Infantile SpasmsPhase 3NCT02954887
Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
A Phase 3 study testing GWP42003-P in people with Infantile Spasms, run by Jazz Pharmaceuticals at 7 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 24 months
- Conditions the study is for
- Infantile Spasms
- What is being tested?
- GWP42003-P
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 12 May 2017to 13 Jun 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.
Who cannot join
- 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.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States5 sites
Nationwide Children's Hospital
Columbus, Ohio, 43205
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
Valley Health Clinical Research
Winchester, Virginia, 22601
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Poland2 sites
Uniwersyteckie Centrum Kliniczne
Gdansk
Centrum Medyczne POMOC
Lodz
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants With Infantile Spasms Following an Initial Open-label Pilot Study
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.
- Conditions
- Infantile Spasms
- Being tested
- GWP42003-P
- Keywords
- GWP42003-P, Cannabidiol
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GWP42003-PDrug
Also called CBD, Cannabidiol
Clear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: GWP42003-P |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From signing of informed consent up to Day 417
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Measured: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From signing of informed consent up to Day 389
Secondary outcomes (17)
Number of Participants Free of Clinical Spasms
Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants Free of Clinical Spasms
Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Number of Participants With a Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants With a Resolution of Hypsarrhythmia
Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Number of Participants Experiencing Spasms and Seizures by Subtype
Caregivers recorded the participant's spasms and seizures by category in a daily diary. Subtypes of spasms and seizure included, clonic, tonic-clonic, myoclonic, focal, and absence.
Measured: Days 19, 29, 127, 211, 295, and 379
Caregiver Global Impression of Change (CGIC)
The CGIC is a single-question assessment completed by the caregiver. The question assessed the status of the participant's condition since treatment start. The caregiver provided a rating on a 7-point scale: 1, very much improved; 2, much Improved; 3, slightly improved; 4, no change; 5, slightly worse; 6, much worse; 7, very much worse.
Measured: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Physician Global Impression of Change (PGIC)
The PGIC is a single-question assessment completed by the investigator. The question assessed the status of the participant's condition since treatment start. The investigator provided a rating on a 7-point scale: 1, very much improved; 2, much Improved; 3, slightly improved; 4, no change; 5, slightly worse; 6, much worse; 7, very much worse.
Measured: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Number of Responders
A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Percentage of Responders
A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Test for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Measured: Days 29, 43, 127, 211, 295, and 379
Change From Baseline in Height
A positive change indicates an increase in the average participant's height. A negative change indicates a decrease in the average participant's height. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Measured: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Body Weight.
A positive change indicates an increase in the average participant's weight. A negative change indicates a decrease in the average participant's weight. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Measured: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Head Circumference
A positive change indicates an increase in the average participant's head circumference. A negative change indicates a decrease in the average participant's head circumference. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Measured: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
The Vineland-II scores were assessed by the participant's caregiver. Caregivers were asked to score questions in the following categories: the participant's communication, daily living, physical activity, problem behaviors, and social skills and relationships. Scoring was slightly different for each section, but generally ranged from "usually" (2) to "never" (0). The total score is calculated as the sum of standard scores from the domains and converted into the adaptive behavior composite score (ranging from 20 to 160). Higher scores represent greater levels of functioning, and lower scores represent lower levels of functioning. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Measured: Baseline (Day 1 of Pilot Study); Day 211, Day 379
Number of Participants With Relapse of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Measured: Day 16 to Day 379
Percentage of Participants With Relapse of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Measured: Day 16 to Day 379
Average Time to Cessation of Spasms
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Measured: Day 1 to Day 379
Average Time to Relapse
Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Measured: Day 16 to Day 379
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2016 (estimated)
- Study start12 May 2017
- Primary completion13 Jun 2019
- Study completion13 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Infantile SpasmsStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02954887
- Sponsor's protocol ID
- GWEP15100 Open-label Extension
- Other IDs
- 2015-004904-50, 2015
- Status last verified
- Aug 2022
- First submitted
- 2 Nov 2016
- Last updated
- 2 Sep 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants With Infantile Spasms Following an Initial Open-label Pilot Study
- ClinicalTrials.gov ID
- NCT02954887
- Acronym / sponsor ID
- GWEP15100 Open-label Extension
- Conditions
- Infantile Spasms
- Keywords
- GWP42003-P, Cannabidiol
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 12 May 2017 (actual)
- Primary completion
- 13 Jun 2019 (actual)
- Study completion
- 13 Jun 2019 (actual)
- First posted
- 4 Nov 2016 (estimated)
- Results
- Posted 23 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GWP42003-P | Experimental | Drug: GWP42003-PAdministered 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. |
Who may qualify
- Ages
- 1 month to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Participant is diagnosed with IS and has failed to respond adequately following treatment with 1 or more approved IS therapies.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GWP42003-PAlso: CBD, CannabidiolClear, colorless to yellow solution containing cannabidiol dissolved in the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring. | Drug | GWP42003-P |
Primary outcome
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
Time frame: From signing of informed consent up to Day 417
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.
Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Number of Participants With Any Clinically Relevant Urinalysis Parameter Value
Time frame: Days 19, 29, 43, 71, 127, 211, 295, 379, and 389
Clinical relevance was determined by the investigator.
Number of Participants With Clinically Significant Electrocardiogram Findings
Time frame: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
Number of Participants With Clinically Significant Vital Sign Findings
Time frame: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
Number of Participants With Clinically Significant Physical Examination Findings
Time frame: From signing of informed consent up to Day 389
Clinical significance was determined by the investigator.
17 secondary outcomes
Number of Participants Free of Clinical Spasms
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants Free of Clinical Spasms
Time frame: Days 29, 43, 127, 211, 295, and 379
Number of Participants With a Resolution of Hypsarrhythmia
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Participants With a Resolution of Hypsarrhythmia
Time frame: Days 29, 43, 127, 211, 295, and 379
Number of Participants Experiencing Spasms and Seizures by Subtype
Time frame: Days 19, 29, 127, 211, 295, and 379
Caregiver Global Impression of Change (CGIC)
Time frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Physician Global Impression of Change (PGIC)
Time frame: Baseline; Days 29, 43, 71, 127, 211, 295, and 379
Number of Responders
Time frame: Days 29, 43, 127, 211, 295, and 379
Percentage of Responders
Time frame: Days 29, 43, 127, 211, 295, and 379
Change From Baseline in Height
Time frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Body Weight.
Time frame: Baseline (Day 1 of Pilot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Head Circumference
Time frame: Baseline (Day 1 of PIlot Study); Days 29, 43, 71, 127, 211, 295, 379, and 389
Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Score
Time frame: Baseline (Day 1 of Pilot Study); Day 211, Day 379
Number of Participants With Relapse of Spasms
Time frame: Day 16 to Day 379
Percentage of Participants With Relapse of Spasms
Time frame: Day 16 to Day 379
Average Time to Cessation of Spasms
Time frame: Day 1 to Day 379
Average Time to Relapse
Time frame: Day 16 to Day 379
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Infantile Spasms
Not yet recruitingObservational study
IBIS - Investigating Reliability of BIS and SEDLINE Monitoring in Children With Developmental and Epileptic Encephalopathies (DEE).
Epileptic Encephalopathy, Early Infantile Epileptic Encephalopathy
Active, not recruitingPhase 3
A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.
CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure
RecruitingPhase 2
Treatment of Refractory Infantile Spasms With Fenfluramine
Infantile Spasm
RecruitingObservational study
Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents
Epilepsy, West Syndrome
RecruitingPhase 2
A Study to Prevent Infantile Spasms Relapse
Infantile Spasms, Infantile Epileptic Spasms Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GWP42003-P in people with Infantile Spasms, run by Jazz Pharmaceuticals at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Jul 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) (time frame: From signing of informed consent up to Day 417). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2022.