A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 29 April 2024
• Main ID: NCT03355209
• Date of registration: 19/06/2017
• Prospective Registration: Yes
• Primary sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
• Public title: A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome
• Scientific title: A Two-Part Study of ZX008 in Children and Adults With Lennox-Gastaut Syndrome (LGS); Part 1: A Randomized, Double-blind, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults With LGS, Followed by Part 2: An Open-label Extension to Assess Long-Term Safety of ZX008 in Children and Adults With LGS
• Date of first enrolment: November 27, 2017
• Target sample size: 296
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03355209
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Spain, Sweden, 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 35 years, inclusive as of the day
of the Screening Visit.

- Clinical diagnosis of Lennox-Gastaut syndrome, where seizures that result in drops are
not completely controlled by current antiepileptic treatments.

- Onset of seizures at 11 years of age or younger.

- Abnormal cognitive development.

- Must be receiving at least 1 concomitant AED and up to 4 concomitant anti-epileptic
treatments.

Key Exclusion Criteria:

- Etiology of seizures is a degenerative neurological disease.

- History of hemiclonic seizures in the first year of life.

- Subject only has drop seizures in clusters, where individual seizures cannot be
counted reliably.

- Pulmonary arterial hypertension.

- Current or past history of cardiovascular or cerebrovascular disease, such as cardiac
valvulopathy, myocardial infarction or stroke.

- 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.

- Taking felbamate for less than 1 year prior to screening and/or does not have stable
liver function and hematology laboratory tests, and/or the dose has not been stable
for at least 60 days prior to the Screening Visit.

- Currently receiving an investigational product.

- Institutionalized in a general nursing home (ie, in a facility that does not
specialize in epilepsy care).

- 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.



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

Health Condition(s) or Problem(s) studied

Lennox Gastaut Syndrome

Intervention(s)

Drug: ZX008 0.2 or 0.8 mg/kg/day

Drug: Matching Placebo

Primary Outcome(s)

Part 1: Change from baseline in frequency of seizures that result in drops in subjects receiving ZX008 compared to placebo [Time Frame: Up to 20 weeks maintenance and taper period (T+M)]

Secondary Outcome(s)

Part 2: Adverse events and related safety parameters in subjects receiving ZX008 compared to placebo [Time Frame: Up to 12 months open label]

Secondary ID(s)

2017-002628-26

ZX008-1601

Source(s) of Monetary Support

Please refer to primary and secondary sponsors