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Sponsor elected not to continue with study
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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.
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:
Part B:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cannabidiol Oral Solution | Drug | 20 or 40 mg/kg/day BID |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 14 |
| Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs) | Up to Week 64 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14 | Day 14 | |
| Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14 | Day 14 | |
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Inclusion Criteria:
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:
During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
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| Name | Affiliation | Role |
|---|---|---|
| Neha Parikh | INSYS Therapeutics Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mattel Children's Hospital at UCLA | Los Angeles | California | 90095 | United States | ||
| University of California - San Francisco |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Percentage of participants | Day 14 |
|
Up to 64 weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cannabidiol Oral Solution: 20-40 mg/kg/Day BID | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infantile Spasms | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Clinical Development | Insys Therapeutics, Inc. | 480-500-3105 | gdecastro@insysrx.com |
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| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| D002185 | Cannabidiol |
| ID | Term |
|---|---|
| D002186 | Cannabinoids |
| D013729 | Terpenes |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
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| Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14 |
| Baseline, 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 | Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. |
| 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. | 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. | 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. | 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 | Up to Week 64 |
| 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 | Up to Week 64 |
| Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B. | Up to Week 64 |
| Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG | Up to Week 64 |
| Part B: Time to Relapse as Confirmed by Video-EEG | Up to Week 64 |
| San Francisco |
| California |
| 94143 |
| United States |
| Miami Children's Hospital | Miami | Florida | 33155 | United States |
| Beaumont Health System | Royal Oak | Michigan | 48073 | United States |
| Months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Primary | Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs) | Safety Analysis Population: included all participants who received at least one dose of study drug. | Posted | Number | Percentage of participants | Up to Week 64 |
|
|
|
| Secondary | Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14 | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Percentage of participants | Day 14 |
|
|
|
| Secondary | Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14 | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Percentage of participants | Day 14 |
|
|
|
| Secondary | Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14 | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Median | Inter-Quartile Range | Number of spasms | Baseline, Day 14 |
|
|
|
| Secondary | 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 | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Mean | Standard Deviation | Units on a scale | Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. |
|
|
|
| Secondary | 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. | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Percentage of participants | Day 14 |
|
|
|
| Secondary | 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. | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Percentage of participants | Day 14 |
|
|
|
| Secondary | 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. | Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14. | Posted | Number | Days | Day 14 |
|
|
|
| Secondary | 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 | Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination. | Posted | Up to Week 64 |
|
|
| Secondary | 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 | Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination. | Posted | Up to Week 64 |
|
|
| Secondary | Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B. | Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination. | Posted | Up to Week 64 |
|
|
| Secondary | Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG | Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination. | Posted | Up to Week 64 |
|
|
| Secondary | Part B: Time to Relapse as Confirmed by Video-EEG | Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination. | Posted | Up to Week 64 |
|
|
| 0 |
| 9 |
| 0 |
| 9 |
| 4 |
| 9 |
| Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
|
| Irritability | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
|
| Sedation | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
|
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| D009422 |
| Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
| Title | Measurements |
|---|
|
|
| Visit 5 (Week 8) |
|
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| Visit 6 (Week 10) |
|
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| Early Discontinuation/ End of Study |
|
|