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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 14

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%

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

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