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 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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