Ketogenic Diet for Infantile Spasms

Overseen By Heather E Olson, MD, MS

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Heather Olson

Must be taking: Ketogenic diet

Must not be taking: Vigabatrin, ACTH, corticosteroids, others

Disqualifiers: Epileptic spasms, cortical malformation, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 12 months of age or until 3 months after diagnosis of Epileptic Spasms

10 Participants Needed

Trial Summary

What is the purpose of this trial?

Epileptic spasms (ES) are a predominantly infantile seizure type observed frequently in certain genetic disorders. Ketogenic diet (high ratio of fat to carbohydrate/protein) is an established non-medication treatment for difficult to control seizures, including ES. Because ES are associated with worse developmental and cognitive outcomes if not detected or treated quickly and effectively, this trial aims to test the ketogenic diet to prevent ES in this high-risk population. This trial is a single-center pilot study of 10 infants with genetic seizure disorders to establish if the protocol of early ketogenic diet administration and ES evaluation is safe and feasible.

Will I have to stop taking my current medications?

You may need to stop taking certain medications like vigabatrin, ACTH, corticosteroids, topiramate, or zonisamide, as they are not allowed in the trial. However, other anti-seizure medications are permitted.

What data supports the effectiveness of the ketogenic diet treatment for infantile spasms?

Research shows that the ketogenic diet can significantly reduce seizures in children with infantile spasms, with over half of the patients achieving a seizure-free state and many experiencing a major reduction in seizure frequency. The diet is also noted to be well-tolerated and can improve brain activity and development. ^{1 2 3 4 5}

Is the ketogenic diet safe for treating infantile spasms?

The ketogenic diet has been used successfully for treating infantile spasms with fewer side effects compared to some other treatments, suggesting it is generally safe for this condition. ^{1 2 4 6 7}

How is the ketogenic diet treatment different from other treatments for infantile spasms?

The ketogenic diet is unique because it is a high-fat, low-carbohydrate diet that can be used as a first-line treatment for infantile spasms, especially when other treatments like ACTH have failed. It works by producing ketones, such as beta-hydroxybutyrate, which may help stabilize neurons and reduce seizures with fewer side effects. ^{2 4 6 8 9}

Eligibility Criteria

This trial is for infants with genetic seizure disorders, aimed at preventing epileptic spasms (ES) which can lead to developmental and cognitive issues. It's a small study of just 10 babies to see if starting them early on a ketogenic diet—a high-fat, low-carb/protein eating plan—is safe and doable.

Inclusion Criteria

Epilepsy onset at less than 6 months of age

My weight allows for safe blood draws as needed for this study.

I am a baby less than 9 months old.

Exclusion Criteria

I have a brain development disorder.

I have tuberous sclerosis complex or Down syndrome.

I am currently taking medication for seizures.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the ketogenic diet starting within 6 weeks of enrollment, with adherence to EEG testing every 6 weeks

Up to 12 months of age or until 3 months after diagnosis of Epileptic Spasms

Follow-up

Participants are monitored for safety and effectiveness after treatment, including EEG compliance and safety outcomes

Up to 18 months of age

Treatment Details

Interventions

• Ketogenic Diet

Trial Overview The intervention being tested is the ketogenic diet as a preventive measure against epileptic spasms in infants with genetic epilepsy. The study will assess whether this dietary approach can be safely implemented and if it has the potential to prevent ES in these high-risk patients.

Participant Groups

Group I: Ketogenic diet Experimental Treatment 1 Intervention

KetoVie® 4:1 Formula will be administered by oral feeding or gastrostomy tube as indicated. The duration of the diet will be from the time of enrollment up to 12 months of age or until 3 months after a diagnosis of Epileptic Spasms (range 3-15 months).

Who Is Running the Clinical Trial?

Heather Olson

Lead Sponsor

Trials

1

Recruited

10+

Boston Children's Hospital Translational Research Program (TRP) Pappendick Family Therapeutic Acceleration Award

Collaborator

Trials

1

Recruited

10+

References

1. United States pubmed.ncbi.nlm.nih.gov

Efficacy of the ketogenic diet for infantile spasms. [2022]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Efficacy of Ketogenic Diet for Infantile Spasms in Chinese Patients With or Without Monogenic Etiology. [2022]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Prognostic factors of infantile spasms: role of treatment options including a ketogenic diet. [2017]

4. Iran pubmed.ncbi.nlm.nih.gov

The Efficacy of the Ketogenic Diet in Improving Seizures and EEG Findings in Patients with Refractory Infantile Spasms. [2023]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Ketogenic diet for treatment of infantile spasms. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Infantile spasms treated with the ketogenic diet: prospective single-center experience in 104 consecutive infants. [2017]

7. India pubmed.ncbi.nlm.nih.gov

Modified Atkins Diet vs. Ketogenic Diet in the Management of Children with Epileptic Spasms Refractory to First Line Treatment: An Open Labelled, Randomized Controlled Trial. [2023]

8. Netherlands pubmed.ncbi.nlm.nih.gov

β-Hydroxybutyrate attenuates NMDA-induced spasms in rats with evidence of neuronal stabilization on MR spectroscopy. [2015]

9. Netherlands pubmed.ncbi.nlm.nih.gov

Ketogenic diet for infantile spasms refractory to first-line treatments: an open prospective study. [2017]