EpilepsyPhase 2KDNCT01906398
Efficacy and Safety of Ketogenic Diet as Adjunctive Treatment in Adults With Refractory Epilepsy
A Phase 2 study testing ketogenic diet in people with Epilepsy, run by Mid-Atlantic Epilepsy and Sleep Center, LLC at 1 site in United States.
Trial summary
The purpose of the study is to obtain pilot data on safety and efficacy of ketogenic diet (KD) as adjunctive treatment of adults with refractory epilepsy. This will be an open label study comparing seizure frequency during 4 months of prospective baseline observation period with seizure frequency during 4 months of… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Epilepsy
- What is being tested?
- ketogenic diet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2013to Dec 2018
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-65
- Stable epilepsy, either primary generalized or localization-related with partial complex seizures including partial complex seizures with or without secondary generalization, partial simple seizures with a clear motor component with or without secondary generalization, and partial simple seizures with secondary generalization; primary generalized tonic clonic seizures; and absence seizures of > 10 sec duration.
- Stable AED doses for at least 30 days
- Epilepsy duration for > 1 year
- Past/current treatment with > 3 AEDs. Vagal nerve stimulation treatment will be allowed and will not count as an AED. Vagal nerve stimulation setting must be stable for 3 months prior to enrollment
- Seizure frequency of > 0.5/month
Who cannot join
- Exclusively myoclonic seizures or absence seizures of ≤ 10 sec duration; simple partial seizures without motor components or secondary generalization
- Non-epileptic seizures
- Progressive neurological disease including neoplasm, central nerve system degenerative disorders including Alzheimer's disease, other forms of dementia
- Any systemic illness or unstable medical condition that might pose additional risk, including: renal or liver disease, past history of renal calculi, hyperuricemia, hypercalcemia, mitochondrial disease, known disorder of fatty acid metabolism, porphyria, other unstable metabolic or endocrine disturbances, and active systemic cancer
- Familial hyperlipidemia or uncontrolled hyperlipidemia
- Body Mass Index (BMI) < 18
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- Change in the dose of any Antiepileptic Drug within 30 days prior to enrollment
- Psychosis within six months of enrollment.
- Active drug or alcohol dependence or any other factors that, in the opinion of the site investigators would interfere with adherence to study requirements;
- Pregnancy
- Use of any CNS-active investigational drugs within 3 months of enrollment.
- Inability or unwillingness of subject or legal guardian/representative to give written informed consent.
Trial locations and contacts
1 site in 1 country.
United States1 site
MidAtlantic Epilepsy and Sleep Center
Bethesda, Maryland, 20817
About this study
Official title: Evaluation of the Efficacy and Safety of Ketogenic Diet as Adjunctive Treatment in Adults With Refractory Epilepsy: a Pilot Study.
The purpose of the study is to obtain pilot data on safety and efficacy of ketogenic diet (KD) as adjunctive treatment of adults with refractory epilepsy. This will be an open label study comparing seizure frequency during 4 months of prospective baseline observation period with seizure frequency during 4 months of add-on KD treatment. 18-65 year old men and women with refractory epilepsy, defined as seizures persisting in spite of past/present treatments with ≥ 3 AEDs, with seizure frequency of ≥ 0.5/month, will be evaluated. Subjects with both primary generalized and localization-related epilepsy (PGE, LRE) will be recruited. Subjects will have had epilepsy for at least 2 years prior to enrollment. Following initial screening, subjects will be observed for 4 months, with no change in AEDs except when deemed necessary by the patient's neurologist according to standard clinical care. Patients will then start ketogenic diet. Evaluations will include seizure frequency using a seizure diary, adverse events, treatment compliance using urine and plasma ketone levels. Quality of life will be evaluated with a standardized questionnaire of Quality Of Life In patients with Epilepsy, QOLIE-31-P. Level of alertness will be evaluated with Epworth Sleepiness Scale. These questionnaires will be administered at each visit.
Read the detailed description
The goal of the present open label study is to obtain pilot data to evaluate the efficacy and safety of KD in adults with intractable epilepsy. Investigators will evaluate the effect of KD on seizure frequency and on adverse events. Investigators will also evaluate serum levels of the ketone body, βhydroxybutyrate (BOH) and of glucose in order to determine whether changes in serum levels of these substances correlate with KD-associated changes in seizure frequency. The data from the present study will be used to design a large randomized study.
