ZX008 Expanded Access Protocol

NCT ID: NCT03780127

Last Updated: 2023-10-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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The treatment plan for this Expanded Access Protocol is for patients with Dravet syndrome who do not qualify for participation in one of the ongoing ZX008 clinical trials.

Detailed Description

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The treatment plan consists of an up to 24-month Treatment Period. Access is via application by your health care provider and available at one of the Expanded Access treatment centers. The dose of ZX008 for the duration of the Treatment Period will range from 0.2 mg/kg/day to a maximum of 0.8 mg/kg/day, not to exceed a total daily dose of 30 mg/day; for patients concurrently being prescribed stiripentol, the maximum will be 0.5mg/kg/day, not to exceed a total daily dose of 20 mg/day.

The fenfluramine US Expanded Access Program (US EAP) is available to full-time US Residents only.

Participation in the US EAP is limited to patients currently residing in the US. Residency in the US must be for the duration of participation in the US EAP. Drug may not be shipped outside the US.

Conditions

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Dravet Syndrome

Interventions

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Fenfluramine Hydrochloride

ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD.

Intervention Type DRUG

Other Intervention Names

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ZX008

Eligibility Criteria

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Inclusion Criteria

* Patient is male or female, age 2 years and older, inclusive as of Study Day 1.
* Patient is diagnosed with Dravet syndrome.
* Patient is experiencing convulsive seizures which are not controlled by current AEDs.
* Patient is receiving at least one AED and will remain on at least one AED for the duration of treatment.
* Patient has been approved for inclusion by Zogenix.

Exclusion Criteria

* Patient requires or starts using an unacceptable or contraindicated concomitant medication.
* Patient has valvulopathy.
* Patient is at risk for pulmonary hypertension.
* Patient exclusion will be at the sole discretion of the Sponsor.
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Zogenix, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

References

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Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.

Reference Type DERIVED
PMID: 34768178 (View on PubMed)

Other Identifiers

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ZX008-1800

Identifier Type: -

Identifier Source: org_study_id

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