Phase 3
N=412
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
Dravet Syndrome · Lennox Gastaut Syndrome · Epileptic Encephalopathy
Bottom Line
View on ClinicalTrials.gov: NCT03936777 ↗Enrolled (actual)
412
Serious AEs
16.3%
Results posted
Nov 2025
Primary outcome: Primary: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) — 75.5 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ZX008 (Fenfluramine Hydrochloride) (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Zogenix, Inc.
- Primary completion
- May 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) |
75.5 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver |
39; 88; 87; 124; 23; 7 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator |
23; 86; 85; 141; 26; 4 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver |
26; 75; 87; 161; 15; 4 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver |
20; 51; 75; 174; 35; 12 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver |
17; 45; 86; 190; 26; 4 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator |
13; 54; 101; 184; 9; 2 | — |
| SECONDARY Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator |
10; 42; 80; 200; 27; 5 | — |
| SECONDARY Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator |
10; 33; 81; 221; 17; 1 | — |
| SECONDARY Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator |
242; 67; 42; 35; 12; 262 | — |
Summary
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
Eligibility Criteria
Inclusion Criteria
- Male or nonpregnant, nonlactating female
- Satisfactory completion of a core study
- Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008
- Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability
Exclusion Criteria
- Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
- Moderate or severe hepatic impairment
- Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and serotonin reuptake inhibitors within 14 days of receiving ZX008
Data sourced from ClinicalTrials.gov (NCT03936777). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.