Clinical trial NCT03421496: A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Infantile Spasm
Keywords: Infantile spasm
Vigabatrin
Cannabidiol oral solution
Lead sponsor: Radius Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2018-02-05
Enrollment: 2
Sex: ALL
Minimum age (years): 1
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS).

Detailed description: This was a randomized, double-blind, placebo-controlled, parallel-group study in which participants were randomized in a 1:1 ratio to 1 of 2 treatment groups. During the Initial Treatment Period, participants received either vigabatrin plus CBD or vigabatrin plus matching placebo and were dosed approximately every 12 hours, with a meal. This study was comprised of five periods: Screening, Initial Treatment, Extended Treatment, Taper, and Follow up Periods, with a maximum duration of approximately 140 days.

Eligibility 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, confirmed by video-EEG (including at least one cluster of electroclinical spasms \[≥3 in any 10-minute epoch\] and hypsarrythmia) obtained during the Screening Period and read by a 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).
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. Participant is diagnosed or suspected of having tuberous sclerosis.
6. Participant 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. Participant currently on any disallowed CYP3A4-related medication (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 participant eligible if he or she judges the laboratory values to be not clinically significant.

Primary outcomes:
measure: Percentage of Participants Considered Complete Responders
timeFrame: Up to Day 15
Secondary outcomes:
measure: Percentage of Participants With Resolution of Infantile Spasms
timeFrame: Up to Day 15
measure: Percentage of Participants With Resolution of Hypsarrhythmia
timeFrame: Up to Day 15
measure: Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)
timeFrame: Day 15
measure: Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15
timeFrame: Up to Day 15
measure: Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period
timeFrame: Up to Day 75
measure: Time to Relapse During the Extended Treatment Period
timeFrame: Up to Day 75
Other outcomes:
Locations:
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Royal Oak
country: United States
facility: Beaumont Children's Hospital
state: Michigan
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Tacoma
country: United States
facility: Institute for Research and Innovation | MultiCare Health System
state: Washington
Study officials:
References: