NCT02954887

Brief Summary

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 2017

Geographic Reach
2 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 2, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 4, 2016

Completed
6 months until next milestone

Study Start

First participant enrolled

May 12, 2017

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 13, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 13, 2019

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

July 23, 2020

Completed
Last Updated

September 2, 2022

Status Verified

August 1, 2022

Enrollment Period

2.1 years

First QC Date

November 2, 2016

Results QC Date

June 10, 2020

Last Update Submit

August 31, 2022

Conditions

Infantile Spasms

Keywords

GWP42003-PCannabidiol

Outcome Measures

Primary Outcomes (7)

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

    TEAEs were collected in members of the Safety Population, comprised of all participants who received at least 1 dose of GWP42003-P. 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 417

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

    Days 19, 29, 43, 71, 127, 211, 295, 379, and 389

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

    Days 19, 29, 43, 71, 127, 211, 295, 379, and 389

  • Number of Participants With Any Clinically Relevant Urinalysis Parameter Value

    Clinical relevance was determined by the investigator.

    Days 19, 29, 43, 71, 127, 211, 295, 379, and 389

  • Number of Participants With Clinically Significant Electrocardiogram Findings

    Clinical significance was determined by the investigator.

    From signing of informed consent up to Day 389

  • Number of Participants With Clinically Significant Vital Sign Findings

    Clinical significance was determined by the investigator.

    From signing of informed consent up to Day 389

  • Number of Participants With Clinically Significant Physical Examination Findings

    Clinical significance was determined by the investigator.

    From signing of informed consent up to Day 389

Secondary Outcomes (17)

  • Number of Participants Free of Clinical Spasms

    Days 29, 43, 127, 211, 295, and 379

  • Percentage of Participants Free of Clinical Spasms

    Days 29, 43, 127, 211, 295, and 379

  • Number of Participants With a Resolution of Hypsarrhythmia

    Days 29, 43, 127, 211, 295, and 379

  • Percentage of Participants With a Resolution of Hypsarrhythmia

    Days 29, 43, 127, 211, 295, and 379

  • Number of Participants Experiencing Spasms and Seizures by Subtype

    Days 19, 29, 127, 211, 295, and 379

  • +12 more secondary outcomes

Study Arms (1)

GWP42003-P

EXPERIMENTAL

Administered orally, up to the target dose recommended by the data safety monitoring committee. Participants continue at the target dose, or the highest tolerated dose up to the target dose, for a total of 12 months' treatment.

Drug: GWP42003-P

Interventions

GWP42003-PDRUG

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

Age1 Month - 24 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.

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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

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

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, GeneralizedEpilepsyBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEpileptic Syndromes

Intervention Hierarchy (Ancestors)

CannabinoidsTerpenesHydrocarbonsOrganic 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; 18778862810

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 2, 2016

First Posted

November 4, 2016

Study Start

May 12, 2017

Primary Completion

June 13, 2019

Study Completion

June 13, 2019

Last Updated

September 2, 2022

Results First Posted

July 23, 2020

Record last verified: 2022-08

Locations

PL(2)US(5)