A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder. (GEMZ)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension

This is a Phase 3 Study to examine the efficacy and safety of ZX008 in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Study Overview

• Status: Active, not recruiting
• Conditions: Generalized Tonic Clonic Seizure; CDKL5 Deficiency Disorder; Epileptic Spasm; Refractory Seizures
• Intervention / Treatment: Drug: Fenfluramine; Drug: Placebo

Detailed Description

This is an up to 3-part multicenter study: a double-blind, placebo-controlled part (Part 1) which includes Baseline Period (4 weeks), Titration Period (2 weeks), Maintenance Period (12 weeks) and Transition Period (2 weeks), followed by up to 2 open-label extension (OLE) parts: Part 2 of 54 weeks [Treatment Period (52 weeks), Taper Period (2 weeks)] and Part 3, i.e., OLE1 and OLE2, respectively, with the addition of the second OLE part (Part 3/OLE2) only for participants who will continue on fenfluramine and have no alternative treatment access from another source (e.g., a managed access program [MAP]). Participants can remain in Part 3 until MAP access or approval of fenfluramine (ZX008) has been obtained from regulatory authorities for CDD in the participant's country of residence, or until the investigational product development for CDD is stopped by the Sponsor, whichever comes first. Participants who discontinue early from Part 1, Part 2, or Part 3 will attend a Cardiac Follow-Up Visit 6 months after their last dose of study drug.

• Study Type: Interventional
• Enrollment (Actual): 87
• Phase: Phase 3

Contacts and Locations

Study Locations

• Austria
  • Linz, Austria
    • Ep0216 2505
• Belgium
  • Brussels, Belgium
    • Ep0216 804
  • Edegem, Belgium
    • Ep0216 801
• Georgia
  • Tbilisi, Georgia
    • Ep0216 2802
• Germany
  • Bielefeld, Germany
    • Ep0216 902
  • Kehl-kork, Germany
    • Ep0216 909
  • Kiel, Germany
    • Ep0216 908
  • Vogtareuth, Germany
    • Ep0216 901
• Ireland
  • Dublin, Ireland
    • Ep0216 1803
• Israel
  • Petah Tikva, Israel
    • Ep0216 1909
  • Ramat Gan, Israel
    • Ep0216 1906
  • Tel Aviv, Israel
    • Ep0216 1904
• Italy
  • Florence, Italy
    • Ep0216 1201
  • Genova, Italy
    • Ep0216 1204
  • Modena, Italy
    • Ep0216 1212
  • Roma, Italy
    • Ep0216 1206
  • Roma, Italy
    • Ep0216 1208
  • Verona, Italy
    • Ep0216 1202
• Japan
  • Hiroshima, Japan
    • Ep0216 1512
  • Niigata, Japan
    • Ep0216 1505
  • Omura-shi, Japan
    • Ep0216 1518
  • Shizuoka, Japan
    • Ep0216 1502
• Netherlands
  • Zwolle, Netherlands
    • Ep0216 1401
• Portugal
  • Lisbon, Portugal
    • Ep0216 2104
  • Porto, Portugal
    • Ep0216 2105
• Spain
  • Barcelona, Spain
    • Ep0216 1103
  • Madrid, Spain
    • Ep0216 1117
  • Santiago de Compostela, Spain
    • Ep0216 1118
• United Arab Emirates
  • Dubai, United Arab Emirates
    • Ep0216 3101
• United Kingdom
  • Bristol, United Kingdom
    • Ep0216 607
  • London, United Kingdom
    • Ep0216 602
  • Manchester, United Kingdom
    • Ep0216 611
  • Sheffield, United Kingdom
    • Ep0216 604
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Ep0216 154
  • California
    • Los Angeles, California, United States, 90095
      • Ep0216 144
    • San Francisco, California, United States, 94158
      • Ep0216 101
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Ep0216 173
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Ep0216 149
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Ep0216 157
  • Massachusetts
    • Brookline, Massachusetts, United States, 02115
      • Ep0216 113
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Ep0216 134
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • Ep0216 166
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Ep0216 164
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104-4318
      • Ep0216 120
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Ep0216 124
  • Texas
    • Austin, Texas, United States, 78731
      • Ep0216 171

