NCT03467113

Brief 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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2018

Typical duration for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 19, 2018

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

January 30, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

March 15, 2018

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 24, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 24, 2020

Completed
Last Updated

December 1, 2023

Status Verified

November 1, 2023

Enrollment Period

2.4 years

First QC Date

January 30, 2018

Last Update Submit

November 30, 2023

Conditions

Dravet SyndromeLennox Gastaut Syndrome

Keywords

Dravet SyndromeLGS

Outcome Measures

Primary Outcomes (8)

  • Adverse Events

    Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal

    Baseline to Day 365

  • Change in Heart Rate

    Change in resting heart rate using standard measures

    Baseline to Day 365

  • Change in blood pressure

    Change in resting blood pressure using standard measures

    Baseline to Day 365

  • Change in temperature

    Change in resting temperature using standard measures

    Baseline to Day 365

  • Change in respiratory rate

    Change in resting respiratory rate using standard measures

    Baseline to Day 365

  • Changes in heart rhythm

    Changes in heart beat as measured with 12-lead electrocardiogram

    Baseline to Day 365

  • Changes in heart valve function

    Changes in heart valves as measured with standard echocardiogram

    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

    Baseline to Day 365

Study Arms (1)

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)

Drug: ZX008 0.2 to 0.8 mg/kg/dayDrug: Cannabidiol

Interventions

ZX008 0.2 to 0.8 mg/kg/dayDRUG

ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.

ZX008 0.2 to 0.8 mg/kg/day
CannabidiolDRUG

Cannabidiol

ZX008 0.2 to 0.8 mg/kg/day

Eligibility Criteria

Age2 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

PANDA Neurology/CIRCA

Atlanta, Georgia, 30328, United States

Location

MultiCare Institute for Research & Innovation

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Epilepsies, MyoclonicLennox Gastaut Syndrome

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Epilepsy, GeneralizedEpilepsyBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEpileptic SyndromesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

CannabinoidsTerpenesHydrocarbonsOrganic Chemicals

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 30, 2018

First Posted

March 15, 2018

Study Start

January 19, 2018

Primary Completion

June 24, 2020

Study Completion

June 24, 2020

Last Updated

December 1, 2023

Record last verified: 2023-11

Locations

US(2)