Leucine Enriched Essential Amino Acid Powder as an Add-on to a Classic Ketogenic Diet in Refractory Epilepsy

NCT ID: NCT06387186

Last Updated: 2024-04-26

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-30
• Study Completion Date: 2025-03-31

Brief Summary

The main purpose of this study is to evaluate the safety and tolerability of a Leucine-Enriched Essential Amino Acid (LEAA) Powder as an add-on to a classic ketogenic diet (KD) in pediatric and adult patients with refractory epilepsy.

Detailed Description

This is a single center, single-arm, open label study to evaluate the safety, tolerability, and nutritional effect of Leucine-Enriched Essential Amino Acid powder (LEAA) when added to classical KD in pediatric and adult patients whose epilepsy is insufficiently controlled on the diet. The study will enroll patients who have previously been compliant with their classical KD regimen for at least 28 days prior to enrollment and have experienced some response (≥40% reduction in seizures), but not complete response (<90% reduction in seizures) to dietary management with KD. Study duration is 84 days and will have 3 remote study visits (Baseline- Remote Visit 1, Dose Adjustment- Remote Visit 2, and Final- Remote Visit 3) and a pre-screening communication via telephone/video.

Conditions

Refractory Epilepsy; Drug Resistant Epilepsy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Leucine-Enriched Essential Amino Acid powder (LEAA)

Leucine-Enriched Essential Amino Acid powder (LEAA) as an add-on to a classic ketogenic diet (KD)

Group Type: EXPERIMENTAL

Leucine-Enriched Essential Amino Acid powder

Intervention Type: DRUG

Leucine-Enriched Essential Amino Acid Powder will be orally/enterally administered by mixing in water or ketogenic formula.

Interventions

Leucine-Enriched Essential Amino Acid powder

Leucine-Enriched Essential Amino Acid Powder will be orally/enterally administered by mixing in water or ketogenic formula.

Intervention Type: DRUG

Other Intervention Names

LEAA

Eligibility Criteria

Inclusion Criteria

1. Male or female patients ages 2 years and up at enrollment.

2. A diagnosis of epilepsy, with at least 2 seizures per week according to the parent/guardian report to study staff and investigator medical notes if applicable. All seizure types allowed.

3. Under stable treatment with a classic ketogenic diet (KD) regimen with a KD ratio ≥ 1:1, with or without antiseizure medications (ASMs), for at least 28 days with documented, partial response (≥40% reduction in seizures), but not a complete response (<90% reduction in seizures) based on patient medical records or parental report.

4. Patient and/or parent/caregiver able and willing to follow all study procedures and successfully complete the study.

Exclusion Criteria

1. Patients who have changed their KD regimen or antiseizure medication (ASM) or vagus nerve stimulation (VNS) within the last 28 days.

2. Allergy, sensitivity to, or inability to metabolize any component of leucine enriched essential amino acid (LEAA).

3. Those who are pregnant or breastfeeding.

4. Patients who have received an investigational drug or product or participated in a drug study within 4 weeks before the first dose of leucine enriched essential amino acid (LEAA) powder.

5. Patients who have a clinically significant condition or have had either clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Baseline Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the patient including liver disease.

6. Patients with history of suicidal ideation in past 6-months and suicidal behavior in past 2-years

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ajinomoto USA, INC.

INDUSTRY

Sponsor Role: collaborator

Ajinomoto Co., Inc.

INDUSTRY

Sponsor Role: collaborator

Elizabeth Anne Thiele

OTHER

Sponsor Role: lead

Responsible Party

Elizabeth Anne Thiele

Director, Pediatric epilepsy program, Director, Herscot Center for TSC, Professor of Neurology, Harvard Medical School

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Elizabeth Thiele, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Massachusetts General Hospital

Boston, Massachusetts, United States

Countries

United States

Central Contacts

Lydia Tsega, MD, MPH

Role: CONTACT

ltsega@mgh.harvard.edu

6177266524

Carolyn Wilson

Role: CONTACT

cwilson41@mgh.harvard.edu

Facility Contacts

Lydia Tsega, MD, MPH

Role: primary

ltsega@mgh.harvard.edu

617-726-6524

Carolyn Wilson

Role: backup

cwilson41@mgh.harvard.edu

Other Identifiers

2023P003233

Identifier Type: -

Identifier Source: org_study_id