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 35 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subject has a confirmed pathogenic or likely pathogenic mutation in the CDKL5 gene and a clinical diagnosis of CDKL5 deficiency disorder (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.
• Subject must have failed to achieve seizure control despite previous or current use of 2 or more antiepileptic treatments (AETs).
• Subject is currently receiving at least 1 concomitant antiseizure treatment: antiseizure medication (ASM), vagus nerve stimulation (VNS), responsive neurostimulation (RNS), or ketogenic diet (KD).
• All medications or interventions for epilepsy (including VNS, RNS, and KD) must be stable prior to screening and are expected to remain stable throughout the study.
• At the Screening Visit, parent/caregiver reports that subject has ≥ 4 countable motor seizures (CMS) per week.

Exclusion Criteria:

• Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study drug.
• Subject has a diagnosis of pulmonary arterial hypertension.
• Subject has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, or portal hypertension, or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.
• Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, severe ventricular arrhythmias, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosus, and patent foramen ovale with reversal of shunt. (Note: Patent foramen ovale or a bicuspid aortic valve are not considered exclusionary).
• Subject has current eating disorder that suggests anorexia nervosa or bulimia.
• Subject has a current or past history of glaucoma.
• Subject is taking > 4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count.
• Subject is receiving concomitant treatment with cannabidiol (CBD) other than Epidiolex/Epidyolex or is being actively treated with tetrahydrocannabinol (THC) or any marijuana product for any condition.
• Subject has moderate to severe hepatic impairment.
• Subject is currently receiving another investigational product(s) or has received another investigational product within 30 days or within < 5 times the half-lives of the investigational product, whichever is longer, prior to the Screening Visit.
• Subject has previously been treated with Fintepla® (fenfluramine) prior to the Screening Visit.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Fenfluramine (hydrochloride) 0.8 mg/kg/day; Part 1: Fenfluramine (hydrochloride) 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.	Drug: Fenfluramine; Fenfluramine is supplied as an oral aqueous solution of fenfluramine hydrochloride.; Other Names: ZX008; FINTEPLA; fenfluramine HCl; fenfluramine hydrochloride
Placebo Comparator: Placebo; Part 1: Matching fenfluramine (hydrochloride) placebo will be administered twice a day (BID) in equally divided doses with or without food.	Drug: Placebo; Matching fenfluramine (hydrochloride) placebo is supplied as an oral solution.
Experimental: Fenfluramine (hydrochloride) Open-label; Part 2 and Part 3: Open-label fenfluramine (hydrochloride) will be administered using a flexible dosing regimen, up to fenfluramine 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]). Fenfluramine (hydrochloride) will be administered twice a day (BID) in equally divided doses with or without food.	Drug: Fenfluramine; Fenfluramine is supplied as an oral aqueous solution of fenfluramine hydrochloride.; Other Names: ZX008; FINTEPLA; fenfluramine HCl; fenfluramine hydrochloride

