Diazoxide Session with Dr. Paul Thornton

November 3, 2025 • Research, Science with Sandra

Hello and welcome to Science with Sandra!

For this special edition, I would like to share a summary of the virtual meeting with Dr. Paul Thornton, the leading scientist conducting the upcoming Diazoxide clinical trial, which took place on Thursday, October 30th, 2025.

The focus of the meeting was to share information about the upcoming clinical trial. Dr. Thornton began by reviewing GLUT1 Deficiency and the current standard treatment, the ketogenic diet. He noted that one of the main limitations of this treatment is that the diet does not increase the amount of glucose in the brain and that it can be very challenging to follow strictly throughout one’s life.

Dr. Thornton also shared information about Diazoxide, a drug that has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of hyperinsulinism since 1973. Diazoxide works by preventing pancreatic beta cells from releasing insulin, which leads to higher blood glucose levels.

He also discussed some potential side effects that trial participants might experience, including:

Excessive hair growth on the body
Sodium and water retention*
Pulmonary hypertension (breathing and oxygenation problems)*
Low white blood cell levels, increasing the risk of infection
Elevated uric acid levels

*These side effects are less common in young adults.

Although not a side effect, Dr. Thornton mentioned that Diazoxide has a very unpleasant taste, which can sometimes cause nausea.

Study Design and Aims

This is a multiple ascending dose feasibility study to examine the effectiveness of Diazoxide in increasing glucose levels in individuals with GLUT1 Deficiency.

A multiple ascending dose feasibility study is an early-phase clinical trial designed to evaluate the safety, tolerability, and metabolism of a drug when it is given multiple times, typically over several days or weeks.

The aims of the study are to:

Determine if Diazoxide can increase fasting blood glucose levels in people with GLUT1 Deficiency.
Determine if Diazoxide can increase post-meal glucose levels measured by a Mixed Meal Tolerance Test (MMTT).
Determine if Diazoxide can increase the average blood glucose level over a 7-day period, as measured by continuous glucose monitoring (CGM), compared to the patient’s baseline.

Research Participation

Inclusion criteria:

Confirmed diagnosis of GLUT1 Deficiency.
At least 2 years of age.
Not currently following a classical ketogenic diet (patients on the Modified Atkins Diet are eligible).
No known allergies to Diazoxide.

Additional study information:

Participation is voluntary, and patients may withdraw from the study at any time.
Consent and assent will be obtained prior to any research-related activities. Assent forms will be completed by patients between 7 and 17 years of age, depending on cognitive abilities.
Consent and assent forms will be completed by phone before visiting the study site.
Participants may receive up to $100 to help cover costs such as transportation to the study site.
The study will take place at Cook Children’s Hospital in Fort Worth, Texas.

There are currently four participants enrolled, and Dr. Thornton is looking to recruit six to eight additional participants.

Study Timeline

The study lasts approximately 2–3 months. Below is an overview of the timeline:

Baseline: Once participants are ready to begin the trial, they will complete a Mixed Meal Tolerance Test (MMTT)and have baseline bloodwork done. The MMTT assesses how well the body regulates blood sugar after eating a meal.
Visit 1: Two weeks after starting the study, if eligible, participants will begin taking Diazoxide.
Visit 2: Two weeks after Visit 1, participants will return to the study site to take an increased dose of Diazoxide. MMTT and safety labs will be performed.
Visit 3: Two weeks after Visit 2, participants will again take an increased dose of Diazoxide, and another MMTT will be performed.
Visit 4: Two weeks after Visit 3, participants will return for a final MMTT and safety labs.

Throughout the study, participants will wear continuous glucose monitors and self-monitor their glucose levels.

Once the study concludes at Visit 4, participants who see benefits may have the option to continue Diazoxide.

Those interested in participating in the study can contact Dr. Paul Thornton directly at [email protected].

Finally, Dr. Thornton mentioned that this will be an initial study, and depending on the results, he plans to conduct a multicenter, longer-term study with more participants in the future.

I would like to thank Dr. Thornton for taking the time to meet with the community and share important information about this study.

If you’d like to learn more about diazoxide and Dr. Thornton, please see an earlier blog post with more details.

Thank you for visiting the blog, and please contact me at [email protected] if you have any questions.