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Serotonin Receptor Agonist
Fenfluramine in CDKL5 Deficiency Disorder (CDD)
Phase 2
Waitlist Available
Led By Orrin Devinsky, MD
Research Sponsored by NYU Langone Health
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 14
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing fenfluramine to help control seizures in children and adolescents with CDKL5 Deficiency Disorder. The medication works by changing brain chemicals to reduce overactive signals that cause seizures. Fenfluramine, originally used as an appetite suppressant, has been repurposed and shown to reduce seizures in patients with Dravet syndrome and Lennox-Gastaut syndrome.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 14
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 14
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Change From Baseline in Median Monthly Convulsive Seizure Frequency
Secondary study objectives
Change in Caregiver Global Impression of Change (CGIC) Score
Change in Investigator Global Impression of Change (IGIC) Score
Change in Pediatric Quality Of Life (PEDS-QL) Epilepsy Module Raw Score
+1 moreSide effects data
From 2024 Phase 3 trial • 20 Patients • NCT0379013742%
Reduced Appetite
37%
Fatigue
21%
Viral Illness
11%
Diarrhea
11%
Shortness of Breath
11%
Seasonal Allergies
11%
Nocturnal Enuresis
5%
low serum calcium level
5%
Rhinorrhea
5%
Chest Pain
5%
irritability
5%
palilalia
5%
acne exacerbation
5%
rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment Group
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Fenfluramine HydrochlorideExperimental Treatment1 Intervention
Study medication will be administered as equal doses twice a day in the morning and in the evening approximately 12 hours apart.
Patients will first be titrated over 14 days to a dose of ZX008 0.8 mg/kg/day (maximum dose 30 mg/d).
After completion of the Titration Period, patients will continue to receive the ZX008 0.8 mg/kg/day dose and be treated for an additional 12 weeks (Maintenance Period). Study medication will continue to be administered twice a day in the morning and in the evening, approximately 12 hours apart.
After completion of the Maintenance Period, patients will enter the Taper Period, where they will decrease from 0.8 mg/kg twice a day to a dose of 0.4 mg/kg twice a day (maximum 30 mg/day). After 4 days at this dose level, patients will decrease their dose to 0.2 mg/kg/day. On day 9 of the Taper Period, all participants will stop taking study medication.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fenfluramine Hydrochloride
2019
Completed Phase 3
~30
Find a Location
Who is running the clinical trial?
NYU Langone HealthLead Sponsor
1,415 Previous Clinical Trials
856,196 Total Patients Enrolled
Orrin Devinsky, MDPrincipal InvestigatorNYU Langone Health
5 Previous Clinical Trials
2,449 Total Patients Enrolled