EpilepsyPhase 2FFA-LGSNCT02655198
Add-on Therapy With Low Dose Fenfluramine in Lennox Gastaut Epilepsy
A Phase 2 study testing Fenfluramine in people with Epilepsy and Lennox Gastaut Syndrome, run by KU Leuven at 1 site in Belgium.
Trial summary
In this trial, the potential anti-epileptic effect of low dose fenfluramine in Lennox Gastaut epilepsy will be studied. An exploratory dose finding add-on trial is proposed. At baseline and at the end of the study, ECG and ultrasound of the heart will be performed as part of the safety follow up. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Epilepsy, Lennox Gastaut Syndrome
- What is being tested?
- Fenfluramine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteBelgium
- Study dates
- Jan 2016to Dec 2024
- Phase
- Phase 2
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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Electro-clinical epilepsy syndrome compatible with Lennox Gastaut syndrome:
- Minimum requirements (based on ILAE epilepsydiagnosis.org):
- Multiple seizure types including in any case tonic seizures
- EEG shows slow spike waves and abnormal background
- Abnormal cognitive development
- MRI compatible with Lennox Gastaut epilepsy : no progressive disease
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- Drug resistant:
- at least 4 documented seizures in the last 4 weeks before inclusion (minimum 4 seizures in at least 2 separate weeks) Seizure types eligible for inclusion are : generalized tonic-clonic seizures GTC , tonic seizures TS , atonic seizures AS or clearly recognizable focal seizures FS.
- on >= 2 AEDs (including VNS) during the 4 weeks before inclusion (no changes in treatment before inclusion and during the trial)
- Age between 3 and 18 years
- Subject is male or non-pregnant, non-lactating female. Female subjects of childbearing potential must not be pregnant or breast-feeding. Female subjects of childbearing potential must have a negative urine pregnancy test. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while being treated on this study and for 90 days after the last dose of study drug.
Who cannot join
- Known clinical cardiovascular abnormalities (including valvular problems, shunts, pulmonary hypertension, exercise intolerance)
- Any cardiac ultrasound/ECG abnormalities at baseline
- Weight below percentile 3 for age at baseline
- Subject is receiving concomitant therapy with: centrally-acting anorectic agents; monoamine oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
- Subject is unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with the Baseline Period and throughout the study.
Trial locations and contacts
1 site in 1 country.
About this study
In this trial, the potential anti-epileptic effect of low dose fenfluramine in Lennox Gastaut epilepsy will be studied. An exploratory dose finding add-on trial is proposed. At baseline and at the end of the study, ECG and ultrasound of the heart will be performed as part of the safety follow up.
- Conditions
- Epilepsy, Lennox Gastaut Syndrome
- Being tested
- Fenfluramine
- Keywords
- refractory childhood epilepsy, Lennox Gastaut syndrome, fenfluramine, anti-epileptic treatment
- Medical subject headings
- Epilepsy, Lennox Gastaut Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FenfluramineDrug
study of efficacy and safety of add-on fenfluramine at different dosages in refractory Lennox Gastaut patients : 0.2 - 0.4 and 0.8 mg/kg/day (max 30 mg).
| Study group | Role | What they receive |
|---|---|---|
| fenfluramine | Experimental | Experimental : one armed open label study :
Add-on fenfluramine in refractory Lennox Gastaut patients. Starting dose 0.2mg/kg/day. In non-responders (<50% seizure frequency decrease), dose will be uptitrated every 4 weeks from 0,2 to 0,4 and max 0,8 mg/kg/day (max 30 mg). Total duration study and max exposure to the drug 20 weeks Receives: Drug: Fenfluramine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of add-on FFA in Lennox Gastaut epilepsy: Number of responders and seizure free patients at each FFA dosage (0,2 or 0,4 or 0,8 mg/kg/day)
Measured: up to 20 weeks
Secondary outcomes (4)
Seizure frequency change per patient and per major seizure type (Tonic Clonic Seizures (TCS), Tonic Seizures (TS), Atonic Seizures (AS), Focal Seizures (FS))
Measured: 20 weeks
Adverse events (cardiac and general)
Measured: 20 weeks
Sleep quality : 10 point scale instrument to score sleep quality
Measured: 20 weeks
CGI (clinical global impression) scale at last visit , by patient/caregiver and treating physician
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2016 (estimated)
- Study startJan 2016
- Primary completionSep 2018
- Study completionDec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- KU Leuven
- Sponsor type
- University, hospital or other
- Collaborators
- Zogenix, Inc.
- Responsible party
- Principal investigator, Lieven Lagae, KU Leuven
- Investigators
- Lieven G Lagae, MD, PhD, Principal investigator, Katolieke Universiteit Leuven, University Hospitals Gasthuisberg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02655198
- Sponsor's protocol ID
- S58545
- Acronym
- FFA-LGS
- Status last verified
- Oct 2026
- First submitted
- 12 Jan 2016
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Lagae L, Schoonjans AS, Gammaitoni AR, Galer BS, Ceulemans B. A pilot, open-label study of the effectiveness and tolerability of low-dose ZX008 (fenfluramine HCl) in Lennox-Gastaut syndrome. Epilepsia. 2018 Oct;59(10):1881-1888. doi: 10.1111/epi.14540. Epub 2018 Aug 26.
PubMed 30146701