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Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

Primary Purpose

Spasms, Infantile

Status

Terminated

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Cannabidiol Oral Solution

About this trial

This is an interventional treatment trial for Spasms, Infantile

Eligibility Criteria

6 Months - 36 Months (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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:
1. the safety or well-being of the participant or study staff
2. the analysis of results
During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
1. any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or marijuana)
2. any other investigational drug or investigational device

Sites / Locations

Mattel Children's Hospital at UCLA
University of California - San Francisco
Miami Children's Hospital
Beaumont Health System

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID

Arm Description

The 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.

Outcomes

Primary Outcome Measures

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.

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

Secondary Outcome Measures

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

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

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

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

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.

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.

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.

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

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

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

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

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

Full Information

NCT ID

NCT02551731

First Posted

September 14, 2015

Last Updated

August 21, 2018

Sponsor

INSYS Therapeutics Inc

1. Study Identification

Unique Protocol Identification Number

NCT02551731

Brief Title

Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2018

Overall Recruitment Status

Terminated

Why Stopped

Sponsor elected not to continue with study

Study Start Date

January 27, 2016 (Actual)

Primary Completion Date

September 6, 2016 (Actual)

Study Completion Date

September 6, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

INSYS Therapeutics Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief 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. 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Spasms, Infantile

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Cannabidiol Oral Solution

Intervention Description

20 or 40 mg/kg/day BID

Primary Outcome Measure Information:

Title

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

Description

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

Time Frame

Day 14

Title

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

Time Frame

Up to Week 64

Secondary Outcome Measure Information:

Title

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

Time Frame

Day 14

Title

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

Time Frame

Day 14

Title

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

Time Frame

Baseline, Day 14

Title

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

Description

Time Frame

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

Title

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

Description

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

Time Frame

Day 14

Title

Part A: Percentage of Complete Responders With Relapse

Description

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

Time Frame

Day 14

Title

Part A: Time to Complete Responder Relapse

Description

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

Time Frame

Day 14

Title

Time Frame

Up to Week 64

Title

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

Time Frame

Up to Week 64

Title

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

Time Frame

Up to Week 64

Title

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

Time Frame

Up to Week 64

Title

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

Time Frame

Up to Week 64

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

36 Months

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Neha Parikh

Organizational Affiliation

INSYS Therapeutics Inc

Official's Role

Study Director

Facility Information:

Facility Name

Mattel Children's Hospital at UCLA

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095

Country

United States

Facility Name

University of California - San Francisco

City

San Francisco

State/Province

California

ZIP/Postal Code

94143

Country

United States

Facility Name

Miami Children's Hospital

City

Miami

State/Province

Florida

ZIP/Postal Code

33155

Country

United States

Facility Name

Beaumont Health System

City

Royal Oak

State/Province

Michigan

ZIP/Postal Code

48073

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

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