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
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ClinicalTrials.gov Identifier: NCT02823145 |
Recruitment Status :
Active, not recruiting
First Posted : July 6, 2016
Last Update Posted : November 9, 2022
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Condition or disease | Intervention/treatment | Phase |
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Dravet Syndrome | Drug: ZX008 (Fenfluramine Hydrochloride) | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 373 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official 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 |
Actual Study Start Date : | July 5, 2016 |
Estimated Primary Completion Date : | February 7, 2023 |
Estimated Study Completion Date : | February 7, 2023 |

Arm | Intervention/treatment |
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Experimental: ZX008
ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).
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Drug: ZX008 (Fenfluramine Hydrochloride) |
- Long-term safety and tolerability as measured by treatment emergent adverse events, including clinical labs, vital signs, and examination findings. [ Time Frame: Pre-baseline Up to 156 weeks ]Sensitivity of the key efficacy analysis will be assessed for changes in dose or type of concomitant AED medications
- Proportion of subjects who achieve reduction from baseline in convulsive seizure frequency [ Time Frame: Baseline up to 156 weeks ]
- The longest interval between convulsive seizures will be calculated for each subject over the entire open-label treatment period. [ Time Frame: Up to 156 weeks ]

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Ages Eligible for Study: | 2 Years to 35 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02823145

Study Director: | UCB Cares | 001 844 599 2273 |
Responsible Party: | Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. |
ClinicalTrials.gov Identifier: | NCT02823145 |
Other Study ID Numbers: |
ZX008-1503 2016-002804-14 ( EudraCT Number ) |
First Posted: | July 6, 2016 Key Record Dates |
Last Update Posted: | November 9, 2022 |
Last Verified: | November 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
seizure tonic clonic epilepsy myoclonic encephalopathy |
Epilepsies, Myoclonic Syndrome Disease Pathologic Processes Epilepsy, Generalized Epilepsy Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Epileptic Syndromes Fenfluramine Serotonin Uptake Inhibitors Neurotransmitter Uptake Inhibitors Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Neurotransmitter Agents Serotonin Agents Physiological Effects of Drugs |