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage Change from Baseline in Countable Motor Seizure Frequency (CMSF) (Part 1)	The median percentage change from the Baseline in monthly (per 28 days) CMSF during the combined Titration and Maintenance Periods with the fenfluramine (ZX008) 0.8 mg/kg/day group compared with the placebo group will be reported.	Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) (Part 2)	An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. Treatment Emergent Adverse Events (TEAEs) are defined as any AEs reported on or after the first dose of study medication.	Up to OLE1 Treatment Period Post-Dose Safety Follow-up Visit 17 (up to 70 weeks)
Percentage of Participants With Abnormal Physical Examination Findings (Part 2)	Abnormal findings of physical exam that is considered clinically significant by Principal Investigator (PI).	Up to End of Treatment (EoT)/Early Termination (ET) Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Percentage of Participants With Abnormal Neurological Examination Findings (Part 2)	Abnormal findings of neurological exam that is considered clinically significant by PI.	Up to EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Percentage of Participants with Positive Response to Self-harm Question (Part 2)		OLE1 Treatment Period Day 1 to OLE1 Treatment Period Post-Dose Safety Follow-up Visit 17 (up to 70 weeks)
Percentage of Participants with Increase in Valvular Regurgitation from Baseline (except absent to trace) (Part 2)	Valvular regurgitation will be assessed using a 2-dimensional (2-D) Color Doppler Echocardiography (ECHO).	Baseline (28 days), Cardiac Follow-up Visit 18 (up to 96 weeks)
Percentage of Participants with Pulmonary Arterial Hypertension (PASP > 35 mmHg) at any Time During Treatment on Repeat Testing (Part 2)		OLE1 Treatment Period Day 1 to EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Laboratory Parameters (Hematology) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Laboratory Parameters (Hormones) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Laboratory parameters (Chemistry) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Laboratory Parameters (Urinalysis) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Vital Signs (Blood pressure) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Vital Signs (Heart rate) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Vital Signs (Temperature) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Vital Signs (Respiratory rate) (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Body Weight (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Change from Baseline in Tanner Staging (Part 2)		Baseline (28 days), EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)
Percentage of Participants with TEAEs (Part 3)	An AE is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. Treatment Emergent Adverse Events are defined as any AEs reported on or after the first dose of study medication.	Up to OLE2 Period Post-Dose Safety Follow-up (up to 200 weeks)
Change from Baseline in Height (Part 3)		Baseline (28 days), OLE2 Period EoT/ET Visit 23 (up to 198 weeks)
Change from Baseline in Body Weight (Part 3)		Baseline (28 days), OLE2 Period EoT/ET Visit 23 (up to 198 weeks)
Percentage of Participants with Increase in Valvular Regurgitation from Baseline (except absent to trace) (Part 3)	Valvular regurgitation will be assessed using a 2 D Color Doppler ECHO.	Baseline (28 days), Cardiac Follow-up Visit (up to 295 weeks)
Percentage of Participants with Pulmonary Arterial Hypertension (PASP > 35 mmHg) at any Time During Treatment on Repeat Testing (Part 3)		OLE2 Period Day 1 to EoT/ET Visit 23 of OLE2 Period (up to 198 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Achieve a ≥ 50% Reduction from Baseline in CMSF (Part 1)	The percentage of participants who achieve a ≥ 50% reduction from Baseline in CMSF during T+M periods, in the fenfluramine 0.8 mg/kg/day group compared with the placebo group.	Baseline (28 days), Combined T+M Periods (14 weeks)
Percentage of Participants with a Clinical Global Impression-Improvement (CGI-I) Rating of 'Much Improved' or 'Very Much Improved' as Assessed by Investigator (Part 1)	The percentage of participants who achieve a CGI-I rating of much or very much improved as assessed by the Investigator at the end of T+M periods, in the fenfluramine 0.8 mg/kg group compared with the placebo group. The CGI-I rating scale permits a global evaluation of the participant's improvement over time. The severity of a participant's condition is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse) as follows: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse.	At the end of the combined T+M Periods (14 weeks)
