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Spasms, InfantilePhase 2NCT02551731

Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

A Phase 2 study testing Cannabidiol Oral Solution in people with Spasms, Infantile, run by INSYS Therapeutics Inc at 4 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS. Read more about this study

Who is it for?
Ages 6 to 36 months
Conditions the study is for
Spasms, Infantile
What is being tested?
Cannabidiol Oral Solution
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
4 sitesUnited States
Study dates
27 Jan 2016to 6 Sep 2016
Phase
Phase 2
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 6 to 36 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Meets protocol-specified criteria for qualification, including infantile spasms
  • Parent(s)/caregiver(s) fully comprehend and sign the informed consent form, understand all study procedures, and can communicate satisfactorily with the Investigator and study coordinator.

Who cannot join

  • History or current use of over-the-counter medications, dietary supplements, or drugs outside protocol-specified parameters
  • Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:
  • the safety or well-being of the participant or study staff
  • the analysis of results
  • During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
  • any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or marijuana)
  • any other investigational drug or investigational device

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Mattel Children's Hospital at UCLA

    Los Angeles, California, 90095

  • Miami Children's Hospital

    Miami, Florida, 33155

  • Beaumont Health System

    Royal Oak, Michigan, 48073

  • University of California - San Francisco

    San Francisco, California, 94143

Other trials in United States

About this study

Official title: A Phase 2 Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the Treatment of Refractory Infantile Spasms

Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS. This need is even more important for infants and toddlers still sick after being treated with medicine that is already available.

This is a multi-center study to evaluate the efficacy and safety of Cannabidiol Oral Solution (CBD) in the treatment of children aged 6 months through 36 months with a diagnosis of infantile spasms who have not responded to first line therapies.

The overall study duration is expected to be 64 weeks for those subjects who respond to CBD treatment. The maximum possible study duration for each patient is approximately 64 weeks, however a subject will be deemed to have completed the study after 58 weeks.

Read the detailed description

A protocol amendment in May 2016 created two parts to this trial: Part A (the extended treatment period) and Part B (the safety treatment period), whose objectives are as follows:

Primary Part A: To evaluate the efficacy of Cannabidiol Oral Solution in treating refractory infantile spasms (IS).

Secondary:

Part A:

  • To evaluate the safety of Cannabidiol Oral Solution in treating refractory infantile spasms.

Part B:

  • To assess the long-term safety of Cannabidiol Oral Solution as an adjunctive treatment for subjects with Infantile Spasms (IS)
  • To establish the continued efficacy of Cannabidiol Oral Solution in maintaining seizure control in subjects with IS
  • To assess the global status of subjects taking Cannabidiol Oral Solution for an extended period of time determined by various qualitative assessments
  • To monitor for changes in plasma levels of Cannabidiol Oral Solution during long-term treatment of subjects with IS
Being tested
Cannabidiol Oral Solution
Medical subject headings
Spasms, Infantile

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cannabidiol Oral SolutionDrug

    20 or 40 mg/kg/day BID

Study groups in NCT02551731
Study groupRoleWhat they receive
Cannabidiol Oral Solution: 20 or 40 mg/kg/day BIDExperimentalThe dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Receives: Drug: Cannabidiol Oral Solution

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14

    Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.

    Measured: Day 14

  2. Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)

    Measured: Up to Week 64

Secondary outcomes (12)

  1. Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14

    Measured: Day 14

  2. Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14

    Measured: Day 14

  3. Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14

    Measured: Baseline, Day 14

  4. Part A: Parent Impression of Efficacy and Tolerability of Study Drug

    Parent impression of efficacy and tolerability, as measured by Clinical Global Impression-Global Improvement Scale (CGI-I), was summarized by visit and status of response (Complete/Partial and No Response) at Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. The CGI-I was also analyzed in a continuous scale, as follows: 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, and 7 = Very much worse

    Measured: Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.

  5. Part A: Percentage of Participants With a Partial Response to Treatment

    Partial response was defined as a substantive change in background EEG or reduction in spasms on video EEG obtained at Day 14.

    Measured: Day 14

  6. Part A: Percentage of Complete Responders With Relapse

    Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.

    Measured: Day 14

  7. Part A: Time to Complete Responder Relapse

    Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.

    Measured: Day 14

  8. Part B: Parent Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in Clinical Global Impression of Improvement Assessment (CGI-I), Responses at Every Visit Throughout Part B

    Measured: Up to Week 64

  9. Part B: Investigator Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in CGI-I Responses at Every Visit Throughout Part B

    Measured: Up to Week 64

  10. Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B.

    Measured: Up to Week 64

  11. Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG

    Measured: Up to Week 64

  12. Part B: Time to Relapse as Confirmed by Video-EEG

    Measured: Up to Week 64

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Sep 2015 (estimated)
  2. Study start27 Jan 2016
  3. Primary completion6 Sep 2016
  4. Study completion6 Sep 2016

Why it stopped: Sponsor elected not to continue with study

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02551731
Sponsor's protocol ID
INS011-15-054
Status last verified
Aug 2018
First submitted
14 Sep 2015
Last updated
19 Sep 2018
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cannabidiol Oral Solution in people with Spasms, Infantile, run by INSYS Therapeutics Inc at 4 sites in United States.

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 2 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: stopped early, as of the sponsor's last update on 19 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 19 Sep 2018). 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?

Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14 (time frame: Day 14). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02551731.

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 19 Sep 2018.

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