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

JRCT ID: jRCT2041200095

Registered date:29/01/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Dravet syndrome
• Date of first enrollment: 18/11/2019
• Target sample size: 390
• Countries of recruitment: Australia,Japan,Belgium,Japan,Canda,Japan,Denmark,Japan,France,Japan,Germany,Japan,Italy,Japan,Korea,Japan,Netherland,Japan,Norway,Japan,Spain,Japan,Sweeded,Japan,UK,Japan,US,Japan
• Study type: Interventional

Intervention(s)

ZX008 (Fenfluramine Hydrochloride) Oral Solution
Initial dose: 0.2mg/kg, Max daily dose: 30mg

Outcome(s)

• Primary Outcome: Long-term safety and tolerability as measured by treatment emergent adverse events, including clinical labs, vital signs, and examination findings.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 35age old
• Gender: Both

Include 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.
- 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).

Exclude criteria

- Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
- 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.

Related Information

• Primary Sponsor: YAMAMOTO Hideichiro
• Secondary ID(s): NCT02823145