Percentage Change From Baseline in Monthly Generalized Tonic-Clonic (GTC) Seizure Frequency (Part 1)	The median percentage change from Baseline in monthly GTC seizure frequency during T+M periods, in the fenfluramine 0.8 mg/kg/day group compared with the placebo group.	Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)
Categorized Percentage Change in Seizures from Baseline in CMSF (Part 1)	The categorized median percentage change in seizures from the Baseline in monthly (per 28 days) CMSF during the combined T+ M periods will be presented in the following categories: no reduction or worsening, ≥25%, ≥75%, or 100% reduction.	Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)
Percentage of Participants who Achieve "Near Seizure Freedom" (Part 1)	The percentage of participants who achieve near seizure freedom, defined as 0 or 1 seizures, during T+M, in the fenfluramine 0.8 mg/kg/day group compared with the placebo group.	Combined T+M Periods (14 weeks)
Percentage of Participants with a CGI-I Rating of 'Much Improved' or 'Very Much Improved' as Assessed by the Parent/Caregiver (Part 1)	The percentage of participants who achieve a CGI-I rating of much or very much improved as assessed by the by the parent/caregiver at the end of T+M periods, in the fenfluramine 0.8 mg/kg group compared with the placebo group.	At the end of the combined T+M Periods (14 weeks)
Percentage of Participants with Improvement (Minimal, Much or Very Much Improved) on the CGI-I Rating as Assessed, Independently, by the Investigator (Part 1)	The percentage of participants who achieve a CGI-I rating of minimal, much or very much improved as assessed, independently, by the Investigator at the end of T+M periods, in the fenfluramine 0.8 mg/kg group compared with the placebo group.	At the end of the combined T+M Periods (14 weeks)
Percentage of Participants with Improvement (Minimal, Much or Very Much Improved) on the CGI-I Rating as Assessed, Independently, by the Parent/Caregiver (Part 1)	The percentage of participants who achieve a CGI-I rating of minimal, much or very much improved as assessed, independently, by the parent/caregiver at the end of T+M periods, in the fenfluramine 0.8 mg/kg group compared with the placebo group.	At the end of the combined T+M Periods (14 weeks)
Percentage Change from Baseline in the Monthly Frequency of all Seizures (Part 1)	The percentage change from the Baseline in the monthly (per 28 days) frequency of all seizures during the combined T+M period.	Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)
Change from Baseline in the Monthly Frequency of Countable Motor Seizure (CMS)-free Days (Part 1)	The change from the Baseline in the monthly (per 28 days) frequency of CMS-free days during the combined T+M periods.	Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)
Percentage of Participants with TEAEs (Part 1)	An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. A TEAEs was defined as any adverse events with a start date/time on or after dosing of the study medication and up to end of the maintenance period (14 weeks) inclusive after dosing of the study medication.	From Day of first dose to the End of the Maintenance Periods (up to 14 weeks)
Percentage of Participants With Abnormal Physical Examination Findings (Part 1)		Baseline, Visit 6 (Day 43), Visit 7 (Day 71), Visit 8 (Day 99)
Percentage of Participants With Abnormal Neurological Examination Findings (Part 1)		Baseline, Visit 6 (Day 43), Visit 8 (Day 99)
Percentage of Participants with Positive Response to Self-harm question (Part 1)		Baseline, Visit 5 (Day 15), Visit 6 (Day 43), Visit 7 (Day 71), Visit 8 (Day 99)
Percentage of Participants with Increase in Valvular Regurgitation from Baseline (except absent to trace) (Part 1)	Valvular regurgitation will be assessed using a 2-D Color Doppler ECHO.	From Day of first dose to the End of the Maintenance Period (up to 14 weeks)
Percentage of Participants with Pulmonary Arterial Hypertension (PASP > 35 mmHg) at any Time During Treatment on Repeat Testing (Part 1)	The safety and tolerability of fenfluramine (ZX008) will be assessed in pediatric and adult participants with CDD.	From Day of first dose to the End of the Maintenance Period (up to 14 weeks)
