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An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

Primary Purpose

Dravet Syndrome

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

ZX008 (Fenfluramine Hydrochloride)

About this trial

This is an interventional treatment trial for Dravet Syndrome focused on measuring seizure, tonic clonic, epilepsy, myoclonic, encephalopathy

Eligibility Criteria

2 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
Satisfactory completion of the core study in the opinion of the investigator and the sponsor.
Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation.
A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
Subject's parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant).

Key Exclusion Criteria:

Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication.
Current or past history of glaucoma.
Moderate or severe hepatic impairment.
Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness at Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

Sites / Locations

Phoenix Children's Hospital
Center for Neurosciences - Tucson
Children's Hospital of Orange County
Rady Children's Hospital San Diego
University of California San Francisco
The Children's Hospital Colorado
NW FL Clinical Research Group, LLC
Miami Children's Hospital Brain Institute
Neurology and Epilepsy Research Center
Clinical Integrative Research Center of Atlanta, Panda Neurology
Ann and Robert H. Lurie Children's Hospital of Chicago
Massachusetts General Hospital
Boston Children's Hospital
Children's Hospital Of Michigan
Mayo Clinic
Minnesota Epilepsy Group, P.A.
Institute of Neurology and Neurosurgery at St. Barnabus
University Hospitals Cleveland Medical Center
Children's Hospital of Philadelphia
Le Bonheur Children's Hospital
Cook Children's Medical Center
University of Utah
Seattle Children's Hospital
MultiCare Institute for Research & Innovation
Melbourne Brain Centre Austin Hospital
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital
The Children's Hospital Westmead Dept. of Neurology and Neurosurgery
Universitair Ziekenhuis Antwerpen
Centre Hospitalier Universitaire Sainte-Justine
British Columbia Children's Hospital
Danish National Epilepsy Centre
CHU Amiens-Picardie Service De Neurologie Pediatrique
CHU De Bordeaux Service De Pédiatrie Médicale
CHRU Lille Antenne Pédiatrique Du CIC - Hôpital Jeanne De Flandre
HOPITAL DEL LA TIMONE - HOPITAL HENRI GASTAUT Hôpital De La Timone Neurologie Pédiatrique Pneumologie Pédiatrique Et Médecine Infantile
Hôpital Robert Debré Pôle: Pédiatrie Médicale Service: Neurologie Et Maladies Métaboliques
Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique Centre de référence épilepsies rares (CReER)
DRK Kliniken Berlin - Westend Epilepsiezentrum / Neuropaediatrie
Krankenhaus Mara Epilepsie-Zentrum Bethel
Universitaetsklinikum Freiburg Zentrum fuer Kinder- und Jugendmedizin
Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie
Universitaetsklinikum Schleswig-Holstein Campus Kiel Klinik fuer Neuropaediatrie
Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH
Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III
Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie
AOU Anna Meyer Clinica di Neurologia Pediatrica
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia
A.O. Carlo Poma
Istituto Neurologica Carlo Besta
Ospedale Fatebenefratelli e Oftalmico
Policlinico A. Gemelli
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù
Ospedale Civile Maggiore di Borgo Trento - Ospedale della Donna e del Bambino
Saitama Children's Medical Center
National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders
Tokyo Women's Medical University Hospital
Kempenhaeghe
Stichting Epilepsie Instellingen Nederland
Hospital Sant Joan de Déu
Hospital Ruber Internacional Primera Planta Servicio de Neurologia
Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria
Birmingham Children Hospital NHS Foundation Trust
Institute of Neurosciences Queen Elizabeth University Hospital
Alder Hey Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust
St. Thomas Hospital
Sheffield Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ZX008

Arm Description

ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).

Outcomes

Primary Outcome Measures

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period

Treatment-emergent adverse events (TEAE) were defined as any AEs that based on start date information occurs after the first intake of study treatment.

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period

A TEAE was defined as any AE that based on start date information occurs after the first intake of study treatment. Percentage of participants with TEAEs leading to withdrawal from IMP during OLE Treatment Period were reported.

Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period

Serious Adverse event (SAE) was defined as any untoward medical occurrence that at any dose: • results in death, • is life-threatening threatening, • results in initial inpatient hospitalization or prolongation of hospitalization, •results in persistent or significant disability or incapacity, • results in a congenital anomaly/birth defect, • results in any medically significant event that did not meet any of the other 5 SAE criteria, but which was judged by a physician to potentially jeopardize the participant or require medical or surgical intervention to prevent one of the above outcomes listed as an SAE criterion.

Secondary Outcome Measures

Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period

Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies (ZX008-1501/ZX008-1502, and ZX008-1504 Cohort 2). Participants in 1504- Cohort 1 and de novo participants (who entered 1503 without having been in any of the core studies) did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Day 1 to EOS was divided by the total number of days from Day 1 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day convulsive seizure frequency (CSF). The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant.

Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period

Baseline (Core) was defined as Baseline prior to double-blind treatment in the core studies. Participants in 1504-Cohort 1 and de novo participants did not have a Baseline (Core), and were not included in the analysis of this outcome measure. The total number of convulsive seizures from Month 2 to EOS was divided by the total number of days from Month 2 to EOS with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF. The change from Baseline for any individual participant was calculated by subtracting the Baseline (Core) from the post-baseline value. Monthly (28 day) CSF was based on electronic diary data obtained for each participant.

Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36)

Monthly (28 day) CSF was based on electronic diary data obtained for each participant. The total number of convulsive seizures in the ith interval (CSF in OLE, where, i=1, 2, 3, … , 14 ) was divided by the total number of days in the ith interval with nonmissing diary data and the result was then multiplied by 28 to get a 28-day CSF of OLE.

Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period

Convulsive seizure frequency over time, reported as per 28 days was analyzed by the actual dose administered. Participants were grouped into low (0.2 to <0.4 mg/kg), medium (0.4 to <0.6 mg/kg), and high dose (>0.6 mg/kg) groups depending on their mean daily doses of ZX008 during the OLE Treatment period. For each participant, the seizure frequency per 28 days was calculated as the number of seizures recorded during the period, divided by the number of days in the period and multiplied by 28. The convulsive seizure frequency was calculated from all available data collected.

Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period

Participants in the study were required to be on stable background therapy for the first 6 months of treatment, after which background AEDs could be reduced or withdrawn so long as one background AED remained. The percentage of participants who had changes in dose or type of concomitant AED medications during the first, second, third, fourth, fifth, and sixth months were analyzed and reported.

Full Information

NCT ID

NCT02823145

First Posted

June 13, 2016

Last Updated

September 22, 2023

Sponsor

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02823145

Brief Title

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

Official Title

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Completed

Study Start Date

June 8, 2016 (Actual)

Primary Completion Date

January 27, 2023 (Actual)

Study Completion Date

January 27, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.

Detailed Description

This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dravet Syndrome

Keywords

seizure, tonic clonic, epilepsy, myoclonic, encephalopathy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

375 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ZX008

Arm Type

Experimental

Arm Description

ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).

Intervention Type

Drug

Intervention Name(s)

ZX008 (Fenfluramine Hydrochloride)

Primary Outcome Measure Information:

Title

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period

Description

Treatment-emergent adverse events (TEAE) were defined as any AEs that based on start date information occurs after the first intake of study treatment.

Time Frame

From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)

Title

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period

Description

Time Frame

From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)

Title

Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period

Description

Time Frame

From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)

Secondary Outcome Measure Information:

Title

Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period

Description

Time Frame

From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)

Title

Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period

Description

Time Frame

From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)

Title

Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36)

Description

Time Frame

At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36

Title

Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period

Description

Time Frame

From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)

Title

Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period

Description

Time Frame

At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit. Satisfactory completion of the core study in the opinion of the investigator and the sponsor. Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation. A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs. Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability. Subject's parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant). Key Exclusion Criteria: Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke. Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication. Current or past history of glaucoma. Moderate or severe hepatic impairment. Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates. Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy. A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness at Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

UCB Cares

Organizational Affiliation

001 844 599 2273

Official's Role

Study Director

Facility Information:

Facility Name

Phoenix Children's Hospital

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85016

Country

United States

Facility Name

Center for Neurosciences - Tucson

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85718

Country

United States

Facility Name

Children's Hospital of Orange County

City

Orange

State/Province

California

ZIP/Postal Code

92868

Country

United States

Facility Name

Rady Children's Hospital San Diego

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

University of California San Francisco

City

San Francisco

State/Province

California

ZIP/Postal Code

94158

Country

United States

Facility Name

The Children's Hospital Colorado

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

NW FL Clinical Research Group, LLC

City

Gulf Breeze

State/Province

Florida

ZIP/Postal Code

32561

Country

United States

Facility Name

Miami Children's Hospital Brain Institute

City

Miami

State/Province

Florida

ZIP/Postal Code

33155

Country

United States

Facility Name

Neurology and Epilepsy Research Center

City

Orlando

State/Province

Florida

ZIP/Postal Code

32819

Country

United States

Facility Name

Clinical Integrative Research Center of Atlanta, Panda Neurology

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30328

Country

United States

Facility Name

Ann and Robert H. Lurie Children's Hospital of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Children's Hospital Of Michigan

City

Detroit

State/Province

Michigan

ZIP/Postal Code

48201

Country

United States

Facility Name

Mayo Clinic

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

Facility Name

Minnesota Epilepsy Group, P.A.

