A Two-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥2 Years Old and Young Adults With Dravet Syndrome
a study on Dravet Syndrome
The primary purpose of this study is to evaluate the safety, tolerability and efficacy of a single dose of ZX008 (Fenfluramine Hydrochloride) when added to standard of care and when added to Adjunctive Antiepleptic therapy to Stiripentol treatment in children and young adults with Dravet Syndrome
A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care , Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults With Dravet Syndrome
This is a multicenter, two-cohort trial to assess the pharmacokinetic and safety profile of a single dose of ZX008 (fenfluramine hydrochloride) oral solution when added to Dravet syndrome treatment regimen containing VPA and CLB, with or without STP (Cohort 1), followed by a randomized, double-blind, placebo-controlled parallel group evaluation of the efficacy, safety, and tolerability of ZX008 as adjunctive therapy for seizures in children and young adults with Dravet syndrome (Cohort 2). Cohort 2 will not be dosed until the PK and safety data from Cohort 1 have been collected and evaluated. The PK and safety data from Cohort 1 will determine the dose of ZX008 to be used in Cohort 2.
Dravet Syndrome seizure tonic clonic epilepsy myoclonic encephalopathy Syndrome Epilepsies, Myoclonic Pharmaceutical Solutions Fenfluramine
For people ages 2–18
Key Inclusion Criteria:
- Subject must be male or non-pregnant, non-lactating female, age 2 to 18 years(inclusive)
- Subject must have documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
- Subject must be receiving a therapeutically relevant and stable dose of CLB, VP, and STP for at least 4 weeks prior to screening and are expected to remain stable throughout the study (Cohort 2 only).
- Subject must be receiving a stable dose of CLB and VPA, administered twice daily, to be eligible for Dose Regimen 1 and 2 or subject must be receiving a stable dose of CLB, VPA, and STP, administered twice daily, to be eligible for Dose Regimen 3 (Cohort 1 only).
Key Exclusion Criteria:
- Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study medication.
- Subject has pulmonary arterial hypertension.
- Subject has a current or past history of cardiovascular or cerebrovascular disease,such as cardiac valvulopathy, myocardial infarction or stroke.
- Subject has a current or recent history of anorexia nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.
- Subject has a current or past history of glaucoma.
- Subject is receiving concomitant therapy with: centrally-acting anorectic agents;monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin re-uptake inhibition; triptans, atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or CYP 2D6/3A4/2B6 inhibitors/substrates.
- Subject is currently taking carbamazepine, oxcarbamazepine, eslicarbazepine,phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
- Subject has a positive result on urine THC Panel or whole blood CBD at the Screening Visit.
- Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness 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.
- University of California San Francisco accepting new patients
San Francisco, California, 94143, United States
- British Columbia Children'S Hospital not yet accepting patients
Vancouver, British Columbia, V6H3V4, Canada
- The Children'S Hospital Colorado accepting new patients
Aurora, Colorado, 80045, United States
- Mayo Clinic accepting new patients
Rochester, Minnesota, 55905, United States
- Ann & Robert Lurie Children'S Hospital of Chicago accepting new patients
Chicago, Illinois, 60611, United States
- Centre Hospitalier Universitaire Sainte-Justine not yet accepting patients
Montreal, Quebec, H3T 1C5, Canada
- Institute of Neurosciences Queens Elizabeth University not yet accepting patients
Glasgow, G51 4TF, United Kingdom
- Alder Hey Children'S Nhs Foundation Trust not yet accepting patients
Liverpool, L12 2AP, United Kingdom
- Evelina Hospital not yet accepting patients
London, SE1 7EH, United Kingdom
- Great Ormonnd Street Hospital For Children Nhs Foundation Trust not yet accepting patients
London, WC1N 3JH, United Kingdom
- Kempenhaeghe not yet accepting patients
Heeze, 5590 Ab, Netherlands
- Stichting Epilepsie Instellingen Nederland accepting new patients
Zwolle, 8025 BV, Netherlands
- Chu Amiens Picardie Service de Neurologie Pediatrique accepting new patients
Amiens, 80054, France
- CHU DE BORDEAUX Service De Pédiatrie Médicale accepting new patients
Bordeaux,, 33000, France
- CHRU LILLE Centre Hospitalier Régional Universitaire De Lille 2 accepting new patients
Lille, 59037, France
- HÔPITAL FEMME-MÈRE-ENFANT Hôpital Femme-mère-enfant Service De Neurologie Pédiatrique not yet accepting patients
Lyon Bron, 69500, France
- Hôpital La Timone, Service de Neurologie Pédiatrique accepting new patients
Marseille, 13385, France
- Hôpital Necker accepting new patients
Paris, 75743, France
- Hôpital Robert Debré accepting new patients
Paris, 75743, France
- CENTRE REFERENT DES EPILEPSIES Hopital De Hautpierre not yet accepting patients
Strasbourg Cedex, F-67098, France
- HÔPITAL DES ENFANTS Pédiatrie accepting new patients
Toulouse Cedex 9, 31059, France
- Hopital Denfants Chru de Nancy accepting new patients
Vandoeuvre Les Nancy Cedex, 54511, France
- Krankenhaus Mara Ggmbh, Epilepsiezentrum Bethel not yet accepting patients
Bielefeld, 33617, Germany
- Universitätsklinikum Schleswig-Holstein, Klinik Für Neuropädiatrie not yet accepting patients
Kiel, 24105, Germany
- Kleinwachau Sächsisches Epilepsiezentrum Radeberg Gemeinnützige Gmbh not yet accepting patients
Radeberg, 1454, Germany
- Hospital Sant Joan de Déu not yet accepting patients
Barcelona, 08950, Spain
- Hospital Ruber Internacional Primera Planta Servicio de Neurologia not yet accepting patients
Madrid, 28034, Spain
- Clinica Universitaria de Navarra not yet accepting patients
Pamplona, 31008, Spain
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