Change from Baseline in Laboratory parameters (Hematology) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Laboratory parameters (Hormones) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Laboratory parameters (Chemistry) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Laboratory Parameters (Urinalysis) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Vital signs (Blood pressure) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Vital signs (Heart rate) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Vital signs (Temperature) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Vital signs (Respiratory rate) (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Body Weight (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Change from Baseline in Tanner Staging (Part 1)		Baseline (28 days), end of the combined Titration and Maintenance (T+M) Periods (Day 99)
Percentage Change from Baseline in CMSF (Part 2)	The median percentage change from the Baseline in monthly (per 28 days) CMSF during the OLE Treatment Period.	Baseline (28 days), OLE1 Treatment Period (up to 52 weeks)
Categorized Percentage Change in Seizures from Baseline in CMSF (Part 2)	The categorized percentage change in seizures from the Baseline in monthly (per 28 days) CMSF during OLE Treatment Period will be presented in the following categories: no reduction or worsening, ≥25%, ≥50%, ≥75%, or 100% reduction.	Baseline (28 days), OLE1 Treatment Period (up to 52 weeks)
Percentage of Participants who Achieve "Near Seizure Freedom" (Part 2)	The percentage of participants who achieve near seizure freedom, defined as 0 or 1 seizures, during the OLE Treatment Period.	OLE1 Treatment Period (up to 52 weeks)
Percentage of Participants with a CGI-I Rating of 'Much or Very Much Improved' as Assessed by the Investigator (Part 2)	The percentage of participants who achieve a CGI-I rating of much or very much improved as assessed by the Investigator at the OLE Treatment Period.	At the End of the OLE1 Treatment Period (up to 52 weeks)
Percentage of Participants with a CGI-I Rating of 'Much or Very Much Improved' as Assessed by the Parent/Caregiver (Part 2)	The percentage of participants who achieve a CGI-I rating of much or very much improved as assessed by the parent/caregiver at the OLE Treatment Period.	At the End of the OLE1 Treatment Period (up to 52 weeks)
Percentage of Participants with Improvement (Minimal, Much or Very Much Improved) on the CGI-I Rating as Assessed, Independently, by the Investigator (Part 2)	The percentage of participants who achieve a CGI-I rating of minimal, much or very much improved as assessed by the Investigator at the OLE Treatment Period.	At the End of the OLE1 Treatment Period (up to 52 weeks)
Percentage of Participants with Improvement (Minimal, Much or Very Much Improved) on the CGI-I Rating as Assessed by the Parent/Caregiver (Part 2)	The percentage of participants who achieve a CGI-I rating of minimal, much or very much improved as assessed by the parent/caregiver at the OLE Treatment Period.	At the End of the OLE1 Treatment Period (up to 52 weeks)
Percentage Change from Baseline in Monthly GTC Seizure Frequency (Part 2)	The median percentage change from baseline in monthly GTC seizure frequency during OLE Treatment Period.	Baseline (28 days), OLE1 Treatment Period (up to 52 weeks)
Change from Baseline in the Monthly Frequency of CMS-free Days (Part 2)	The change from the Baseline in the monthly (per 28 days) frequency of CMS-free days during the OLE Treatment Period.	Baseline (28 days), OLE1 Treatment Period (up to 52 weeks)

Collaborators and Investigators

• Sponsor: Zogenix, Inc.
• Investigators: Study Director: UCB Cares, 001 844 599 2273

Study record dates

Study Major Dates

• Study Start (Actual): March 8, 2022
• Primary Completion (Estimated): November 8, 2027
• Study Completion (Estimated): November 8, 2027

Study Registration Dates

• First Submitted: September 22, 2021
• First Submitted That Met QC Criteria: September 22, 2021
• First Posted (Actual): October 1, 2021

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: CDKL5; fenfluramine; CDD
• Additional Relevant MeSH Terms: Epileptic Syndromes; Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epilepsy; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Seizures; Epilepsy, Tonic-Clonic; Spasms, Infantile; CDKL5 deficiency disorder; Organic Chemicals; Amines; Phenethylamines; Ethylamines; Fenfluramine
• Other Study ID Numbers: ZX008-2103/EP0216; 2021-003222-76 (EudraCT Number); 2023-506269-78-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• IPD Sharing Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• IPD Sharing Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No