Laboratory evaluations will include complete blood count (CBC), serum electrolytes, including calcium, phosphate and magnesium, renal and liver functions, including total protein and albumin, uric acid, fasting serum lipid profile, glucose and b- hydroxybutyrate (BOH) levels, serum carnitine level, serum a.m. trough antiepilepsy drugs levels, and urine calcium and creatinine level. They will be obtained twice at baseline a month apart and monthly during KD treatment.
Primary outcome measures will include average monthly seizure frequency and adverse events. Secondary outcome measures will include treatment compliance, quality of life questionnaire (QOLIE-31-P) scores, and Epworth Sleepiness Scale scores.
- Conditions
- Epilepsy
- Being tested
- ketogenic diet
- Keywords
- Refractory epilepsy, ketogenic diet, efficacy
- Medical subject headings
- Epilepsy, Drug Resistant Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ketogenic dietOther
KD will consist of 3:1 [fat]: [protein + carbohydrate] weight ratio, with 1600 kcal restriction
| Study group | Role | What they receive |
|---|---|---|
| Experimental: ketogenic diet | Other | Treatment will consist of KD will consist of 3:1 [fat]: [protein + carbohydrate] weight ratio, with 1600 kcal restriction for patients with body mass index (BMI) of ≥ 21. The diet will be initiated with a 24 hour fast to induce ketosis. The diet will be supplemented with vitamins, calcium and phosphorus supplements to meet the requirements of US Dietary Reference Intakes (DRI) standard. If seizure frequency does not improve after 3 months of KD treatment, [fat]: [protein + carbohydrate] weight ratio will be increased to 4:1 Receives: Other: ketogenic diet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in epileptic seizure frequency.
Seizure frequency, adverse events and treatment compliance will be reviewed. The diet will be reviewed with the subject by the nutritionist before treatment initiation. Seizure frequency will be counted using a daily seizure diary. Subject's neurological and other clinical progress since the last visit will be reviewed. Concurrent medications will be documented. Vital signs and weight will be obtained, and a complete physical and neurological examination will be performed. Body mass index will be calculated. Subjects seizure/urine ketone diary will be reviewed at each visit.
Treatment compliance will be evaluated with urine ketone levels using the diaries and with serum b-hydroxy-butyrate (b-OH-butyrate, BOH) levels.
Measured: baseline, 8 months
Secondary outcomes (1)
Evaluate the number of participants with adverse events.
Adverse events and treatment compliance will be reviewed.Subject's neurological and other clinical progress since the last visit will be reviewed.
Measured: baseline, 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2013 (estimated)
- Study startJan 2013
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mid-Atlantic Epilepsy and Sleep Center, LLC
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pavel Klein, Mid-Atlantic Epilepsy and Sleep Center, LLC
- Investigators
- Pavel Klein, MD, Principal investigator, Mid-Atlantic Epilepsy and Sleep center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01906398
- Sponsor's protocol ID
- maes 001
- Acronym
- KD
- Status last verified
- Feb 2018
- First submitted
- 11 Jun 2013
- Last updated
- 23 Feb 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy and Safety of Ketogenic Diet as Adjunctive Treatment in Adults With Refractory Epilepsy: a Pilot Study.
- ClinicalTrials.gov ID
- NCT01906398
- Acronym / sponsor ID
- KD / maes 001
- Conditions
- Epilepsy
- Keywords
- Refractory epilepsy, ketogenic diet, efficacy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Mid-Atlantic Epilepsy and Sleep Center, LLCUniversity, hospital or other
- Study start
- Jan 2013
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 24 Jul 2013 (estimated)
- 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 purpose of the study is to obtain pilot data on safety and efficacy of ketogenic diet (KD) as adjunctive treatment of adults with refractory epilepsy. This will be an open label study comparing seizure frequency during 4 months of prospective baseline observation period with seizure frequency during 4 months of add-on KD treatment. 18-65 year old men and women with refractory epilepsy, defined as seizures persisting in spite of past/present treatments with ≥ 3 AEDs, with seizure frequency of ≥ 0.5/month, will be evaluated. Subjects with both primary generalized and localization-related epilepsy (PGE, LRE) will be recruited. Subjects will have had epilepsy for at least 2 years prior to enrollment. Following initial screening, subjects will be observed for 4 months, with no change in AEDs except when deemed necessary by the patient's neurologist according to standard clinical care. Patients will then start ketogenic diet. Evaluations will include seizure frequency using a seizure diary, adverse events, treatment compliance using urine and plasma ketone levels. Quality of life will be evaluated with a standardized questionnaire of Quality Of Life In patients with Epilepsy, QOLIE-31-P. Level of alertness will be evaluated with Epworth Sleepiness Scale. These questionnaires will be administered at each visit.
Detailed description
The goal of the present open label study is to obtain pilot data to evaluate the efficacy and safety of KD in adults with intractable epilepsy. Investigators will evaluate the effect of KD on seizure frequency and on adverse events. Investigators will also evaluate serum levels of the ketone body, βhydroxybutyrate (BOH) and of glucose in order to determine whether changes in serum levels of these substances correlate with KD-associated changes in seizure frequency. The data from the present study will be used to design a large randomized study.
Laboratory evaluations will include complete blood count (CBC), serum electrolytes, including calcium, phosphate and magnesium, renal and liver functions, including total protein and albumin, uric acid, fasting serum lipid profile, glucose and b- hydroxybutyrate (BOH) levels, serum carnitine level, serum a.m. trough antiepilepsy drugs levels, and urine calcium and creatinine level. They will be obtained twice at baseline a month apart and monthly during KD treatment.
Primary outcome measures will include average monthly seizure frequency and adverse events. Secondary outcome measures will include treatment compliance, quality of life questionnaire (QOLIE-31-P) scores, and Epworth Sleepiness Scale scores.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: ketogenic diet | Other | Other: ketogenic dietTreatment will consist of KD will consist of 3:1 [fat]: [protein + carbohydrate] weight ratio, with 1600 kcal restriction for patients with body mass index (BMI) of ≥ 21. The diet will be initiated with a 24 hour fast to induce ketosis. The diet will be supplemented with vitamins, calcium and phosphorus supplements to meet the requirements of US Dietary Reference Intakes (DRI) standard. If seizure frequency does not improve after 3 months of KD treatment, [fat]: [protein + carbohydrate] weight ratio will be increased to 4:1 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age 18-65
- Stable epilepsy, either primary generalized or localization-related with partial complex seizures including partial complex seizures with or without secondary generalization, partial simple seizures with a clear motor component with or without secondary generalization, and partial simple seizures with secondary generalization; primary generalized tonic clonic seizures; and absence seizures of > 10 sec duration.
- Stable AED doses for at least 30 days
- Epilepsy duration for > 1 year
- Past/current treatment with > 3 AEDs. Vagal nerve stimulation treatment will be allowed and will not count as an AED. Vagal nerve stimulation setting must be stable for 3 months prior to enrollment
Key exclusion criteria (12 listed)
- Exclusively myoclonic seizures or absence seizures of ≤ 10 sec duration; simple partial seizures without motor components or secondary generalization
- Non-epileptic seizures
- Progressive neurological disease including neoplasm, central nerve system degenerative disorders including Alzheimer's disease, other forms of dementia
- Any systemic illness or unstable medical condition that might pose additional risk, including: renal or liver disease, past history of renal calculi, hyperuricemia, hypercalcemia, mitochondrial disease, known disorder of fatty acid metabolism, porphyria, other unstable metabolic or endocrine disturbances, and active systemic cancer
- Familial hyperlipidemia or uncontrolled hyperlipidemia
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 |
|---|---|---|
| ketogenic dietKD will consist of 3:1 [fat]: [protein + carbohydrate] weight ratio, with 1600 kcal restriction | Other | Experimental: ketogenic diet |
Primary outcome
Change from baseline in epileptic seizure frequency.
Time frame: baseline, 8 months
Seizure frequency, adverse events and treatment compliance will be reviewed. The diet will be reviewed with the subject by the nutritionist before treatment initiation. Seizure frequency will be counted using a daily seizure diary. Subject's neurological and other clinical progress since the last visit will be reviewed. Concurrent medications will be documented. Vital signs and weight will be obtained, and a complete physical and neurological examination will be performed. Body mass index will be calculated. Subjects seizure/urine ketone diary will be reviewed at each visit. Treatment compliance will be evaluated with urine ketone levels using the diaries and with serum b-hydroxy-butyrate (b-OH-butyrate, BOH) levels.
1 secondary outcome
Evaluate the number of participants with adverse events.
Time frame: baseline, 8 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2018.
- Individual participant dataSharing planned: Undecided
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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.
Pavel Klein, MD principal investigator
Mid-Atlantic Epilepsy and Sleep center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ketogenic diet in people with Epilepsy, run by Mid-Atlantic Epilepsy and Sleep Center, LLC 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 6 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 23 Feb 2018. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Change from baseline in epileptic seizure frequency. (time frame: baseline, 8 months). 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 23 Feb 2018.