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 |
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Recruitment Status :
Enrolling by invitation
First Posted : July 6, 2016
Last Update Posted : February 22, 2018
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Sponsor:
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )
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Brief Summary:
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who have successfully completed 14 weeks of treatment in the core study (ZX008-1501 and ZX008-1502).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Dravet Syndrome | Drug: ZX008 (Fenfluramine Hydrochloride) | Phase 3 |
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who have successfully completed 14 weeks of treatment in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. Subjects will be permitted to participate in the present study after they complete the final evaluation at Day 113 in one of the core studies. This trial will consist of a 12-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period. Thus, subjects who complete this trial will have been treated with ZX0008 for a minimum of up to 1 year (including their participation in both the core study and this study).
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 210 participants |
| 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 |
| Study Start Date : | June 2016 |
| Estimated Primary Completion Date : | December 2018 |
| Estimated Study Completion Date : | December 2018 |
Resource links provided by the National Library of Medicine
| 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) |
Primary Outcome Measures :
- 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 54 weeks ]Sensitivity of the key efficacy analysis will be assessed for changes in dose or type of concomitant AED medications
Secondary Outcome Measures :
- Proportion of subjects who achieve reduction from baseline in convulsive seizure frequency [ Time Frame: Baseline up to 54 weeks ]
- The longest interval between convulsive seizures will be calculated for each subject over the entire open-label treatment period. [ Time Frame: Up to 54 weeks ]
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| Ages Eligible for Study: | 2 Years to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
- 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 the investigator, parent, IPCAB, IDSMC, or sponsor deems 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.
- Currently receiving or has received stiripentol in the past 21 days prior to Visit 1.
- Unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with the Visit 1 and throughout the study.
- 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.
Information from the National Library of Medicine
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
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Sponsors and Collaborators
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
| Responsible Party: | Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. |
| ClinicalTrials.gov Identifier: | NCT02823145 History of Changes |
| Other Study ID Numbers: |
ZX008-1503 |
| First Posted: | July 6, 2016 Key Record Dates |
| Last Update Posted: | February 22, 2018 |
| Last Verified: | February 2018 |
Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
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seizure tonic clonic epilepsy myoclonic encephalopathy |
Additional relevant MeSH terms:
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Syndrome Epilepsies, Myoclonic Disease Pathologic Processes Epilepsy Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Fenfluramine Serotonin Uptake Inhibitors Neurotransmitter Uptake Inhibitors Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Neurotransmitter Agents Serotonin Agents Physiological Effects of Drugs |