City

Saint Paul

State/Province

Minnesota

ZIP/Postal Code

55102

Country

United States

Facility Name

Institute of Neurology and Neurosurgery at St. Barnabus

City

Livingston

State/Province

New Jersey

ZIP/Postal Code

07039

Country

United States

Facility Name

University Hospitals Cleveland Medical Center

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44103

Country

United States

Facility Name

Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Le Bonheur Children's Hospital

City

Memphis

State/Province

Tennessee

ZIP/Postal Code

38103

Country

United States

Facility Name

Cook Children's Medical Center

City

Fort Worth

State/Province

Texas

ZIP/Postal Code

76104

Country

United States

Facility Name

University of Utah

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84113

Country

United States

Facility Name

Seattle Children's Hospital

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

Facility Name

MultiCare Institute for Research & Innovation

City

Tacoma

State/Province

Washington

ZIP/Postal Code

98405

Country

United States

Facility Name

Melbourne Brain Centre Austin Hospital

City

Heidelberg

Country

Australia

Facility Name

Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital

City

South Brisbane

Country

Australia

Facility Name

The Children's Hospital Westmead Dept. of Neurology and Neurosurgery

City

Westmead

Country

Australia

Facility Name

Universitair Ziekenhuis Antwerpen

City

Edegem

Country

Belgium

Facility Name

Centre Hospitalier Universitaire Sainte-Justine

City

Montréal

Country

Canada

Facility Name

British Columbia Children's Hospital

City

Vancouver

Country

Canada

Facility Name

Danish National Epilepsy Centre

City

Dianalund

Country

Denmark

Facility Name

CHU Amiens-Picardie Service De Neurologie Pediatrique

City

Amiens

Country

France

Facility Name

CHU De Bordeaux Service De Pédiatrie Médicale

City

Bordeaux

Country

France

Facility Name

CHRU Lille Antenne Pédiatrique Du CIC - Hôpital Jeanne De Flandre

City

Lille

Country

France

Facility Name

HOPITAL DEL LA TIMONE - HOPITAL HENRI GASTAUT Hôpital De La Timone Neurologie Pédiatrique Pneumologie Pédiatrique Et Médecine Infantile

City

Marseille

Country

France

Facility Name

Hôpital Robert Debré Pôle: Pédiatrie Médicale Service: Neurologie Et Maladies Métaboliques

City

Paris

Country

France

Facility Name

Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique Centre de référence épilepsies rares (CReER)

City

Paris

Country

France

Facility Name

DRK Kliniken Berlin - Westend Epilepsiezentrum / Neuropaediatrie

City

Berlin

Country

Germany

Facility Name

Krankenhaus Mara Epilepsie-Zentrum Bethel

City

Bielefeld

Country

Germany

Facility Name

Universitaetsklinikum Freiburg Zentrum fuer Kinder- und Jugendmedizin

City

Freiburg

Country

Germany

Facility Name

Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie

City

Jena

Country

Germany

Facility Name

Universitaetsklinikum Schleswig-Holstein Campus Kiel Klinik fuer Neuropaediatrie

City

Kiel

Country

Germany

Facility Name

Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH

City

Radeberg

Country

Germany

Facility Name

Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III

City

Tübingen

Country

Germany

Facility Name

Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie

City

Vogtareuth

Country

Germany

Facility Name

AOU Anna Meyer Clinica di Neurologia Pediatrica

City

Firenze

Country

Italy

Facility Name

Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia

City

Genova

Country

Italy

Facility Name

A.O. Carlo Poma

City

Mantova

Country

Italy

Facility Name

Istituto Neurologica Carlo Besta

City

Milano

Country

Italy

Facility Name

Ospedale Fatebenefratelli e Oftalmico

City

Milano

Country

Italy

Facility Name

Policlinico A. Gemelli

City

Roma

Country

Italy

Facility Name

U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù

City

Roma

Country

Italy

Facility Name

Ospedale Civile Maggiore di Borgo Trento - Ospedale della Donna e del Bambino

City

Verona

Country

Italy

Facility Name

Saitama Children's Medical Center

City

Saitama

Country

Japan

Facility Name

National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders

City

Shizuoka

Country

Japan

Facility Name

Tokyo Women's Medical University Hospital

City

Tokyo

Country

Japan

Facility Name

Kempenhaeghe

City

Heeze

Country

Netherlands

Facility Name

Stichting Epilepsie Instellingen Nederland

City

Zwolle

Country

Netherlands

Facility Name

Hospital Sant Joan de Déu

City

Barcelona

Country

Spain

Facility Name

Hospital Ruber Internacional Primera Planta Servicio de Neurologia

City

Madrid

Country

Spain

Facility Name

Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria

City

Pamplona

Country

Spain

Facility Name

Birmingham Children Hospital NHS Foundation Trust

City

Birmingham

Country

United Kingdom

Facility Name

Institute of Neurosciences Queen Elizabeth University Hospital

City

Glasgow

Country

United Kingdom

Facility Name

Alder Hey Hospital

City

Liverpool

Country

United Kingdom

Facility Name

Great Ormond Street Hospital for Children NHS Foundation Trust

City

London

Country

United Kingdom

Facility Name

St. Thomas Hospital

City

London

Country

United Kingdom

Facility Name

Sheffield Children's Hospital

City

Sheffield

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing 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 prespecified 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 URL

https://www.Vivli.org

Citations:

PubMed Identifier

34768178

Citation

Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.

Results Reference

derived

Learn more about this trial

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

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