| ID | Type | Description | Link |
|---|---|---|---|
| B4511001 | Other Identifier | Alias Study Number |
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To evaluate the efficacy and safety of diazepam in the management of refractory epilepsy in selected patients who require intermittent medical intervention for the control of episodes of acute repetitive seizures. In addition, to assess the support provided by caregivers who are not themselves or not under the direct supervision of health care professionals at the time of administration.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of acute repetitive seizures (ARS). |
|
| Diazepam | Experimental | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of diazepam solution, ranging from 0.2 to 0.5 mg/kg, as a deep intramuscular injection in the mid to outer thigh. Additional doses were permissible during the Open-label Period. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Intramuscular autoinjector; administered at onset of an episode |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Next Seizure or Rescue Medication During the Double-blind Period (Kaplan-Meier 50th Percentile) | An event was defined as an episode of or required rescue medication for an episode of acute repetitive seizures (ARS) within 15 minutes to 12 hours following study drug administration. Patients without an ARS event were censored at 12 hours. Diaries were provided; if no diary was returned, or the diary did not provide answers to questions about seizures and rescue during the 12-hour follow-up period, the patient was considered censored as of 15 minutes past the treatment time, unless another contact was documented. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. Patients and their caregivers were trained to recognize the onset of an episode of ARS and when and how to administer study drug. | From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period |
| Percentage of Participants With an Event (Next Seizure or Rescue Medication) During the Open-label Period | An event was defined as an episode of or required rescue medication for an episode of acute repetitive seizures (ARS) within 15 minutes to 12 hours following study drug administration. Patients without an ARS event were censored at 12 hours. Diaries were provided; if no diary was returned, or the diary did not provide answers to questions about seizures and rescue during the 12-hour follow-up period, the patient was considered censored as of 15 minutes past the treatment time, unless another contact was documented. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. Patients and their caregivers were trained to recognize the onset of an episode of ARS and when and how to administer study drug. | From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Requiring Rescue Medication During the Double-blind Period | If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the acute repetitive seizure (ARS) episode. Each patient's specific criteria for seizure and an episode of ARS were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. An episode of ARS was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. |
Not provided
Key Inclusion Criteria
For Patient:
For Caregiver:
Age of 18 years or older and has demonstrated responsibility as a caregiver through training to:
Key Exclusion Criteria
For Patient:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurology Clinic, PC | Northport | Alabama | 35476 | United States | ||
| Clinical Trials, Inc |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24111974 | Derived | Abou-Khalil B, Wheless J, Rogin J, Wolter KD, Pixton GC, Shukla RB, Sherman NA, Sommerville K, Goli V, Roland CL. A double-blind, randomized, placebo-controlled trial of a diazepam auto-injector administered by caregivers to patients with epilepsy who require intermittent intervention for acute repetitive seizures. Epilepsia. 2013 Nov;54(11):1968-76. doi: 10.1111/epi.12373. Epub 2013 Sep 20. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Of 234 participants randomized, 71 (29 placebo, 42 diazepam) did not receive/attempt to receive treatment. Discontinuation prior to treatment due to: 3,0 protocol violations; 6,5 sponsor's request; 4,8 Principal Investigator request; 5,11 other; 0,2 lost to follow-up, and 9,10 withdrawn consent.
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| ID | Title | Description |
|---|---|---|
| FG000 | Diazepam | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of diazepam solution, ranging from 0.2 to 0.5 mg/kg, administered by caregivers untrained as and unsupervised by healthcare professionals. Drug was delivered by a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of acute repetitive seizures (ARS). Additional doses were permissible as needed during the Open-label and Open-label Extension Periods. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-blind Period |
|
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| Vanquix Auto-Injector (Diazepam Injection) |
| Drug |
Intramuscular autoinjector: 5, 10, 15, or 20 mg (based on participant's age and weight); administered at the onset of an episode |
|
| From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period |
| Number of Participants Requiring Emergency Department Visits During the Double-blind Period | Any use of emergency treatment (such as an emergency room visit) was recorded in the patient's diary, along with the date, time, and reason for the emergency treatment. Emergency department visits required some type of rescue action taken, other than the visit itself. An episode of acute repetitive seizures (ARS) was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | From 15 minutes to 12 hours following study drug administration for onset of an episode of ARS during the Double-blind Period |
| Number of Participants Requiring Rescue Medical Care Other Than Rescue Medication or Emergency Department Visits During the Double-blind Period | Other rescue medical care consisted of care other than rescue medication or emergency department visits. Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. An episode of ARS was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period |
| Mean Score on Caregiver Global Treatment Assessment During the Double-blind Period | Caregiver global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | Assessments completed at the end of each treated episode of ARS in the Double-blind Period |
| Mean Score on Physician Global Treatment Assessment During the Double-blind Period | Physician global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes. The physician global evaluation is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | At Visit 2 and subsequent visits in the Double-blind Period |
| Number of Participants Requiring Rescue Medication During the Open-label Period | Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. | From 15 minutes to 12 hours after study drug administration during the Open-label Period |
| Number of Participants Requiring Emergency Department Visits During the Open-label Period | Any use of emergency treatment (such as an emergency room visit) was recorded in the patient's diary, along with the date, time, and reason for the emergency treatment. Emergency department visits required some type of rescue action taken, other than the visit itself. | From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period |
| Number of Participants Requiring Rescue Medical Care Other Than Medication or Emergency Department Visits During the Open-label Period | Other rescue medical care consisted of care other than rescue medication or emergency department visits. Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. | From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period |
| Mean Score on Caregiver Global Treatment Assessment During the Open-label Period | Caregiver global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | Assessments completed at the end of each treated episode of ARS in the Open-label Period |
| Mean Score on Physician Global Treatment Assessment During the Open-label Period | Physician global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | From Visit 2 and subsequent visits in the Open-label Period to discharge or study termination |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Stein Life Child Neurology Medical Specialists Inc. | Irvine | California | 92606 | United States |
| Collaborative NeuroScience Network, LLC | Long Beach | California | 90806 | United States |
| Hoag Memorial Hospital Presbyterian | Newport Beach | California | 92658 | United States |
| Brain and Spine Surgeons of Orange County | Newport Beach | California | 92663 | United States |
| Drug Shipment | Sacramento | California | 95816 | United States |
| Sacramento Comprehensive Epilepsy Program | Sacramento | California | 95816 | United States |
| Sutter Cancer Center Specialty Clinic | Sacramento | California | 95816 | United States |
| Sutter Institute for Medical Research | Sacramento | California | 95816 | United States |
| Northern California Cardiology | Sacramento | California | 95819 | United States |
| Bradenton Research Center, Inc. | Bradenton | Florida | 34205 | United States |
| Morton Plant Hospital Epilepsy Clinic | Clearwater | Florida | 33756 | United States |
| Morton Plant Hospital Pharmacy | Clearwater | Florida | 33756 | United States |
| Child Neurology Center of NorthWest Florida | Gulf Breeze | Florida | 32561 | United States |
| NorthWest Florida Clinical Research Group, LLC | Gulf Breeze | Florida | 32561 | United States |
| Emery Neuroscience Center | Lighthouse PT | Florida | 33064 | United States |
| Pediatric Neurology and Epilepsy Center | Loxahatchee Groves | Florida | 33470 | United States |
| Miami Children's Hospital | Miami | Florida | 33155 | United States |
| EKG only | Orlando | Florida | 32806 | United States |
| Pediatric Neurology, PA | Orlando | Florida | 32819 | United States |
| AMO Corp | Tallahassee | Florida | 32308 | United States |
| Tallahassee Neurological Clinic | Tallahassee | Florida | 32308 | United States |
| Pediatric Epilepsy & Neurology Specialists | Tampa | Florida | 33609 | United States |
