Dravet SyndromePhase 3NCT05560282
Fenfluramine for Adult Dravet Patients
A Phase 3 study testing Fenfluramine in people with Dravet Syndrome and Dravet Syndrome, Intractable, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients Short Title: Fenfluramine for Adult Dravet patients Clinical Phase: Phase III Sample Size: A total of 15 participants will be included in the study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dravet Syndrome, Dravet Syndrome, Intractable
- What is being tested?
- Fenfluramine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteCanada
- Study dates
- 11 Jan 2023to 13 May 2025
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Male and female patients 18 years of age and older. 2. Diagnosis of DS secondary to a genetically confirmed pathogenic variant. 3. Patients recruited from the Adult Epilepsy Genetics Clinic at Toronto Western Hospital. We will also accept referrals from other academic epilepsy centers across Ontario and that are referred to our program, making this a single site study.
- 4. At least 4 convulsive seizures per month at the start of the study, despite having been treated with at least two different antiepileptic drugs (given concurrently or sequentially) for at least one year.
- 5. At least 4 convulsive seizures per month during the prospective Baseline Phase (4 weeks).
- 6. Able to provide written consent signed by study participant's caregiver prior to entering the study or undergoing any study procedures.
- 7. Participant's caregivers should be able to record seizures and report adverse events for the full duration of the study.
- 8. No evidence of non-compliance with ongoing ASM therapy. 9. Stable dose(s) of the same ASMs for one month prior to screening. 10. If participant has a vagal nerve stimulator or deep brain stimulator, it must have been implanted for at least six months prior to recruitment. Stimulator parameters may not be changed for at least one month prior to screening or thereafter during the study. Magnet use will be allowed but must be documented throughout the study.
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- 11. Patient's caregivers agree that patient will not to take any cannabinoids or any other investigational compound for 1 month before the study or outside cannabinoids during the study.
- 12. Patient's caregivers agree that patient will have blood samples taken and stored for assays.
- 13. Is planning to stay in the country for the duration of the trial. 14. Can travel to the study site for the initial in-person visit with the study physicians and to a local lab for blood collection (Lifelabs).
- 15. Patient's caregiver has access to telephone, computer, and internet to complete the study questionnaires.
- 16. Sexually active women of child-bearing potential (WCBP) and males must be using a medically acceptable method of birth control. Women who are of non child-bearing potential, i.e., post-menopause, must have this condition captured in their medical history. A medically acceptable method of birth control includes barrier methods of contraception (e.g. male condom, female condom, cervical cap, diaphragm, contraceptive sponge), intrauterine devices in place for at least 3 months, surgical sterilization, or adequate barrier methods (e.g., diaphragm and foam). Use of oral contraceptives in combination with another method (e.g., a spermicidal cream) is acceptable. In participants who are not sexually active, abstinence is an acceptable form of birth control.
Who cannot join
- 1. Participation in a study involving administration of an investigational compound within one month of First appointment.
- 2. Evidence of clinically significant non-epileptic disease (cardiac, respiratory, gastrointestinal, hepatic, hematologic or renal disease, etc.) that in the opinion of the investigators could affect the patient's safety or trial conduct.
- 3. Pulmonary hypertension (systolic pressure>35 mmHg) 4. Cardiovascular or cerebrovascular disease 5. Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy defined as greater than moderate and/or mild aortic regurgitation on echocardiogram 6. Treatment with centrally acting anorectic agents, monoamine oxidase inhibitors or other centrally acting agent with serotonin antagonist or agonist properties.
- 7. Current or past history of glaucoma 8. Hypersensitivity to FEN or any of the excipients in the study medication 9. Use of CBD oil 10. Pregnant and/or breastfeeding women are excluded from this study. Sexually active women must have negative pregnancy urine test in first in-person visit.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients
Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients
Short Title: Fenfluramine for Adult Dravet patients
Read more
Clinical Phase: Phase III
Sample Size: A total of 15 participants will be included in the study.
Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study.
Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol.
