Refractory EpilepsyPhase 1NCT06387186
Leucine Enriched Essential Amino Acid Powder as an Add-on to a Classic Ketogenic Diet in Refractory Epilepsy
A Phase 1 study testing Leucine-Enriched Essential Amino Acid powder in people with Refractory Epilepsy and Drug Resistant Epilepsy, run by Elizabeth Anne Thiele at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Refractory Epilepsy, Drug Resistant Epilepsy
- What is being tested?
- Leucine-Enriched Essential Amino Acid powder
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 10 May 2024to 18 Feb 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
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
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.
Read the 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
- Being tested
- Leucine-Enriched Essential Amino Acid powder
- Keywords
- Refractory Epilepsy, Ketogenic Diet, Leucine-Enriched Essential Amino Acid
- Medical subject headings
- Drug Resistant Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Leucine-Enriched Essential Amino Acid powderDrug
Also called LEAA
Leucine-Enriched Essential Amino Acid Powder will be orally/enterally administered by mixing in water or ketogenic formula.
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Leucine-Enriched Essential Amino Acid powder |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events
Number of participants with treatment-related adverse events as reported by the trial participants.
Measured: Baseline, 3 weeks, 12 weeks
Secondary outcomes (3)
Number of participants with improved nutritional status as assessed by weight.
Nutritional status will be assessed by measuring participants' weight in kilograms.
Measured: Baseline, 3 weeks, 12 weeks
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).
Measured: Baseline, 3 weeks, 12 weeks
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.
Measured: At each visit and on next day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2024
- Study start10 May 2024
- Primary completion18 Feb 2026
- Study completion18 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory EpilepsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elizabeth Anne Thiele
- Sponsor type
- University, hospital or other
- Collaborators
- Ajinomoto USA, INC., Ajinomoto Co., Inc.
- Responsible party
- Sponsor-investigator, Elizabeth Anne Thiele, Massachusetts General Hospital
- Investigators
- Elizabeth Thiele, MD, PhD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06387186
- Sponsor's protocol ID
- 2023P003233
- Status last verified
- May 2026
- First submitted
- 16 Apr 2024
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06387186
- Acronym / sponsor ID
- 2023P003233
- Conditions
- Refractory Epilepsy, Drug Resistant Epilepsy
- Keywords
- Refractory Epilepsy, Ketogenic Diet, Leucine-Enriched Essential Amino Acid
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Elizabeth Anne ThieleUniversity, hospital or other
- Collaborators
- Ajinomoto USA, INC., Ajinomoto Co., Inc.
- Study start
- 10 May 2024 (actual)
- Primary completion
- 18 Feb 2026 (actual)
- Study completion
- 18 Feb 2026 (actual)
- First posted
- 26 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Leucine-Enriched Essential Amino Acid powder (LEAA) | Experimental | Drug: Leucine-Enriched Essential Amino Acid powderLeucine-Enriched Essential Amino Acid powder (LEAA) as an add-on to a classic ketogenic diet (KD) |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Leucine-Enriched Essential Amino Acid powderAlso: LEAALeucine-Enriched Essential Amino Acid Powder will be orally/enterally administered by mixing in water or ketogenic formula. | Drug | Leucine-Enriched Essential Amino Acid powder (LEAA) |
Primary outcome
Number of participants with treatment-related adverse events
Time frame: Baseline, 3 weeks, 12 weeks
Number of participants with treatment-related adverse events as reported by the trial participants.
3 secondary outcomes
Number of participants with improved nutritional status as assessed by weight.
Time frame: Baseline, 3 weeks, 12 weeks
Number of participants with improved nutritional status as assessed by body mass index (BMI).
Time frame: Baseline, 3 weeks, 12 weeks
Number of participants with improved nutritional status as assessed by serum ketone and glucose level.
Time frame: At each visit and on next day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elizabeth Thiele, MD, PhD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Leucine-Enriched Essential Amino Acid powder in people with Refractory Epilepsy and Drug Resistant Epilepsy, run by Elizabeth Anne Thiele at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events (time frame: Baseline, 3 weeks, 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.