A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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. |
Drug: Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
Drug: Vigabatrin
Powder suspension
Other Names:
|
| 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. |
Drug: Placebo
Matching oral solution
Drug: Vigabatrin
Powder suspension
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Percentage of Participants Considered Complete Responders [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.
Secondary Outcome Measures
- Percentage of Participants with Resolution of Infantile Spasms [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 [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 [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 [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 [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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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.
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Clinical diagnosis of Infantile Spasms and hypsarrythmia, confirmed by a 9-hour video-EEG obtained during screening Period and read by the central reader.
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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).
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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:
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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.
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Known or suspected allergy to cannabidiol.
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History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
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Use of any cannabidiol/cannabis product within 30 days of study entry.
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Patient is diagnosed or suspected of having tuberous sclerosis.
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Patient has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
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Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
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Patient currently on any disallowed CYP3A4-related medication listed in Appendix 1 (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
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Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nicklaus Children's Hospital | Miami | Florida | United States | 33155 |
| 2 | Beaumont Children's Hospital | Royal Oak | Michigan | United States | 48073 |
| 3 | Akron Children's Hospital | Akron | Ohio | United States | 44308 |
| 4 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
| 5 | Institute for Research and Innovation | MultiCare Health System | Tacoma | Washington | United States | 98405 |
Sponsors and Collaborators
- Benuvia Therapeutics Inc.
Investigators
- Study Director: Ahmed Elkashef, MD, INSYS Therapeutics Inc
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- INS011-16-082