| Willsey Research Inc | Tampa | Florida | 33613 | United States |
| Child Neurology Associates, PC | Atlanta | Georgia | 30342 | United States |
| Savannah Neurology, PC | Savannah | Georgia | 31405 | United States |
| Consultants in Epilepsy and Neurology, PLLC | Boise | Idaho | 83702 | United States |
| Comer Children's Hospital | Chicago | Illinois | 60637 | United States |
| University of Chicago Hospital Pharmacy | Chicago | Illinois | 60637 | United States |
| University of Chicago Medical Center (UCMC) Center for Advanced Medicine (CAM) | Chicago | Illinois | 60637 | United States |
| University of Chicago Medical Center (UCMC) | Chicago | Illinois | 60637 | United States |
| University Neurologists PSC Pediatric Division | Louisville | Kentucky | 40202 | United States |
| University of Louisville Ambulatory Care Building Clinic | Louisville | Kentucky | 40202 | United States |
| University of Louisville Clinical Trials Unit | Louisville | Kentucky | 40202 | United States |
| Lahey Clinic Medical Center | Burlington | Massachusetts | 01805 | United States |
| Neurology Clinic of St Cloud | Saint Cloud | Minnesota | 56303 | United States |
| The Comprehensive Epilepsy Care Center For Children and Adults | Chesterfield | Missouri | 63017 | United States |
| MRI Location | Kansas City | Missouri | 64111 | United States |
| St Luke's Hospital Neurological Consultants | Kansas City | Missouri | 64111 | United States |
| St Luke's Hospital | Kansas City | Missouri | 64111 | United States |
| Dartmouth-Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Clinical Research Center of New Jersey | Gibbsboro | New Jersey | 08026 | United States |
| University of Medicine and Dentistry of New Jersey Robert Wood Johnson Medical Group | New Brunswick | New Jersey | 08901 | United States |
| University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| Elmwood Clinic | Buffalo | New York | 14207 | United States |
| Millard Fillmore Gates Hospital/Comprehensive Epilepsy Center | Buffalo | New York | 14209 | United States |
| Women and Children's Hospital of Buffalo | Buffalo | New York | 14222 | United States |
| NYU Medical Center, Comprehensive Epilepsy Center | New York | New York | 10016 | United States |
| Strong Memorial Hospital | Rochester | New York | 14642 | United States |
| University of Rochester, Strong Epilepsy Center | Rochester | New York | 14642 | United States |
| Cone Health Child Neurology | Greensboro | North Carolina | 27401 | United States |
| Guilford Neurologic Associates | Greensboro | North Carolina | 27405 | United States |
| Raleigh Neurology Associates, PA | Raleigh | North Carolina | 27607 | United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195-5102 | United States |
| The Cleveland Clinic Health Systems | Cleveland | Ohio | 44195-5102 | United States |
| Ohio State University Neurology Clinic | Columbus | Ohio | 43210 | United States |
| Ohio State University University Hospital | Columbus | Ohio | 43210 | United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| North Pacific Epilepsy Research/The Northrup Center | Portland | Oregon | 97210 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| St. Christopher's Hospital for Children | Philadelphia | Pennsylvania | 19134-1095 | United States |
| St. Christopher's Hospital for Children | Philadelphia | Pennsylvania | 19134 | United States |
| Wellspan Neurosciences | York | Pennsylvania | 17402 | United States |
| Apple Hill Medical Center (EKG & Lab Draw) | York | Pennsylvania | 17403 | United States |
| Medical University of South Carolina Hospitals and Clinics | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina/Department of Pharmacy Service | Charleston | South Carolina | 29425 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| The Neurology and Pain Clinic | Orangeburg | South Carolina | 29118 | United States |
| Mid-South Physicians Group, PLLC | Germantown | Tennessee | 38138 | United States |
| University of Tennessee Lebonheur Pediatric Specialists Inc. | Memphis | Tennessee | 38103 | United States |
| Access Clinical Trial, Inc. | Nashville | Tennessee | 37203 | United States |
| CMC - Physician's Park | Nashville | Tennessee | 37203 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232-2551 | United States |
| Vanderbilt Epilepsy Clinic | Nashville | Tennessee | 37232 | United States |
| Vanderbilt University Hospital Pharmacy | Nashville | Tennessee | 37232 | United States |
| Neurological Clinic of Texas, PA | Dallas | Texas | 75230 | United States |
| Cook Children's Medical Center Office of Grants and Research | Fort Worth | Texas | 76104 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Cook Children's Physcian Network | Fort Worth | Texas | 76104 | United States |
| Alamo City Clinical Research, LLC | San Antonio | Texas | 78258 | United States |
| Virginia Commonwealth University Health System | Richmond | Virginia | 23298-0042 | United States |
| Virginia Commonwealth University Division of Child Neurology | Richmond | Virginia | 23298-0211 | United States |
| Virginia Commonwealth University Ambulatory Care Center/Department of Neurology | Richmond | Virginia | 23298-0599 | United States |
| Harborview Medical Center | Seattle | Washington | 98104 | United States |
| University of Washington Regional Epilepsy Center, Harborview Medical Center | Seattle | Washington | 98104 | United States |
| FG001 | Placebo | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. |
| Received Treatment |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open-label Period |
|
|
| Open-label Extension Period |
|
|
Participants who were randomized to receive treatment
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Diazepam | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of diazepam solution, ranging from 0.2 to 0.5 mg/kg, administered by caregivers untrained as and unsupervised by healthcare professionals. Drug was delivered by a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of acute repetitive seizures (ARS). Additional doses were permissible as needed during the Open-label and Open-label Extension Periods. |
| BG001 | Placebo | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. |
| 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 | Mean | Standard Deviation | years |
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| Age, Customized | Number | participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | Participants |
| ||||||||||||||||
| Place of Residence | Number | Participants |
| ||||||||||||||||
| Height | Mean | Standard Deviation | Centimeters |
| |||||||||||||||
| Weight | Mean | Standard Deviation | Kilograms |
|
| 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 | Time to Next Seizure or Rescue Medication During the Double-blind Period (Kaplan-Meier 50th Percentile) | An event was defined as an episode of or required rescue medication for an episode of acute repetitive seizures (ARS) within 15 minutes to 12 hours following study drug administration. Patients without an ARS event were censored at 12 hours. Diaries were provided; if no diary was returned, or the diary did not provide answers to questions about seizures and rescue during the 12-hour follow-up period, the patient was considered censored as of 15 minutes past the treatment time, unless another contact was documented. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. Patients and their caregivers were trained to recognize the onset of an episode of ARS and when and how to administer study drug. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Double-blind Period. | Posted | Median | 95% Confidence Interval | Hours | From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period |
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| Primary | Percentage of Participants With an Event (Next Seizure or Rescue Medication) During the Open-label Period | An event was defined as an episode of or required rescue medication for an episode of acute repetitive seizures (ARS) within 15 minutes to 12 hours following study drug administration. Patients without an ARS event were censored at 12 hours. Diaries were provided; if no diary was returned, or the diary did not provide answers to questions about seizures and rescue during the 12-hour follow-up period, the patient was considered censored as of 15 minutes past the treatment time, unless another contact was documented. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. Patients and their caregivers were trained to recognize the onset of an episode of ARS and when and how to administer study drug. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Open-label Period. | Posted | Number | Percentage of participants | From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period |
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| Secondary | Number of Participants Requiring Rescue Medication During the Double-blind Period | If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the acute repetitive seizure (ARS) episode. Each patient's specific criteria for seizure and an episode of ARS were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. An episode of ARS was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS during the Double-blind Period. | Posted | Number | Participants | From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Requiring Emergency Department Visits During the Double-blind Period | Any use of emergency treatment (such as an emergency room visit) was recorded in the patient's diary, along with the date, time, and reason for the emergency treatment. Emergency department visits required some type of rescue action taken, other than the visit itself. An episode of acute repetitive seizures (ARS) was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an episode of ARS during the Double-blind Period. | Posted | Number | Participants | From 15 minutes to 12 hours following study drug administration for onset of an episode of ARS during the Double-blind Period |
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| Secondary | Number of Participants Requiring Rescue Medical Care Other Than Rescue Medication or Emergency Department Visits During the Double-blind Period | Other rescue medical care consisted of care other than rescue medication or emergency department visits. Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. An episode of ARS was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Double-blind Period. | Posted | Number | Participants | From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Score on Caregiver Global Treatment Assessment During the Double-blind Period | Caregiver global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an episode of ARS during the Double-blind Period. | Posted | Mean | Standard Deviation | Units on a scale | Assessments completed at the end of each treated episode of ARS in the Double-blind Period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Score on Physician Global Treatment Assessment During the Double-blind Period | Physician global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes. The physician global evaluation is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) was defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an episode of ARS during the Double-blind Period. | Posted | Mean | Standard Deviation | Units on a scale | At Visit 2 and subsequent visits in the Double-blind Period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Requiring Rescue Medication During the Open-label Period | Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. If seizure control following study drug administration was inadequate, diazepam rectal gel was provided as a rescue medication, given only in the first 4 hours after study drug administration and only if the caregiver was directed to do so by the Investigator or designee at the time of the ARS episode. | All randomized participants in whom an attempt (successful or not) was made to administer study drug for an ARS episode during the Open-label Period of the study. | Posted | Number | Participants | From 15 minutes to 12 hours after study drug administration during the Open-label Period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Requiring Emergency Department Visits During the Open-label Period | Any use of emergency treatment (such as an emergency room visit) was recorded in the patient's diary, along with the date, time, and reason for the emergency treatment. Emergency department visits required some type of rescue action taken, other than the visit itself. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Open-label Period. | Posted | Number | Participants | From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Requiring Rescue Medical Care Other Than Medication or Emergency Department Visits During the Open-label Period | Other rescue medical care consisted of care other than rescue medication or emergency department visits. Each patient's specific criteria for seizure and an episode of acute repetitive seizure (ARS) were determined by the Investigator. Patients and their caregivers were trained to use these criteria to recognize the onset of an episode of ARS and when and how to administer study drug. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Double-blind Period. | Posted | Number | Participants | From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Score on Caregiver Global Treatment Assessment During the Open-label Period | Caregiver global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Open-label Period and who were available for participation. | Posted | Mean | Standard Deviation | Units on a scale | Assessments completed at the end of each treated episode of ARS in the Open-label Period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Mean Score on Physician Global Treatment Assessment During the Open-label Period | Physician global evaluation is based on seizure frequency, severity, and overall outcome compared with previous episodes and is rated on a 10-cm visual analogue scale, where 0=much worse and 10=much better. A higher score indicates greater improvement. An episode of acute repetitive seizures (ARS) is defined as an episode of multiple complex, partial, or generalized seizures occurring over a brief period (minutes to 12 hours) with the patient regaining consciousness between seizures, which were readily recognizable by the patient or a trained caregiver. ARS includes seizures sometimes referred to as serial, cluster, crescendo, or stuttering prolonged. | All randomized participants for whom an attempt (successful or not) was made to administer study drug for an ARS event during the Open-label Period and who were available for evaluation. | Posted | Mean | Standard Deviation | Units on a scale | From Visit 2 and subsequent visits in the Open-label Period to discharge or study termination |
|
Not provided
An adverse event is any untoward unfavorable unintended sign, symptom, or disease temporally associated with the use of drug, whether or not considered related to that drug. A serious adverse event is any untoward medical occurrence that results in death or disability/incapacity; is life-threatening; or requires or prolongs hospitalization.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Diazepam (Double-blind Period) | Participants received a single, age- and weight-appropriate dose of diazepam solution, ranging from 0.2 to 0.5 mg/kg, administered by caregivers untrained as and unsupervised by healthcare professionals. Drug was delivered by a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. | 2 | 81 | 33 | 81 | ||
| EG001 | Diazepam (Open-label Period) | Participants received an age- and weight-appropriate dose of placebo solution administered using a spring-driven, pressure-activated, prefilled autoinjector. Additional doses were permissible as needed. | 20 | 128 | 43 | 128 | ||
| EG002 | Diazepam (Open-label Extension) | Participants continued to receive diazepam in an age- and weight-appropriate dose of administered using a spring-driven, pressure-activated, prefilled autoinjector. Additional doses were permissible as needed at the onset of an ARS episode. Participants were to continue until drug became marketed. | 11 | 33 | 20 | 33 | ||
| EG003 | Placebo (Double-blind Period) | Participants received a single dose of diazepam-matching solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. | 3 | 79 | 32 | 79 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| GAIT DISTURBANCES | General disorders | MedDRA | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| STAPHYLOCOCCAL BACTERAEMIA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| EPILEPSY | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| LOSS OF CONSCIOUSNESS | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| STATUS EPILEPTICUS | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| POSTICTAL STATE | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| FEMUR FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| GASTROINTESTINAL STOMA COMPLICATION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| HEAT STROKE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| JAW FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| CORNEAL ABRASION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| TOXICITY TO VARIOUS AGENTS | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| AFFECTIVE DISORDER | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ANXIETY DISORDER | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DEPRESSION | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HALLUCINATION, AUDITORY | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MENTAL STATUS CHANGES | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PSYCHOTIC DISORDER | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SUICIDAL IDEATION | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ACUTE RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| RESPIRATORY DISTRESS | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MUSCULAR WEAKNESS | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MUSCULOSKELETAL CHEST PAIN | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| SEPTIC SHOCK | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| APPENDICITIS PERFORATED | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| CELLULITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| CELLULITIS ORBITAL | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| HORDEOLUM | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| HYPOCALCAEMIA | Metabolism and nutrition disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPOMAGNESAEMIA | Metabolism and nutrition disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ANGINA PECTORIS | Cardiac disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ATRIAL FIBRILLATION | Cardiac disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MYOCARDIAL INFARCTION | Cardiac disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DYSPHAGIA | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| GASTROINTESTINAL HAEMORRHAGE | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| NEPHROTIC SYNDROME | Renal and urinary disorders | MedDRA 10.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| INJECTION SITE PAIN | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE HEMORRHAGE | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE BRUISING | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE SWELLING | General disorders | MedDRA | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA | Systematic Assessment |
| |
| ADVERSE DRUG REACTIONS | General disorders | MedDRA | Systematic Assessment |
| |
| CHILLS | General disorders | MedDRA | Systematic Assessment |
| |
| ENERGY INCREASED | General disorders | MedDRA | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE ERYTHEMIA | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE LACERATION | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE REACTION | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE ANESTHESIA | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE DISCOMFORT | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE IRRITATION | General disorders | MedDRA | Systematic Assessment |
| |
| INJECTION SITE MASS | General disorders | MedDRA | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| EPILEPSY | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| HYPOESTHESIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| SOMNOLENCE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| ALTERED STATE OF CONCIOUSNESS | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| POSTICTAL HEADACHE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| CONTUSION | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| EXCORIATION | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| MOUTH INJURY | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| PROCEDURAL COMPLICATION | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| AGITATION | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| AGGRESSION | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| CONFUSIONAL STATE | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| CRYING | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| LOGORRHEA | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| DISORIENTATION | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| MOOD ALTERED | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| NERVOUSNESS | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| RESTLESSNESS | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| HAEMATOCHEZIA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| RECTAL HEMORRHAGE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| MUSCLE TIGHTNESS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCULAR WEAKNESS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCULOSKELETAL PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| HICCUPS | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| YAWNING | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| PHARYNGOLARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| HEART RATE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| WEIGHT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| INCREASED APPETITE | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| HYPERTENSION | Vascular disorders | MedDRA | Systematic Assessment |
| |
| FLUSHING | Vascular disorders | MedDRA | Systematic Assessment |
| |
| CONJUNCTIVAL HYPERAEMIA | Eye disorders | MedDRA | Systematic Assessment |
| |
| VISUAL ACUITY REDUCED | Eye disorders | MedDRA | Systematic Assessment |
| |
| EAR INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| EDEMA PERIPHERAL | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ASTHENIA | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| CYST | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| GAIT DISTURBANCE | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PAIN | General disorders | MedDRA 10.0 | Systematic Assessment |
| |