Study Duration:
- Treatment period: 12 months Study duration: 28 months
Study Agent/ Intervention/ Procedure:
Name of study drug: fenfluramine (FINTEPLA)
Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day.
Duration:
Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol)
Route of administration:
Oral
Efficacy and safety points of interest
- Monthly convulsive seizure frequency (MCSF) reduction ≥ 50%
- Improvement in motor function
- Improvement in Cognition and Behavior
- Improvement in Quality of Sleep
- Improvement in Quality of life
- Determination of Cardiovascular safety in adults
- Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF)
- Longest period of seizure freedom
- Number of Emergency room visits
- Use of rescue medication (number of days in 28 day-periods)
- Duration of post-ictal stage
- Frequency of other seizure types
- Body weight changes
- Patient's global functioning prior to and after study (Clinical Global Impressions Scale)
Trial registration:
www.clinicaltrials.gov
- Conditions
- Dravet Syndrome, Dravet Syndrome, Intractable
- Being tested
- Fenfluramine
- Medical subject headings
- Epilepsies, Myoclonic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FenfluramineDrug
Also called ZX008, Fintepla
FINTEPLA oral solution contains 2.2 mg/mL fenfluramine, equivalent to 2.5 mg/mL of the hydrochloride salt. The active ingredient, fenfluramine hydrochloride, is designated chemically as N-ethyl-α- methyl-3-(trifluoromethyl) phenethylamine hydrochloride.
Fenfluramine hydrochloride is a white to off-white crystalline solid. The pKa of fenfluramine is 10.2. FINTEPLA is a clear, colorless solution, pH 5.
| Study group | Role | What they receive |
|---|---|---|
| Fenfluramine | Experimental | Fenfluramine, oral,
1. starting at 0.1mg/kg twice daily, maximum 26mg/day in patients not taking concomitant stiripentol
2. starting at 0.1mg/kg twice daily, maximum 17mg/day in patients taking concomitant stiripentol Receives: Drug: Fenfluramine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Seizure reduction
Comparing the proportion of participants with >50% reduction in the monthly countable convulsive seizure frequency between 4- week Baseline period (Observational Phase) and after 12-week treatment phase using seizure diary.
Comparing the proportion of participants with >50% reduction in the monthly countable convulsive seizure frequency between 4- week Baseline period (Observational Phase) and after 12-week treatment phase using seizure diary.
Measured: 16 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2022
- Study start11 Jan 2023
- Primary completion13 May 2025
- Study completion13 May 2025
Why it stopped: Study recruitment was too slow, not enough funds to sustain it.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Zogenix, Inc.
- Responsible party
- Principal investigator, Danielle Andrade, University Health Network, Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05560282
- Sponsor's protocol ID
- 21-5681
- Status last verified
- Apr 2026
- First submitted
- 26 Sep 2022
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients
- ClinicalTrials.gov ID
- NCT05560282
- Acronym / sponsor ID
- 21-5681
- Conditions
- Dravet Syndrome, Dravet Syndrome, Intractable
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study recruitment was too slow, not enough funds to sustain it.
- Enrolment
- 7 participants (actual)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Collaborators
- Zogenix, Inc.
- Study start
- 11 Jan 2023 (actual)
- Primary completion
- 13 May 2025 (actual)
- Study completion
- 13 May 2025 (actual)
- First posted
- 29 Sep 2022 (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.
Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients
Short Title: Fenfluramine for Adult Dravet patients
Clinical Phase: Phase III
Sample Size: A total of 15 participants will be included in the study.
Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study.
Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol.
Study Duration:
- Treatment period: 12 months Study duration: 28 months
Study Agent/ Intervention/ Procedure:
Name of study drug: fenfluramine (FINTEPLA)
Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day.
