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

Infantile Spasm Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03421496
• Other study ID #: INS011-16-082
• Status: Terminated
• Phase: Phase 3
• Start date: September 5, 2018
• Est. completion date: May 29, 2019

Study information

• Verified date: May 2023
• Source: Radius Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: May 29, 2019
• Est. primary completion date: May 29, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 24 Months

Eligibility

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.

Related Conditions & MeSH terms

• Infantile Spasm
• Muscle Cramp
• Spasm
• Spasms, Infantile

Intervention

Drug:
• Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).
• Placebo
Matching oral solution
• Vigabatrin
Powder suspension

Locations

Country	Name	City	State
United States	Akron Children's Hospital	Akron	Ohio
United States	Nicklaus Children's Hospital	Miami	Florida
United States	Oregon Health & Science University	Portland	Oregon
United States	Beaumont Children's Hospital	Royal Oak	Michigan
United States	Institute for Research and Innovation | MultiCare Health System	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Radius Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of Participants Considered Complete Responders	Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG).	Up to Day 15
Secondary	Percentage of Participants With Resolution of Infantile Spasms	Resolution of IS was assessed by 24-hour video-EEG.	Up to Day 15
Secondary	Percentage of Participants With Resolution of Hypsarrhythmia	Resolution of hypsarrhythmia was assessed by 24-hour video-EEG.	Up to Day 15
Secondary	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.	Day 15
Secondary	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.	Up to Day 15
Secondary	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.	Up to Day 75
Secondary	Time to Relapse During the Extended Treatment Period		Up to Day 75