Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)

NCT02953548 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: GWEP15100 Pilot Phase2015-004904-50
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 8

Brief Summary

This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The pilot phase only will be described in this record. 2 cohorts of 5 participants will be enrolled sequentially. All participants will receive GWP42003-P.

Conditions

Infantile Spasms

Keywords

GWP42003-P; Cannabidiol

Outcome Measures

Primary Outcomes (7)

• Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)

  TEAEs are defined as all adverse events not present prior to the first investigational medicinal product (IMP) or placebo administration or any event already present that worsened in severity or frequency following IMP.

  From signing of informed consent up to Day 15

• Number of Participants With Any Low or High Hematology Laboratory Parameter Value

  Day 4 and Day 15

• Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value

  Day 4 and Day 15

• Number of Participant With Any Clinically Relevant Urinalysis Parameter Value

  Clinical relevance was determined by the investigator.

  Day 4 and Day 15

• Number of Participants With Clinically Significant Electrocardiogram Findings

  Clinical significance was determined by the investigator.

  From signing of informed consent up to Day 15

• Number of Participants With Clinically Significant Physical Examination Findings

  Clinical significance was determined by the investigator.

  From signing of informed consent up to Day 15

• Number of Participants With Clinically Significant Vital Sign Findings

  Clinical significance was determined by the investigator.

  From signing of informed consent up to Day 15

Secondary Outcomes (10)

• Number of Participants Free of Clinical Spasms

  Day 15

• Percentage of Participants Free of Clinical Spasms

  Day 15

• Number of Participants With Resolution of Hypsarrhythmia

  Day 15

• Percentage of Participants With Resolution of Hypsarrhythmia

  Day 15

• Number of Participants Experiencing Spasms and Seizures by Subtype

  Day 4 and Day 15

Study Arms (1)

GWP42003-P

EXPERIMENTAL

Administered orally, titrating to a target dose of 40 mg/kg/day. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for the remainder of the 2-week treatment period.

Drug: GWP42003-P

Interventions

GWP42003-P DRUG

Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.

Also known as: CBD, Cannabidiol

GWP42003-P

Eligibility Criteria

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

You may qualify if:

• Participant is aged 6- 24 months (inclusive) in the first cohort or aged 1-24 months (inclusive) in the second cohort, at the time of consent.
• Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.
• To be considered hypsarrhythmia, as defined for use in the study, the electroencephalography (EEG) background must be slowed and have multifocal spikes. In addition, it must be either high voltage (above 300 µV) or have electrodecrement/discontinuity.

You may not qualify if:

• Participant is currently taking or has taken clobazam or any mammalian target of rapamycin (mTOR) inhibitor within the 2 weeks prior to the screening visit.
• Participant has a QT interval, corrected for heart rate with Bazett's formula (QTcB), of 460 msec or greater on ECG.
• Participant's caregiver is currently giving or has given recreational or medicinal cannabis, or synthetic cannabinoid-based medications, within the 1 month prior to the screening visit.
• Participant's caregiver is unwilling to abstain from giving the participant (including the participant's mother abstaining themselves, if breastfeeding)recreational or medicinal cannabis, or synthetic cannabinoid-based medications (other than the study drug) during the trial.
• Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study drug, such as sesame oil.
• Participant has significantly impaired hepatic function at the screening visit.
• Participant has received an investigational medicinal product as part of a clinical trial within a minimum of 5 half-lives prior to the screening visit.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (8)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Le Bonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

The Childrens Hospital of San Antonio

San Antonio, Texas, 78207, United States

Location

Valley Health Clinical Research

Winchester, Virginia, 22601, United States

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland

Location

Centrum Medyczne POMOC

Lodz, Poland

Location

MeSH Terms

Conditions

Spasms, Infantile

Interventions

Cannabidiol

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

Limitations and Caveats

This study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment, and the Pivotal Phase was not initiated. Participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.

Results Point of Contact

• Title: Medical Enquires
• Organization: GW Research Ltd

medinfo@gwpharm.com, medinfo@greenwichbiosciences.com

+44 01223 238170; 1877886281

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Masking Details: Open-label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 1, 2016
• First Posted: November 2, 2016
• Study Start: April 24, 2017
• Primary Completion: May 7, 2018
• Study Completion: May 7, 2018
• Last Updated: September 2, 2022
• Results First Posted: July 23, 2020

Record last verified: 2022-08

Locations

PL (2); US (6)