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Fenfluramine for CDKL5 Deficiency Disorder

Phase 3
Recruiting
Research Sponsored by Zogenix, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subject has a confirmed pathogenic or likely pathogenic mutation in the CDKL5 gene and a clinical diagnosis of CDD with epilepsy onset in the first year of life, plus motor and developmental delays.
Subject is male or female, aged 1 to 35 years, inclusive, as of the day of the Screening Visit.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 14 weeks
Awards & highlights

Study Summary

This trial will test if ZX008 is an effective and safe adjunctive therapy for people with uncontrolled seizures due to CDKL5 deficiency disorder.

Who is the study for?
This trial is for children and adults aged 1 to 35 with CDKL5 Deficiency Disorder (CDD) who have seizures starting in the first year of life, along with motor and developmental delays. They must have tried at least two seizure treatments without success and be on a stable epilepsy treatment plan.Check my eligibility
What is being tested?
The study tests ZX008 (Fenfluramine Hydrochloride) as an additional therapy against a placebo for reducing uncontrolled seizures in CDD patients. It's double-blind, meaning neither participants nor researchers know who gets the real drug or placebo.See study design
What are the potential side effects?
Potential side effects are not listed here, but since ZX008 contains fenfluramine, possible risks may include heart-related issues like valve problems, pulmonary hypertension, decreased appetite or mood changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a CDKL5 mutation and was diagnosed with CDD, having seizures in my first year along with motor and developmental delays.
Select...
I am between 1 and 35 years old.
Select...
I still have seizures despite trying 2 or more epilepsy treatments.
Select...
I am currently on a treatment for seizures.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
The median percentage change from the Baseline Period (Baseline) in "monthly (28 days) countable motor seizure frequency
Secondary outcome measures
The median percentage change from Baseline in monthly Generalized Tonic-Clonic (GTC) seizure frequency
The percentage of subjects who achieve a ≥ 50% reduction from Baseline in CMSF
The percentage of subjects who achieve improvement in the Clinical Global Impression-Improvement (CGI-I) rating as assessed by the Investigator

Side effects data

From 2020 Phase 3 trial • 262 Patients • NCT02682927
38%
Decreased appetite
21%
Somnolence
19%
Pyrexia
17%
Blood glucose decreased
17%
Echocardiogram abnormal
15%
Diarrhoea
13%
Tremor
10%
Fatigue
8%
Upper respiratory tract infection
8%
Platelet count decreased
8%
Weight decreased
8%
Rash
6%
Cough
6%
Vomiting
6%
Lethargy
6%
Asthenia
6%
Hypoglycaemia
4%
Drooling
4%
Viral infection
4%
Rhinitis
4%
Blood pressure increased
4%
Ataxia
4%
Balance disorder
4%
Abnormal behaviour
2%
Irritability
2%
Urinary tract infection
2%
Varicella
2%
Toxicity to various agents
2%
Hepatic enzyme increased
2%
Nasopharyngitis
2%
Blood pressure diastolic increased
2%
Constipation
2%
Gait disturbance
2%
Influenza
2%
Sinusitis
2%
Fall
2%
Hypotonia
2%
Seizure
2%
Urinary incontinence
2%
Rhinorrhoea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Study 3: ZX008 0.8 mg/kg/Day
Study 1: Placebo
Study 1: ZX008 0.2 mg/kg/Day
Study 1: ZX008 0.8 mg/kg/Day
Study 3: Placebo
Study 3: ZX008 0.2 mg/kg/Day

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: ZX008 0.8 mg/kg/dayExperimental Treatment1 Intervention
Part 1: ZX008 0.8 mg/kg/day will be administered twice a day (BID) in equally divided doses; maximum of 30 mg/day, (subjects taking concomitant stiripentol will receive 0.5 mg/kg/day, [maximum of 20 mg/day]) with or without food.
Group II: ZX008Experimental Treatment1 Intervention
Part 2: Open-label ZX008 will be administered using a flexible dosing regimen, up to ZX008 0.8 mg/kg/day; maximum dose: 30 mg/day (subjects taking concomitant stiripentol will receive 0.5 mg/kg/day, [maximum of 20 mg/day]). ZX008 will be administered twice a day (BID) in equally divided doses with or without food.
Group III: PlaceboPlacebo Group1 Intervention
Part 1: Matching ZX008 placebo will be administered twice a day (BID) in equally divided doses with or without food.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ZX008 (Fenfluramine Hydrochloride)
2016
Completed Phase 3
~730

Find a Location

Who is running the clinical trial?

Zogenix, Inc.Lead Sponsor
24 Previous Clinical Trials
2,473 Total Patients Enrolled
UCB CaresStudy Director001 844 599 2273
205 Previous Clinical Trials
44,863 Total Patients Enrolled

Media Library

ZX008 (Fenfluramine Hydrochloride) Clinical Trial Eligibility Overview. Trial Name: NCT05064878 — Phase 3
CDKL5 Deficiency Disorder Research Study Groups: Placebo, ZX008, ZX008 0.8 mg/kg/day
CDKL5 Deficiency Disorder Clinical Trial 2023: ZX008 (Fenfluramine Hydrochloride) Highlights & Side Effects. Trial Name: NCT05064878 — Phase 3
ZX008 (Fenfluramine Hydrochloride) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05064878 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the ZX008 (Fenfluramine Hydrochloride) approval status from the FDA?

"ZX008 (Fenfluramine Hydrochloride) received a safety score of 3 from our team at Power. This is because it is a Phase 3 trial, which suggests that there is some data affirming its efficacy and multiple rounds of data confirming its safety."

Answered by AI

Have these methods been used before in other medical trials?

"Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc., sponsored the earliest study for ZX008 (Fenfluramine Hydrochloride) in 2016. This Phase 3 drug approval stage trial involved 373 participants and completed its 5th year in 2020. There are presently 6 active studies across 56 cities and 15 countries."

Answered by AI

Does this research include patients who are octogenarians?

"This particular clinical trial is meant for patients that are between 1 year and 35 years old. Out of the 45 trials for patients under 18, this one has the most restrictive age requirement. 61 other trials exist for patients over 65."

Answered by AI

Are there any patients who have not yet been enrolled in this trial?

"The clinical trial is currently looking for patients, according to the information available on clinicaltrials.gov. This particular study was posted on June 1st, 2020 and updated last October 27th, 2020."

Answered by AI

What other scientific literature is there on ZX008 (Fenfluramine Hydrochloride)?

"At present, there are 6 on-going studies investigating ZX008 (Fenfluramine Hydrochloride), with 5 of them in Phase 3. Although a few of the experiments regarding ZX008 (Fenfluramine Hydrochloride) are finishing up in Boston, Massachusetts, there are still 236 other places conducting trials for this medication."

Answered by AI

What are the ideal characteristics for participants in this trial?

"Eligible patients for this study must be aged 1-35, have seizures, and meet the CDKL5 gene mutation criteria. Additionally, the patient must be male or female, currently receiving antiseizure medication (ASM), vagus nerve stimulation (VNS), responsive neurostimulation (RNS), or ketogenic diet (KD). Lastly, it is important that the subject has failed to achieve seizure control with 2 or more AETs."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder
2022. "A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05064878.
~26 spots leftby Feb 2025
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