Amino Acid Supplement for Epilepsy

Overseen By Carolyn Wilson

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Elizabeth Anne Thiele

Must be taking: Ketogenic diet

Disqualifiers: Pregnancy, Liver disease, Suicidal history, others

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

Compensation: Varies

Number of Visits: 3

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety of a special amino acid powder, Leucine-Enriched Essential Amino Acid Powder, when added to the ketogenic diet for individuals with hard-to-treat epilepsy. Researchers mix the powder with the diet to determine its safety and tolerability for those who continue to experience seizures despite current treatment. The trial seeks participants who have epilepsy, experience at least two seizures per week, and are on a stable ketogenic diet that has reduced but not nearly eliminated their seizures. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the trial?

The trial does not require you to stop your current medications. You need to be on a stable ketogenic diet and antiseizure medications for at least 28 days before joining.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that Leucine-Enriched Essential Amino Acid (LEAA) Powder is being explored as a new treatment for epilepsy. The study is in its early stages, focusing on the treatment's safety and tolerability. Researchers carefully monitor the treatment for any side effects, as this is the first step in testing.

While specific safety data for LEAA Powder is not yet available, amino acids like leucine are generally considered safe and are already part of many diets. However, since this is a new treatment for epilepsy, the study will help ensure no unexpected issues arise when used with a ketogenic diet (a high-fat, low-carbohydrate diet).

Prospective participants can be assured that their safety is the top priority. The trial includes detailed monitoring to ensure the treatment's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for epilepsy?

Unlike the standard treatments for epilepsy, such as anti-seizure medications, the leucine-enriched essential amino acid (LEAA) powder works by supporting brain metabolism when used alongside a classic ketogenic diet. This supplement is unique because it provides a specific mix of amino acids, particularly leucine, which might enhance the brain's energy use and stabilize neuronal activity. Researchers are excited about LEAA because it offers a novel approach to seizure management, potentially reducing reliance on medications and their associated side effects.

What evidence suggests that this treatment might be an effective treatment for epilepsy?

Research has shown that the ketogenic diet (KD) can reduce seizures in 50-70% of people with medication-resistant epilepsy. However, it does not work for everyone. This trial will investigate the effects of adding leucine-enriched essential amino acids to the KD. Studies suggest that leucine, a key amino acid, may boost the brain's energy and help control seizures more effectively. This combination could offer a promising option for those needing additional help managing their epilepsy. ^{1 2 3 4 5}

Who Is on the Research Team?

Elizabeth Thiele, MD,PhD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for pediatric and adult patients with epilepsy who experience at least 2 seizures per week, are on a stable classic ketogenic diet (KD) with partial response, and can follow study procedures. It's not for those allergic to LEAA components, recently ill in a way that affects participation, pregnant or breastfeeding individuals, anyone on recent investigational drugs, or those who've changed their KD regimen or seizure medication lately.

Inclusion Criteria

I have epilepsy with 2 or more seizures weekly.

Patient and/or parent/caregiver able and willing to follow all study procedures

I've been on a ketogenic diet with a ratio of at least 1:1 for over 28 days and have seen some improvement.

Exclusion Criteria

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

Clinically significant condition or illness in the 4 weeks prior to Baseline Visit 1 that would negatively impact study participation

Pregnant or breastfeeding patients

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-screening communication via telephone/video

Treatment

Participants receive Leucine-Enriched Essential Amino Acid powder as an add-on to a classic ketogenic diet

12 weeks

3 remote visits (Baseline, Dose Adjustment, Final)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Leucine-Enriched Essential Amino Acid Powder

Trial Overview The trial tests the safety of adding Leucine-Enriched Essential Amino Acid (LEAA) Powder to a classic ketogenic diet in patients with refractory epilepsy. The goal is to see if this add-on helps manage seizures better than the diet alone.

How Is the Trial Designed?

Group I: Leucine-Enriched Essential Amino Acid powder (LEAA) Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Elizabeth Anne Thiele

Lead Sponsor

Trials

2

Recruited

40+

Ajinomoto USA, INC.

Industry Sponsor

Trials

11

Recruited

1,600+

Ajinomoto Co., Inc.

Industry Sponsor

Trials

21

Recruited

1,200+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06387186

Leucine Enriched Essential Amino Acid Powder as an Add- ... 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 ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8017216/

Leucine-Enriched Essential Amino Acids Enhance the ... However, the KD reduces seizures in 50–70% of patients with medically refractory epilepsy, and its antiseizure effect is limited. In the current ...

3. withpower.com withpower.com/trial/phase-1-drug-resistant-epilepsy-3-2024-b99ff

Amino Acid Supplement for Epilepsy · Info for Participants 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 ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/33815043/

Leucine-Enriched Essential Amino Acids Enhance the ... However, the KD reduces seizures in 50-70% of patients with medically refractory epilepsy, and its antiseizure effect is limited. In the current ...

5. semanticscholar.org semanticscholar.org/paper/Leucine-Enriched-Essential-Amino-Acids-Enhance-the-Takeuchi-Nishikata/f70f2d582c86296a6818f3a4757c2b219183fa92

Leucine-Enriched Essential Amino Acids Enhance the ... A new modified KD containing leucine (Leu)-enriched essential amino acids may be a new therapeutic option for patients with epilepsy, including medically ...