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

NCT03421496 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: INS011-16-082
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 5

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).

Conditions

Infantile Spasm

Keywords

Infantile spasm; Vigabatrin; Cannabidiol oral solution

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Percentage of Participants With Resolution of Infantile Spasms

  Up to Day 15

• Percentage of Participants With Resolution of Hypsarrhythmia

  Up to Day 15

• Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I)

  Day 15

• Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15

  Up to Day 15

• Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period

  Up to Day 75

Study Arms (2)

CBD with Vigabatrin

EXPERIMENTAL

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 with Vigabatrin

PLACEBO COMPARATOR

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

Cannabidiol Oral Solution DRUG

An oral solution containing pharmaceutical grade cannabidiol (nonplant-based).

CBD with Vigabatrin

Placebo DRUG

Matching oral solution

Placebo with Vigabatrin

Vigabatrin DRUG

Powder suspension

Also known as: Sabril

CBD with Vigabatrin; Placebo with Vigabatrin

Eligibility Criteria

• Age: 1 Month - 24 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Radius Pharmaceuticals, Inc.: lead

Study Sites (5)

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Beaumont Children's Hospital

Royal Oak, Michigan, 48073, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Institute for Research and Innovation | MultiCare Health System

Tacoma, Washington, 98405, United States

Location

MeSH Terms

Conditions

Spasms, Infantile

Interventions

Cannabidiol; Vigabatrin

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals; gamma-Aminobutyric Acid; Aminobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Amino Acids; Amino Acids, Peptides, and Proteins

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.

Results Point of Contact

• Title: Bruce Mitlak, MD; Head of Discovery Science and Chief Medical Officer
• Organization: Radius Health

bmitlak@radiuspharm.com

617-444-1943

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2018
• First Posted: February 5, 2018
• Study Start: September 5, 2018
• Primary Completion: May 29, 2019
• Study Completion: May 29, 2019
• Last Updated: June 7, 2023
• Results First Posted: June 7, 2023

Record last verified: 2023-05

Locations

US (5)