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The study was terminated due to slow enrollment and failure to identify adequate patients that met entry criteria.
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The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment of infants newly diagnosed with Infantile Spasms (IS).
This was a randomized, double-blind, placebo-controlled, parallel-group study in which participants were randomized in a 1:1 ratio to 1 of 2 treatment groups. During the Initial Treatment Period, participants received either vigabatrin plus CBD or vigabatrin plus matching placebo and were dosed approximately every 12 hours, with a meal. This study was comprised of five periods: Screening, Initial Treatment, Extended Treatment, Taper, and Follow up Periods, with a maximum duration of approximately 140 days.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CBD with Vigabatrin | Experimental | Participants received up to 40 milligrams per kilogram per day (mg/kg/day) divided twice daily (BID) of CBD with food. Participants also received up to 150 mg/kg/day BID of vigabatrin with food. |
|
| Placebo with Vigabatrin | Placebo Comparator | Participants received a matching placebo to CBD with food, and also received up to 150 mg/kg/day BID of vigabatrin with food. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cannabidiol Oral Solution | Drug | An oral solution containing pharmaceutical grade cannabidiol (nonplant-based). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Considered Complete Responders | Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG). | Up to Day 15 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Resolution of Infantile Spasms | Resolution of IS was assessed by 24-hour video-EEG. | Up to Day 15 |
| Percentage of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was assessed by 24-hour video-EEG. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States | ||
| Beaumont Children's Hospital |
This study was comprised of five periods. The Screening Period lasted up to 14 days, the Initial Treatment period lasted up to 15 days, the Extended Treatment period lasted up to 75 days, followed by a 6-day Taper period and a 30 day Follow up period, with a maximum duration of approximately 140 days.
This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
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| ID | Title | Description |
|---|---|---|
| FG000 | CBD With Vigabatrin | Participants received up to 40 mg/kg/day twice daily (BID) of CBD orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus CBD 40 mg/kg/day up to Day 75. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 10, 2018 | Apr 28, 2023 |
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| Placebo | Drug | Matching oral solution |
|
| Vigabatrin | Drug | Powder suspension |
|
|
| Up to Day 15 |
| Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I) | Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse. Higher scores indicated worse condition. | Day 15 |
| Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15 | Increase in spasm-free days will be determined by seizure diary entries. | Up to Day 15 |
| Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period | Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse. | Up to Day 75 |
| Time to Relapse During the Extended Treatment Period | Up to Day 75 |
| Royal Oak |
| Michigan |
| 48073 |
| United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Institute for Research and Innovation | MultiCare Health System | Tacoma | Washington | 98405 | United States |
| FG001 | Placebo With Vigabatrin | Participants received a matching placebo to CBD, orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus matching placebo up to Day 75. |
| COMPLETED |
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| NOT COMPLETED |
|
This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
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| ID | Title | Description |
|---|---|---|
| BG000 | CBD With Vigabatrin | Participants received up to 40 mg/kg/day BID of CBD orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus CBD 40 mg/kg/day up to Day 75. |
| BG001 | Placebo With Vigabatrin | Participants received a matching placebo to CBD, orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus matching placebo up to Day 75. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | |||||||||||||||||||||||
| Sex: Female, Male |
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| Ethnicity (NIH/OMB) |
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| 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 | Percentage of Participants Considered Complete Responders | Complete response is defined as complete resolution of spasms and hypsarrhythmia confirmed by 24-hour video-electroencephalogram (EEG). | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 15 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Resolution of Infantile Spasms | Resolution of IS was assessed by 24-hour video-EEG. | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 15 |
| |||||||||||||||||||||||
| Secondary | Percentage of Participants With Resolution of Hypsarrhythmia | Resolution of hypsarrhythmia was assessed by 24-hour video-EEG. | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 15 |
| |||||||||||||||||||||||
| Secondary | Investigator Impression of Efficacy and Tolerability of Study Drug Clinical Global Impression- Global Improvement (CGI-I) | Investigators will use the CGI-I scale, which is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and is rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse. Higher scores indicated worse condition. | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Day 15 |
| |||||||||||||||||||||||
| Secondary | Percentage of Participants With Increase in Number of Spasm-Free Days Between Day 1 and Day 15 | Increase in spasm-free days will be determined by seizure diary entries. | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 15 |
| |||||||||||||||||||||||
| Secondary | Percentage of Participants With Complete Response During the Initial Treatment Period Who Relapse During the Extended Treatment Period | Relapse during the extended treatment period will be confirmed by video-EEG following parent report of relapse. | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 75 |
| |||||||||||||||||||||||
| Secondary | Time to Relapse During the Extended Treatment Period | This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported. | Posted | Up to Day 75 |
|
|
This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
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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 | CBD With Vigabatrin | Participants received up to 40 mg/kg/day BID of CBD orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus CBD 40 mg/kg/day up to Day 75. | 0 | 0 | 0 | 0 | 0 | 0 |
| EG001 | Placebo With Vigabatrin | Participants received a matching placebo to CBD, orally with food during the 15-day Initial Treatment Period. Participants also received up to 150 mg/kg/day BID of vigabatrin orally with food. During the Extended Treatment Period, complete responders continued their assigned treatment of vigabatrin plus matching placebo up to Day 75. | 0 | 0 | 0 | 0 | 0 | 0 |
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This study was terminated by the Sponsor. The Sponsor terminated the study due to slow enrollment and a failure to identify adequate participants that met eligibility criteria. Due to study termination and only 2 participants being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Bruce Mitlak, MD; Head of Discovery Science and Chief Medical Officer | Radius Health | 617-444-1943 | bmitlak@radiuspharm.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 30, 2017 | Apr 28, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D013036 | Spasms, Infantile |
| ID | Term |
|---|---|
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000073376 | Epileptic Syndromes |
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| ID | Term |
|---|---|
| D002185 | Cannabidiol |
| D020888 | Vigabatrin |
| ID | Term |
|---|---|
| D002186 | Cannabinoids |
| D013729 | Terpenes |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D005680 | gamma-Aminobutyric Acid |
| D000613 | Aminobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
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