Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03421496
Recruitment Status : Active, not recruiting
First Posted : February 5, 2018
Last Update Posted : June 11, 2019
Sponsor:
Information provided by (Responsible Party):
INSYS Therapeutics Inc

Tracking Information
First Submitted Date  ICMJE January 26, 2018
First Posted Date  ICMJE February 5, 2018
Last Update Posted Date June 11, 2019
Actual Study Start Date  ICMJE September 5, 2018
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 27, 2018)
Percentage of Participants Considered Complete Responders [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG) at Visit 5.
Original Primary Outcome Measures  ICMJE
 (submitted: February 1, 2018)
Percentage of Participants Considered Complete Responders [ Time Frame: Visit 1 ( Days -14 to -1 of Screening Period) up to Visit 4 (Day 13 to Day 14 of Initial Treatment Period) ]
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).
Change History Complete list of historical versions of study NCT03421496 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 27, 2018)
  • Percentage of Participants with Resolution of Infantile Spasms [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period ]
    Resolution of infantile spasms will be assessed by 24-hour video-EEG on Visit 5.
  • Percentage of Participants with Resolution of Hypsarrhythmia [ Time Frame: Visit 1 (Day -14 to -1 of Screening Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
    Resolution of hypsarrhythmia will be assessed by 24-hour video-EEG on Visit 5.
  • Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I) at Visit 6 [ Time Frame: Visit 6 (Day 15 of Initial Treatment Period) ]
    Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse.
  • Increase in Number of Spasm-Free Days Between Day 1 and Day 15 [ Time Frame: Visit 2 (Day 1 of Initial Treatment Period) up through Visit 6 (Day 15 of Initial Treatment Period) ]
    Increase in spasm-free days will be determined by seizure diary entries.
  • Participants with Complete Response During Initial Treatment Period: Percentage of Participants Who Relapse During the Extended Treatment Period [ Time Frame: Visit 6 (Day 15 of Initial Treatment Period) up through Visit 9 (Week 13 of Extended Treatment Period) ]
    Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse, and time to relapse.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 1, 2018)
  • Participants with Complete Response During Initial Treatment Period: Percentage of Participants Who Relapse During the Extended Treatment Period [ Time Frame: Visit 5 (Day 14 of Initial Treatment Period) up to Visit 8 ( Week 13 of Extended Treatment Period) ]
    Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse, and time to relapse.
  • Percentage of Participants with Resolution of Infantile Spasms [ Time Frame: Visit 1 ( Days -14 to -1 of Screening Period) up to Visit 4 (Day 13 to Day 14 of Initial Treatment Period) ]
    Resolution of infantile spasms will be assessed by 24-hour video-EEG on Visit 4.
  • Percentage of Participants with Resolution of Hypsarrhythmia [ Time Frame: Visit 1 ( Days -14 to -1 of Screening Period) up to Visit 4 (Day 13 to Day 14 of Initial Treatment Period) ]
    Resolution of hypsarrhythmia will be assessed by 24-hour video-EEG on Visit 4.
  • Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I) at Visit 5 [ Time Frame: Visit 5 (Day 14 of Initial Treatment Period) ]
    Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse.
  • Percentage Reduction in Spasm-Free Days Between the Initial Treatment Period and Screening Period [ Time Frame: Visit 1 ( Day -14 to -1 of Screening Period) up to Visit 5 ( Day 14 of Initial Treatment Period) ]
    Reduction in spasm-free days will be determined by seizure diary entries.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
Official Title  ICMJE A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution as Adjunctive Therapy With Vigabatrin as Initial Therapy in Patients With Infantile Spasms
Brief Summary The primary objective of this study is to evaluate the efficacy of Cannabidiol Oral Solution as adjunctive therapy with vigabatrin as initial therapy in treating participants with Infantile Spasms. The secondary objectives for this study are to evaluate the continued efficacy of Cannabidiol Oral Solution after the 14-day treatment with vigabatrin or vigabatrin plus Cannabidiol Oral Solution is complete and to evaluate the safety and tolerability of Cannabidiol Oral Solution as adjunctive therapy with vigabatrin as initial therapy in treating participants with infantile spasms.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Infantile Spasm
Intervention  ICMJE
  • Drug: Cannabidiol Oral Solution
    An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
  • Drug: Placebo
    Matching oral solution
  • Drug: Vigabatrin
    Powder suspension
    Other Name: Sabril
Study Arms  ICMJE
  • Experimental: Cannabidiol Oral Solution (CBD)

    Cannabidiol Oral Solution, up to 40 milligrams per kilogram per day (mg/kg/day), participants will be dosed approximately every 12 hours with food.

    Participants will also be taking vigabatrin, up to 150 mg/kg/day, divided twice daily with food.

    Interventions:
    • Drug: Cannabidiol Oral Solution
    • Drug: Vigabatrin
  • Placebo Comparator: Placebo

    Matching CBD placebo, up to 40 mg/kg/day, participants will be dosed approximately every 12 hours with food.

    Participants will also be taking vigabatrin, up to 150 mg/kg/day, divided twice daily with food.

    Interventions:
    • Drug: Placebo
    • Drug: Vigabatrin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: February 1, 2018)
190
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Parent(s)/caregiver(s) fully comprehends and signs the informed consent form, understands all study procedures, and can communicate satisfactorily with the Investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
  2. Clinical diagnosis of Infantile Spasms and hypsarrythmia, confirmed by a 9-hour video-EEG obtained during screening Period and read by the central reader.
  3. General good health (defined as the absence of any clinically relevant abnormalities as determined by the Investigator) based on physical and neurological examinations, medical history, and clinical laboratory values completed during the Screening Visit (Visit 1).
  4. In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules.

Exclusion Criteria:

  1. Is considered by the investigator, for any reason (including, but not limited to, the risks described as precautions, warnings, and contraindications in the current version of the Investigator's Brochure for Cannabidiol Oral Solution) to be an unsuitable candidate to receive the study drug.
  2. Known or suspected allergy to cannabidiol.
  3. History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
  4. Use of any cannabidiol/cannabis product within 30 days of study entry.
  5. Patient is diagnosed or suspected of having tuberous sclerosis.
  6. Patient has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
  7. Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
  8. Patient currently on any disallowed CYP3A4-related medication listed in Appendix 1 (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
  9. Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
  10. Clinically significant abnormal laboratory values, including: liver function tests (LFTs) such as albumin, direct bilirubin, total bilirubin, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) ≥3 times the upper limit of normal (ULN). The investigator may deem the patient eligible if he or she judges the laboratory values to be not clinically significant.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 24 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03421496
Other Study ID Numbers  ICMJE INS011-16-082
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party INSYS Therapeutics Inc
Study Sponsor  ICMJE INSYS Therapeutics Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Ahmed Elkashef, MD INSYS Therapeutics Inc
PRS Account INSYS Therapeutics Inc
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP