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

Drug Resistant Epilepsy Clinical Trial

Official title:

Open Label, Single-Arm Study of the Safety and Tolerability of a Leucine Enriched Essential Amino Acid Powder as an Add-on to a Classic Ketogenic Diet in Refractory Epilepsy

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06387186
• Other study ID #: 2023P003233
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: April 2024
• Est. completion date: March 31, 2025

Study information

• Verified date: April 2024
• Source: Massachusetts General Hospital
• Contact: Lydia Tsega, MD, MPH
• Phone: 6177266524
• Email: ltsega[@]mgh.harvard.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 15
• Est. completion date: March 31, 2025
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Drug Resistant Epilepsy
• Epilepsy
• Refractory Epilepsy

Intervention

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

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (3)

Lead Sponsor	Collaborator
Elizabeth Anne Thiele	Ajinomoto Co., Inc., Ajinomoto USA, INC.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Changes in seizure frequency as assessed by seizure diary.	Daily record of seizure type, duration, and frequency on seizure diary will be used to measure seizure changes.	Baseline, 3 weeks, 12 weeks
Other	Changes in behavior and cognitive function as assessed by a questionnaire.	Global assessment on behavior and cognitive function questionnaire will be used by study subject/parent/caregiver to assess behavior and cognitive function.	Baseline, 3 weeks, 12 weeks
Other	Changes in seizure severity and overall health as assessed by a questionnaire.	Global assessment on seizure severity, and overall health questionnaire will be used by clinician/dietitian and study subject/parent/caregiver to assess changes in seizure severity and overall health.	Baseline, 3 weeks, 12 weeks
Primary	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	Number of participants with improved nutritional status as assessed by weight.	Nutritional status will be assessed by measuring participants' weight in kilograms.	Baseline, 3 weeks, 12 weeks
Secondary	Number of participants with improved nutritional status as assessed by body mass index (BMI).	Nutritional status will be assessed by measuring participants' weight in kilograms and height in meters to assess their body mass index (BMI).	Baseline, 3 weeks, 12 weeks
Secondary	Number of participants with improved nutritional status as assessed by serum ketone and glucose level.	Serum ketone and glucose level will be measure before and after intake of investigational medicine.	At each visit and on next day