Dravet SyndromeNCT03780127
ZX008 Expanded Access Protocol
A study testing Fenfluramine Hydrochloride in people with Dravet Syndrome, run by Zogenix, Inc..
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Dravet Syndrome
- What is being tested?
- Fenfluramine Hydrochloride
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this study
Official title: ZX008 Expanded Access Protocol - Dravet Syndrome Treatment Plan
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.
Read the detailed description
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
- Dravet Syndrome
- Being tested
- Fenfluramine Hydrochloride
- Medical subject headings
- Epilepsies, Myoclonic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fenfluramine HydrochlorideDrug
Also called ZX008
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Zogenix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03780127
- Sponsor's protocol ID
- ZX008-1800
- Status last verified
- Oct 2023
- First submitted
- 14 Dec 2018
- Last updated
- 24 Oct 2023
- Results posted
- No
Publications
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.
PubMed 34768178
Study at a glance
From the registry record, last updated by the sponsor on 24 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ZX008 Expanded Access Protocol - Dravet Syndrome Treatment Plan
- ClinicalTrials.gov ID
- NCT03780127
- Acronym / sponsor ID
- ZX008-1800
- Conditions
- Dravet Syndrome
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Zogenix, Inc.Industry (pharma, biotech, devices)
- First posted
- 19 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fenfluramine HydrochlorideAlso: ZX008ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Oct 2023.
- Derived from this study: PubMed 34768178Cross 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Fenfluramine Hydrochloride in people with Dravet Syndrome, run by Zogenix, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03780127, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Oct 2023; AzTrials last refreshed its copy on 28 Oct 2026.