Duration:
Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol)
Route of administration:
Oral
Efficacy and safety points of interest
- Monthly convulsive seizure frequency (MCSF) reduction ≥ 50%
- Improvement in motor function
- Improvement in Cognition and Behavior
- Improvement in Quality of Sleep
- Improvement in Quality of life
- Determination of Cardiovascular safety in adults
- Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF)
- Longest period of seizure freedom
- Number of Emergency room visits
- Use of rescue medication (number of days in 28 day-periods)
- Duration of post-ictal stage
- Frequency of other seizure types
- Body weight changes
- Patient's global functioning prior to and after study (Clinical Global Impressions Scale)
Trial registration:
www.clinicaltrials.gov
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fenfluramine | Experimental | Drug: FenfluramineFenfluramine, oral, 1. starting at 0.1mg/kg twice daily, maximum 26mg/day in patients not taking concomitant stiripentol 2. starting at 0.1mg/kg twice daily, maximum 17mg/day in patients taking concomitant stiripentol |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 1. Male and female patients 18 years of age and older. 2. Diagnosis of DS secondary to a genetically confirmed pathogenic variant. 3. Patients recruited from the Adult Epilepsy Genetics Clinic at Toronto Western Hospital. We will also accept referrals from other academic epilepsy centers across Ontario and that are referred to our program, making this a single site study.
- 4. At least 4 convulsive seizures per month at the start of the study, despite having been treated with at least two different antiepileptic drugs (given concurrently or sequentially) for at least one year.
- 5. At least 4 convulsive seizures per month during the prospective Baseline Phase (4 weeks).
- 6. Able to provide written consent signed by study participant's caregiver prior to entering the study or undergoing any study procedures.
- 7. Participant's caregivers should be able to record seizures and report adverse events for the full duration of the study.
Key exclusion criteria (4 listed)
- 1. Participation in a study involving administration of an investigational compound within one month of First appointment.
- 2. Evidence of clinically significant non-epileptic disease (cardiac, respiratory, gastrointestinal, hepatic, hematologic or renal disease, etc.) that in the opinion of the investigators could affect the patient's safety or trial conduct.
- 3. Pulmonary hypertension (systolic pressure>35 mmHg) 4. Cardiovascular or cerebrovascular disease 5. Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy defined as greater than moderate and/or mild aortic regurgitation on echocardiogram 6. Treatment with centrally acting anorectic agents, monoamine oxidase inhibitors or other centrally acting agent with serotonin antagonist or agonist properties.
- 7. Current or past history of glaucoma 8. Hypersensitivity to FEN or any of the excipients in the study medication 9. Use of CBD oil 10. Pregnant and/or breastfeeding women are excluded from this study. Sexually active women must have negative pregnancy urine test in first in-person visit.
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 |
|---|---|---|
| FenfluramineAlso: ZX008, FinteplaFINTEPLA oral solution contains 2.2 mg/mL fenfluramine, equivalent to 2.5 mg/mL of the hydrochloride salt. The active ingredient, fenfluramine hydrochloride, is designated chemically as N-ethyl-α- methyl-3-(trifluoromethyl) phenethylamine hydrochloride. Fenfluramine hydrochloride is a white to off-white crystalline solid. The pKa of fenfluramine is 10.2. FINTEPLA is a clear, colorless solution, pH 5. | Drug | Fenfluramine |
Primary outcome
Seizure reduction
Time frame: 16 week
Comparing the proportion of participants with >50% reduction in the monthly countable convulsive seizure frequency between 4- week Baseline period (Observational Phase) and after 12-week treatment phase using seizure diary. Comparing the proportion of participants with >50% reduction in the monthly countable convulsive seizure frequency between 4- week Baseline period (Observational Phase) and after 12-week treatment phase using seizure diary.
Results and safety
The study was stopped early (reason given: Study recruitment was too slow, not enough funds to sustain it.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fenfluramine in people with Dravet Syndrome and Dravet Syndrome, Intractable, run by University Health Network, Toronto at 1 site in Canada.
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 11 inclusion and 4 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: stopped early, as of the sponsor's last update on 5 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 Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was stopped early (reason given: Study recruitment was too slow, not enough funds to sustain it.). 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?
Seizure reduction (time frame: 16 week). 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 5 May 2026.