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

• Register: ClinicalTrials.gov
• Last refreshed on: 3 October 2023
• Main ID: NCT02823145
• Date of registration: 13/06/2016
• Prospective Registration: No
• Primary sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
• Public 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
• Scientific 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
• Date of first enrolment: June 8, 2016
• Target sample size: 375
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02823145
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Norway, Spain, Sweden, United Kingdom, United States

Contacts

• Name: UCB Cares
• Affiliation: 001 844 599 2273

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 35 Years
Gender: All

Health Condition(s) or Problem(s) studied

Dravet Syndrome

Intervention(s)

Drug: ZX008 (Fenfluramine Hydrochloride)

Primary Outcome(s)

Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period [Time Frame: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)]

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period [Time Frame: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)]

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period [Time Frame: From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)]

Secondary Outcome(s)

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

Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period [Time Frame: From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)]

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

Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period [Time Frame: At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period]

Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period [Time Frame: From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)]

Secondary ID(s)

2016-002804-14

ZX008-1503

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/09/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02823145