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in Ketosis (Beta-hydroxybutyrate Levels)

Secondary study objectives

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%

Headache

33%

Fever

33%

Stomach Virus

17%

Decreased Appetite

17%

Rash-Right Leg

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%

Study treatment Arm

Schedule A

Schedule B

Awards & Highlights

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: 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

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Glucose Transporter Deficiency Syndrome (G1D) include the ketogenic diet and triheptanoin (C7 oil). The ketogenic diet induces ketosis, allowing the body to use ketone bodies instead of glucose for energy, which is essential for G1D patients who have impaired glucose transport to the brain. Ketone bodies can cross the blood-brain barrier and provide an alternative energy source, potentially improving brain function and reducing seizures. Triheptanoin (C7 oil) is a medium-chain triglyceride that can be converted into ketone bodies, offering a similar benefit by supplying an alternative energy source to the brain, thereby potentially improving brain energy metabolism and neurological outcomes in G1D patients.

Triheptanoin alters [U-13C6]-glucose incorporation into glycolytic intermediates and increases TCA cycling by normalizing the activities of pyruvate dehydrogenase and oxoglutarate dehydrogenase in a chronic epilepsy mouse model.Cucurbitane Triterpenoids from the Fruits of Momordica Charantia Improve Insulin Sensitivity and Glucose Homeostasis in Streptozotocin-Induced Diabetic Mice.The hepatocyte glucose-6-phosphatase subcomponent T3: its relationship to GLUT2.