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

NCT06387186 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: 2023P003233
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Refractory Epilepsy; Drug Resistant Epilepsy

Keywords

Refractory Epilepsy; Ketogenic Diet; Leucine-Enriched Essential Amino Acid

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-related adverse events

  Number of participants with treatment-related adverse events as reported by the trial participants.

  Baseline, 3 weeks, 12 weeks

Secondary Outcomes (3)

• Number of participants with improved nutritional status as assessed by weight.

  Baseline, 3 weeks, 12 weeks

• Number of participants with improved nutritional status as assessed by body mass index (BMI).

  Baseline, 3 weeks, 12 weeks

• Number of participants with improved nutritional status as assessed by serum ketone and glucose level.

  At each visit and on next day

Other Outcomes (3)

• Changes in seizure frequency as assessed by seizure diary.

  Baseline, 3 weeks, 12 weeks

• Changes in behavior and cognitive function as assessed by a questionnaire.

  Baseline, 3 weeks, 12 weeks

• Changes in seizure severity and overall health as assessed by a questionnaire.

  Baseline, 3 weeks, 12 weeks

Study Arms (1)

Leucine-Enriched Essential Amino Acid powder (LEAA)

EXPERIMENTAL

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

Drug: Leucine-Enriched Essential Amino Acid powder

Interventions

Leucine-Enriched Essential Amino Acid powder DRUG

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

Also known as: LEAA

Leucine-Enriched Essential Amino Acid powder (LEAA)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients ages 2 years and up at enrollment.
• 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.
• 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.
• Patient and/or parent/caregiver able and willing to follow all study procedures and successfully complete the study.

You may not qualify if:

• Patients who have changed their KD regimen or antiseizure medication (ASM) or vagus nerve stimulation (VNS) within the last 28 days.
• Allergy, sensitivity to, or inability to metabolize any component of leucine enriched essential amino acid (LEAA).
• Those who are pregnant or breastfeeding.
• 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.
• 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.
• Patients with history of suicidal ideation in past 6-months and suicidal behavior in past 2-years

Sponsors & Collaborators

• Elizabeth Anne Thiele: lead
• Ajinomoto USA, INC.: collaborator
• Ajinomoto Co., Inc.: collaborator

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Drug Resistant Epilepsy

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Elizabeth Thiele, MD, PhD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lydia Tsega, MD, MPH

CONTACT

6177266524; ltsega@mgh.harvard.edu

Carolyn Wilson

CONTACT

cwilson41@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director, Pediatric epilepsy program, Director, Herscot Center for TSC, Professor of Neurology, Harvard Medical School

Study Record Dates

• First Submitted: April 16, 2024
• First Posted: April 26, 2024
• Study Start: April 1, 2024
• Primary Completion: March 31, 2025
• Study Completion: March 31, 2025
• Last Updated: April 26, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)