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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03421496
• Date of registration: 26/01/2018
• Prospective Registration: Yes
• Primary sponsor: Benuvia Therapeutics Inc.
• Public title: A Study to Assess the Efficacy, Safety, and Tolerability of Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
• Scientific title: 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
• Date of first enrolment: September 5, 2018
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03421496
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Ahmed Elkashef, MD
• Affiliation: INSYS Therapeutics Inc

Key inclusion & exclusion criteria

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.



Age minimum: 1 Month
Age maximum: 24 Months
Gender: All

Health Condition(s) or Problem(s) studied

Infantile Spasm

Intervention(s)

Drug: Cannabidiol Oral Solution

Drug: Vigabatrin

Drug: Placebo

Primary Outcome(s)

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)]

Secondary Outcome(s)

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)]

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)]

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)]

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)]

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]

Secondary ID(s)

INS011-16-082

Source(s) of Monetary Support

Please refer to primary and secondary sponsors