Dravet SyndromePhase 1NCT03467113
A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol
A Phase 1 study testing ZX008 0.2 to 0.8 mg/kg/day and Cannabidiol in people with Dravet Syndrome and Lennox Gastaut Syndrome, run by Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. at 2 sites in United States.
Trial summary
This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD). Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Dravet Syndrome, Lennox Gastaut Syndrome
- What is being tested?
- ZX008 0.2 to 0.8 mg/kg/day and Cannabidiol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesUnited States
- Study dates
- 19 Jan 2018to 24 Jun 2020
- Phase
- Phase 1
- 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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject has a clinical diagnosis of DS or LGS, where convulsive seizures (generalized tonic clonic seizures, tonic seizures, atonic seizures, tonic/atonic seizures, focal seizures with clear observable motor signs) are not completely controlled by current AEDs.
- All medications or interventions for epilepsy (including ketogenic diet [KD] and vagal nerve stimulation [VNS]) must be stable for at least 4 weeks prior to first ZX008 dose and are expected to remain stable throughout the study.
- Subjects must be taking a stable dose of CBD for at least 4 weeks prior to first ZX008 dose.
- Subject's source of CBD is expected to be consistent for at least 3 months during study participation.
Who cannot join
- Subject has current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
- Subject with current cardiac valvulopathy or pulmonary hypertension that the Investigator, parent/caregiver, Independent Cardiac Advisory Board, Independent Data and Safety Monitoring Committee, or sponsor deems clinically significant.
- Subject has a current or past history of glaucoma.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
PANDA Neurology/CIRCA
Atlanta, Georgia, 30328
MultiCare Institute for Research & Innovation
Tacoma, Washington, 98405
About this study
Official title: An Open-Label Trial to Assess the Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Combination With Cannabidiol, as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD).
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Being tested
- ZX008 0.2 to 0.8 mg/kg/day, Cannabidiol
- Keywords
- Dravet Syndrome, LGS
- Medical subject headings
- Epilepsies, Myoclonic, Lennox Gastaut Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZX008 0.2 to 0.8 mg/kg/dayDrug
ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.
CannabidiolDrug
Cannabidiol
| Study group | Role | What they receive |
|---|---|---|
| ZX008 0.2 to 0.8 mg/kg/day | Experimental | ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive open-label ZX008 (flexible dosing 0.2 mg/kg/day to 0.8 mg/kg/day) Receives: Drug: ZX008 0.2 to 0.8 mg/kg/day; Drug: Cannabidiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Measured: Baseline to Day 365
Change in Heart Rate
Change in resting heart rate using standard measures
Measured: Baseline to Day 365
Change in blood pressure
Change in resting blood pressure using standard measures
Measured: Baseline to Day 365
Change in temperature
Change in resting temperature using standard measures
Measured: Baseline to Day 365
Change in respiratory rate
Change in resting respiratory rate using standard measures
Measured: Baseline to Day 365
Changes in heart rhythm
Changes in heart beat as measured with 12-lead electrocardiogram
Measured: Baseline to Day 365
Changes in heart valve function
Changes in heart valves as measured with standard echocardiogram
Measured: Baseline to Day 365
Changes in treatment-emergent body weight and height
Changes in body weight and height by report of BMI in kg/m^2
Measured: Baseline to Day 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2018
- Study start19 Jan 2018
- Primary completion24 Jun 2020
- Study completion24 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03467113
- Sponsor's protocol ID
- ZX008-1602
- Status last verified
- Nov 2023
- First submitted
- 30 Jan 2018
- Last updated
- 1 Dec 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Trial to Assess the Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Combination With Cannabidiol, as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome or Lennox-Gastaut Syndrome
- ClinicalTrials.gov ID
- NCT03467113
- Acronym / sponsor ID
- ZX008-1602
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome
- Keywords
- Dravet Syndrome, LGS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Jan 2018 (actual)
- Primary completion
- 24 Jun 2020 (actual)
- Study completion
- 24 Jun 2020 (actual)
- First posted
- 15 Mar 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.
This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ZX008 0.2 to 0.8 mg/kg/day | Experimental | Drug: ZX008 0.2 to 0.8 mg/kg/day, Drug: CannabidiolZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive open-label ZX008 (flexible dosing 0.2 mg/kg/day to 0.8 mg/kg/day) |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Subject has a clinical diagnosis of DS or LGS, where convulsive seizures (generalized tonic clonic seizures, tonic seizures, atonic seizures, tonic/atonic seizures, focal seizures with clear observable motor signs) are not completely controlled by current AEDs.
- All medications or interventions for epilepsy (including ketogenic diet [KD] and vagal nerve stimulation [VNS]) must be stable for at least 4 weeks prior to first ZX008 dose and are expected to remain stable throughout the study.
- Subjects must be taking a stable dose of CBD for at least 4 weeks prior to first ZX008 dose.
- Subject's source of CBD is expected to be consistent for at least 3 months during study participation.
Key exclusion criteria (3 listed)
- Subject has current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
- Subject with current cardiac valvulopathy or pulmonary hypertension that the Investigator, parent/caregiver, Independent Cardiac Advisory Board, Independent Data and Safety Monitoring Committee, or sponsor deems clinically significant.
- Subject has a current or past history of glaucoma.
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 |
|---|---|---|
| ZX008 0.2 to 0.8 mg/kg/dayZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free. | Drug | ZX008 0.2 to 0.8 mg/kg/day |
| CannabidiolCannabidiol | Drug | ZX008 0.2 to 0.8 mg/kg/day |
Primary outcome
Adverse Events
Time frame: Baseline to Day 365
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Change in Heart Rate
Time frame: Baseline to Day 365
Change in resting heart rate using standard measures
Change in blood pressure
Time frame: Baseline to Day 365
Change in resting blood pressure using standard measures
Change in temperature
Time frame: Baseline to Day 365
Change in resting temperature using standard measures
Change in respiratory rate
Time frame: Baseline to Day 365
Change in resting respiratory rate using standard measures
Changes in heart rhythm
Time frame: Baseline to Day 365
Changes in heart beat as measured with 12-lead electrocardiogram
Changes in heart valve function
Time frame: Baseline to Day 365
Changes in heart valves as measured with standard echocardiogram
Changes in treatment-emergent body weight and height
Time frame: Baseline to Day 365
Changes in body weight and height by report of BMI in kg/m^2
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2023.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ZX008 0.2 to 0.8 mg/kg/day and Cannabidiol in people with Dravet Syndrome and Lennox Gastaut Syndrome, run by Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. at 2 sites in United States.
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 4 inclusion and 3 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: finished, as of the sponsor's last update on 1 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Adverse Events (time frame: Baseline to Day 365). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 1 Dec 2023.