| LETHARGY | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ATAXIA | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| TREMOR | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| FACIAL PARESIS | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MIGRAINE | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| NYSTAGMUS | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| TARDIVE DYSKINESIA | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| VOCAL CORD PARALYSIS | Nervous system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SKIN LACERATION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| THERAPEUTIC AGENT TOXICITY | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| LACERATION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| LIMB INJURY | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| ANKLE FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| FIBULA FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| FOOT FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| HEAD INJURY | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| INCISION SITE PAIN | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| JOINT DISLOCATION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| JOINT INJURY | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| LIGAMENT SPRAIN | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| LIP INJURY | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| LOWER LIMB FRACTURE | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| MUSCLE STRAIN | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| PERIORBITAL CONTUSION | Injury, poisoning and procedural complications | MedDRA 10.0 | Systematic Assessment |
| |
| INSOMNIA | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ADJUSTMENT DISORDER | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HALLUCINATION, VISUAL | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| MENTAL STATUS CHANGES | Psychiatric disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| GASTROESOPHAGEAL REFLUX DISEASE | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| COELIAC DISEASE | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DIARRHEA | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HEMORRHOIDS | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPOAESTHESIA ORAL | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SALIVARY GLAND MASS | Gastrointestinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| BONE PAIN | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| JOINT SWELLING | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PAIN IN JAW | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SCOLIOSIS | Musculoskeletal and connective tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PLEURAL EFFUSION | Respiratory, thoracic and mediastinal disorders | MedDRA 10.0 | Systematic Assessment |
| |
| WEIGHT INCREASED | Investigations | MedDRA 10.0 | Systematic Assessment |
| |
| ASPIRATION TRACHEAL | Investigations | MedDRA 10.0 | Systematic Assessment |
| |
| URINE OUTPUT DECREASED | Investigations | MedDRA 10.0 | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPERLIPIDEMIA | Metabolism and nutrition disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPERHIDROSIS | Skin and subcutaneous tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPERKERATOSIS | Skin and subcutaneous tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| RASH PAPULAR | Skin and subcutaneous tissue disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DEEP VEIN THROMBOSIS | Vascular disorders | MedDRA 10.0 | Systematic Assessment |
| |
| HYPOTENSION | Vascular disorders | MedDRA 10.0 | Systematic Assessment |
| |
| VISION BLURRED | Eye disorders | MedDRA 10.0 | Systematic Assessment |
| |
| DIPLOPIA | Eye disorders | MedDRA 10.0 | Systematic Assessment |
| |
| ANAEMIA | Blood and lymphatic system disorders | MedDRA 10.0 | Systematic Assessment |
| |
| PERICARDIAL EFFUSION | Cardiac disorders | MedDRA 10.0 | Systematic Assessment |
| |
| SINUS TACHYCARDIA | Cardiac disorders | MedDRA 10.0 | Systematic Assessment |
| |
| EAR PAIN | Ear and labyrinth disorders | MedDRA 10.0 | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| SINUSITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| OTITIS MEDIA | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| ACUTE SINUSITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| GASTROENTERITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| PHARYNGITIS STREPTOCOCCAL | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
| |
| TONSILLITIS | Infections and infestations | MedDRA 10.0 | Systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| ID | Term |
|---|---|
| D012640 | Seizures |
| D004828 | Epilepsies, Partial |
| D017029 | Epilepsy, Complex Partial |
| D004829 | Epilepsy, Generalized |
| D004827 | Epilepsy |
| ID | Term |
|---|---|
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D003975 | Diazepam |
| ID | Term |
|---|---|
| D001570 | Benzodiazepinones |
| D001569 | Benzodiazepines |
| D001552 | Benzazepines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
Not provided
Not provided
| Withdrawal by Subject |
|
| Principal Investigator decision |
|
| Sponsor request |
|
| Lost to Follow-up |
|
| Did not receive treatment |
|
| Other |
|
| Other |
|
| Principal investigator decision |
|
| Sponsor request |
|
| Withdrawal by Subject |
|
| 6 to 11 years |
|
| 12 years and older |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black/African American |
|
| Asian |
|
| Native Hawaiian or other Pacific Islander |
|
| American Indian or Alaska Native |
|
| Other |
|
| Missing |
|
| Foster home |
|
| Group home |
|
| Residential facility |
|
| Other |
|
|
|
| OG001 | Placebo | During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. |
|
|
|
During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS.
|
|
|
| Placebo |
During the Double-blind Period, participants received a single, age- and weight-appropriate dose of placebo solution as a deep intramuscular injection in the mid to outer thigh. Drug was administered by a caregiver using a spring-driven, pressure-activated, prefilled autoinjector at the onset of an episode of ARS. |
|
|
|
|
|
|
|
|
|
| Participants |
|
|
|
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| Units | Counts |
|---|
| Participants |
|
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| Units | Counts |
|---|
| Participants |
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|