TerminatedNCT03421496Updated Jun 7, 2023Results posted

A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms

A Phase 3 interventional study of Cannabidiol Oral Solution and Placebo in Infantile Spasm, sponsored by Radius Pharmaceuticals, Inc.. Terminated at 5 sites in United States. Open to participants aged 1 Month to 24 Months. Per ClinicalTrials.gov, last updated 2023-06-07.

Sponsored by Radius Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Why this study was terminated

The study was terminated due to slow enrollment and failure to identify adequate patients that met entry criteria.

Phase: Phase 3
Study type: Interventional
Enrollment: 2
Allocation: Randomized
Ages: 1 Month to 24 Months
Sex: All

Study 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).

Read the 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.

Conditions studied

Infantile Spasm

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Keywords

Infantile spasm
Vigabatrin
Cannabidiol oral solution

Who can participate

Ages eligible: 1 Month to 24 Months
Sexes eligible: All
Accepts healthy volunteers: No

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.
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.
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).
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

Exclusion Criteria:

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.
Known or suspected allergy to cannabidiol.
History of an allergic reaction or a known or suspected sensitivity to any substance that is contained in the investigational product formulation.
Use of any cannabidiol/cannabis product within 30 days of study entry.
Participant is diagnosed or suspected of having tuberous sclerosis.
Participant has received treatment with either vigabatrin, ACTH, or high-dose steroids previously.
Previous or concomitant therapy with felbamate, clobazam, valproic acid, or the ketogenic diet.
Participant currently on any disallowed CYP3A4-related medication (phenytoin, fluvoxamine, carbamazepine, and St. John's Wort).
Previously received any investigational drug or device or investigational therapy within 30 days before Screening.
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.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 2 participants (actual)

Study arms

Experimental
CBD with Vigabatrin
Participants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food.
Drug: Cannabidiol Oral Solution · Drug: Vigabatrin
Placebo comparator
Placebo with Vigabatrin
Participants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food.
Drug: Placebo · Drug: Vigabatrin

Interventions

DrugCannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
DrugPlacebo
Matching oral solution
DrugVigabatrin
Powder suspension
Also known as: Sabril

What researchers measure

Primary outcomes

Percentage of Participants Considered Complete Responders
Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).
Time frame: Up to Day 15

Secondary outcomes

Percentage of Participants With Resolution of Infantile Spasms
Resolution of IS was assessed by 24-hour video-EEG.
Time frame: Up to Day 15
Percentage of Participants With Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was assessed by 24-hour video-EEG.
Time frame: Up to Day 15
Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)
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. Higher scores indicated worse condition.
Time frame: Day 15
Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15
Increase in spasm-free days will be determined by seizure diary entries.
Time frame: Up to Day 15
Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period
Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse.
Time frame: Up to Day 75
Time to Relapse During the Extended Treatment Period
Time frame: Up to Day 75

Results

Posted Jun 7, 2023

Limitations and caveats

This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.

Participant flow

Participant flow — Overall Study
Milestone	CBD With Vigabatrin	Placebo With Vigabatrin
Started	0	0
Completed	0	0
Not completed	0	0

Outcome measures

PrimaryPercentage of Participants Considered Complete Responders

Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).

Time frame:: Up to Day 15