Recruiting
Phase 1

Ketogenic Diet

Sponsor:

Heather Olson

Code:

NCT06700811

Conditions

Developmental and Epileptic Encephalopathies

Epileptic Spasms

Genetic Epilepsy

Neonatal and Infant Epilepsy

Eligibility Criteria

Sex: All

Age: 0

Healthy Volunteers: Not accepted

Interventions

Ketogenic diet

Study Details

Brief summary:

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 suspected or confirmed genetic seizure disorders to establish if the protocol of early ketogenic diet administration and ES evaluation is safe and feasible.

Conditions

Developmental and Epileptic Encephalopathies

Epileptic Spasms

Genetic Epilepsy

Neonatal and Infant Epilepsy

Study ID

NCT06700811

Start date

Aug 11, 2025

Status verified date

Jul, 2026

Completion date

May, 2028

Anticipated

Primary completion date

Nov, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Plan for initiation of ketogenic diet by clinical team for treatment of epilepsy
  • The clinical team initiating the ketogenic diet agrees that the use of the KetoVie formula is appropriate for the subject, as all study subjects need to receive the same formula
  • Male or female, age 0 to less than 9 months (including neonates per investigator's judgment)
  • Epilepsy onset at less than 6 months of age
  • Abnormal development (any sub score of the Bayley-4 less than 1 standard deviation below the mean) and/or neurologic exam (microcephaly, macrocephaly, strabismus, abnormal vision/CVI, hypotonia, spasticity, dystonia, movement disorder), per investigators judgment
  • Suspected or confirmed genetic diagnosis as a cause for epilepsy
  • Weight adequate to complete required study laboratory testing without exceeding maximum allowable blood draws per draw or in a 30 day period per BCH policy

Exclusion Criteria:

  • Epileptic spams prior to enrollment
  • Tuberous sclerosis complex, trisomy 21 (based on differential response to ES treatment)
  • Metabolic diagnosis with targeted treatment (including specific indication for ketogenic diet such as glucose transporter disorder, vitamin dependent epilepsies, and others) or exclusion for the ketogenic diet
  • Ongoing treatment with vigabatrin, ACTH, corticosteroids, topiramate or zonisamide. Other anti-seizure medications are permitted.

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

experimental: Ketogenic diet

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).

Interventions

Ketogenic diet

The ketogenic diet formula will be KetoVie®, supplied by Ajinomoto Cambrooke. Ketogenic diet ratio will aim to achieve ketosis, with a minimum level of beta-hydroxybutyrate of 1.0mmol/L. Ratios of ketogenic diet generally range from 1:1 to 4:1. Ratio will increase per standard clinical care for ketogenic diet initiation. For the trial we will aim to reach a maximum of 4:1 by the 6 week follow-up visit, but stopping at a lower ratio if BHB is ≥ 5mmol/L, CO2 ≤ 18 mmol/L, for tolerance, or to meet protein needs. A minimum 1:1 ratio is required to continue in the trial. Ratios higher than 1:1 are often required to obtain ketosis of ideally 2-5mmol/L in infants.

Primary outcome measure

  • Achievement of ketosis [ Time Frame: rom ketogenic diet initiation until 3 months after the diagnosis of ES (if it occurs) or until 12 months of age ]
  • EEG compliance [ Time Frame: From ketogenic diet initiation until the onset of ES (if it occurs) or until 12 months of age ]

Central Contacts and Locations

Central contacts

Heather E Olson, MD, MS

617-355-7970Heather.Olson@childrens.harvard.edu

Locations

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Jessica Landers, MS

617-355-0578jessica.landers@childrens.harvard.edu

More Information

Sponsor

Heather Olson

Last update posted

Jul 6, 2026

Last verified

Jul, 2026

Keywords

  • Ketogenic diet
  • Epileptic spasms prevention
  • epilepsy
  • infantile spasms
  • epileptic spasms
  • seizures
  • infant
  • eeg
  • open label
  • Phase 1
  • dietary treatments
  • developmental and epileptic encephalopathy
  • genetic
  • prevention
  • epileptic encephalopathy
  • refractory epilepsy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Heather Olson on 2026-07-06.