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Serotonin Receptor Agonist

Fenfluramine Hydrochloride for Childhood Disintegrative Disorder

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 weeks
Treatment ~ Varies
Follow Up ~baseline, week 14
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 14 for reporting.

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 more

Side effects data

From 2022 Phase 2 trial • 7 Patients • NCT03861871
86%
Low appetite
86%
Lethargy
43%
Upper respiratory infection
43%
Common cold
29%
Diarrhea
29%
Crying episode
29%
Weight Loss
29%
Constipation
14%
Clostridium dificile infection
14%
Respiratory infection
14%
Aspiration pneumonia
14%
Screaming episode
14%
Teething
14%
Urinary tract infection
14%
Vomitting
14%
Pneumonia
14%
Low grade fever
14%
Bite on finger
14%
Intestinal gas
14%
Grinding teeth
14%
Rash on face
14%
Hair loss
14%
Regurgitation after meals
14%
Loose bowel
14%
Low tone
14%
Loose stool
14%
Mucus plug in throat
14%
Irritability
14%
SARS-CoV2 infection
14%
Sialorrhea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Fenfluramine Hydrochloride

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 2
~10

Find a Location

Who is running the clinical trial?

NYU Langone HealthLead Sponsor
1,400 Previous Clinical Trials
854,942 Total Patients Enrolled
Orrin Devinsky, MDPrincipal InvestigatorNYU Langone Health
5 Previous Clinical Trials
2,149 Total Patients Enrolled
~1 spots leftby Oct 2025