Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT03355209) enrolled 296 participants across two groups — Cohort A (the larger group, with around 87 people per arm) and Cohort B (a smaller group, with around 11 people per arm). The trial was testing a medicine called ZX008 (fenfluramine) at two different daily doses — 0.2 mg/kg/day and 0.8 mg/kg/day — compared to a placebo (a dummy treatment with no active ingredient), in people with Lennox-Gastaut syndrome, a type of epilepsy that causes "drop seizures" (seizures that make a person suddenly fall or drop). The trial had two parts: a blinded period where neither participants nor doctors knew who received which treatment, followed by an open-label extension where everyone knew the treatment being given. The reported data shows that during the blinded period (Part 1), the average percentage change in drop seizure frequency compared to before the trial started was: in Cohort A, the placebo group had a 7.59% reduction, the 0.2 mg/kg/day group had a 14.16% reduction, and the 0.8 mg/kg/day group had a 26.49% reduction. In Cohort B, the placebo group showed a 17.89% reduction, the 0.2 mg/kg/day group showed a 14.12% reduction, and the 0.8 mg/kg/day group showed a 34.52% reduction. As a secondary measure, the proportion of participants whose drop seizures fell by at least half ranged from about 10% in placebo groups up to about 36% in some ZX008 groups. On a doctor-rated improvement scale, the proportion rated as at least "minimally improved" ranged from roughly 10–34% in placebo groups to as high as 73% in one ZX008 group. During the open-label extension period (Part 2), the reported data shows that 83.0% of Cohort A participants and 96.9% of Cohort B participants experienced at least one adverse event (an unwanted or unexpected medical occurrence recorded during the study). Serious adverse events — those considered medically significant, requiring hospitalisation, or life-threatening — were reported in 16.6% of Cohort A and 18.8% of Cohort B participants during this period. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 35499850) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Epilepsy Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are male, or female and not pregnant or breastfeeding, and are between 2 and 35 years old (inclusive) at the time of the screening visit.
- You have been clinically diagnosed with Lennox-Gastaut syndrome, and seizures that cause you to fall or drop are not fully controlled by your current seizure medications.
- Your seizures started when you were 11 years old or younger.
- You have been identified as having abnormal cognitive development (difficulties with thinking, learning, or development).
- You are currently taking at least 1 but no more than 4 anti-seizure medications at the same time.
Who may not be able to join:
- Your seizures are caused by a progressive or degenerating neurological disease.
- You have a history of a specific type of seizure called hemiclonic seizures during the first year of your life (confirm with trial site).
- Your drop seizures only happen in clusters, making it impossible to count individual seizures reliably.
- You have a condition called pulmonary arterial hypertension (high blood pressure in the lungs).
- You have a current or past history of heart or stroke-related conditions, such as heart valve problems, a heart attack, or a stroke.
- You are currently taking certain medications, including specific appetite suppressants, MAO inhibitors, certain serotonin-affecting drugs, atomoxetine or similar medications, or cyproheptadine (confirm with trial site if unsure about your medications).
- You have been taking a medication called felbamate for less than 1 year before screening, or your liver function and blood test results are not stable, or your felbamate dose has not been stable for at least 60 days before the screening visit.
- You are currently taking part in another clinical trial involving an investigational (unapproved) product.
- You live in a general nursing home that does not specialize in epilepsy care.
- You have had another significant illness or health condition (other than epilepsy) in the 4 weeks before the screening visit that could affect your ability to take part in the study or could put you at risk.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: UCB Cares, 001 844 599 2273
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Part 1: Percent Change From Baseline in the Frequency of Seizures That Result in Drops (ESC-confirmed) in the Combined Titration and Maintenance Period (T+M) in the ZX008 0.8 mg/kg/Day Group Compared to the Placebo Group; Part 2: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs); Part 2: Percentage of Participants With Serious TEAEs
Can't join this trial?
Data last synced from ClinicalTrials.gov: 21 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.