Clinical trial NCT00319501: Efficacy and Safety of Diazepam in the Management of Refractory Epilepsy in Selected Patients Who Require Intermittent Medical Intervention for Acute Repetitive Seizures.
Official title: A Phase 3, Randomized, Double-blind, Parallel, Placebo-controlled, Multicenter Study, With Optional Open-label Continuation, Of The Efficacy And Safety Of Vanquix(tm) Auto-injector (Diazepam Injection) For The Management Of Selected, Refractory, Patients With Epilepsy Who Require Intermittent Medical Intervention To Control Episodes Of Acute Repetitive Seizures
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Seizures
Epilepsies, Partial
Epilepsy, Complex Partial
Epilepsy, Generalized
Epilepsy
Keywords: Acute Repetitive Seizures
ARS
Diazepam
Cluster Seizures
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2006-04-27
Enrollment: 234
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Key Inclusion Criteria

For Patient:

* Older than 2 years of age and between 6 and 136 kg body weight
* Recipient of clinical diagnosis of epilepsy, taking a stable antiepileptic drug regimen for at least 2 weeks, and requiring intermittent medical intervention to control episodes of acute repetitive seizures (ARS)
* Experienced at least 2 episodes of ARS in previous year, one of which occurred in previous 6 months
* Has episodes of ARS that include complex partial or generalized seizures
* Has a responsible caregiver available to participate
* Is not pregnant or lactating and is practicing an acceptable method of birth control.

For Caregiver:

* Age of 18 years or older and has demonstrated responsibility as a caregiver through training to:

  * Recognize an episode of repetitive seizures for which the injection was intended,
  * Administer study drug
  * Count and record seizures and respiratory rate in the patient diary,
  * Monitor the patient and record observations in the patient diary for 12 hours following study drug treatment
  * Recognize the need for immediate medical attention.

Key Exclusion Criteria

For Patient:

* Petit mal status or petit mal variant status
* History of ARS consistently progressing to status epilepticus
* History of failure to respond to benzodiazepine treatment
* Hypersensitivity to diazepam
* Acute narrow angle glaucoma
* Alcohol and/or other substance abuse
* Has taken another investigational drug in previous 30 days
* Acute or progressive neurologic or severe psychiatric disease or severe mental abnormality.

Primary outcomes:
measure: Time to Next Seizure or Rescue Medication During the Double-blind Period (Kaplan-Meier 50th Percentile)
timeFrame: From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period
measure: Percentage of Participants With an Event (Next Seizure or Rescue Medication) During the Open-label Period
timeFrame: From 15 minutes to 12 hours after study drug administration for an episode of ARS during the Double-blind Period
Secondary outcomes:
measure: Number of Participants Requiring Rescue Medication During the Double-blind Period
timeFrame: From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period
measure: Number of Participants Requiring Emergency Department Visits During the Double-blind Period
timeFrame: From 15 minutes to 12 hours following study drug administration for onset of an episode of ARS during the Double-blind Period
measure: Number of Participants Requiring Rescue Medical Care Other Than Rescue Medication or Emergency Department Visits During the Double-blind Period
timeFrame: From 15 minutes to 12 hours following study drug administration for an episode of ARS during the Double-blind Period
measure: Mean Score on Caregiver Global Treatment Assessment During the Double-blind Period
timeFrame: Assessments completed at the end of each treated episode of ARS in the Double-blind Period
measure: Mean Score on Physician Global Treatment Assessment During the Double-blind Period
timeFrame: At Visit 2 and subsequent visits in the Double-blind Period
measure: Number of Participants Requiring Rescue Medication During the Open-label Period
timeFrame: From 15 minutes to 12 hours after study drug administration during the Open-label Period
measure: Number of Participants Requiring Emergency Department Visits During the Open-label Period
timeFrame: From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period
measure: Number of Participants Requiring Rescue Medical Care Other Than Medication or Emergency Department Visits During the Open-label Period
timeFrame: From 15 minutes to 12 hours after study drug administration for onset of an episode of ARS during the Open-label Period
measure: Mean Score on Caregiver Global Treatment Assessment During the Open-label Period
timeFrame: Assessments completed at the end of each treated episode of ARS in the Open-label Period
measure: Mean Score on Physician Global Treatment Assessment During the Open-label Period
timeFrame: From Visit 2 and subsequent visits in the Open-label Period to discharge or study termination
Other outcomes:
Locations:
city: Northport
country: United States
facility: Neurology Clinic, PC
state: Alabama
city: Little Rock
country: United States
facility: Clinical Trials, Inc
state: Arkansas
city: Irvine
country: United States
facility: Stein Life Child Neurology Medical Specialists Inc.
state: California
city: Long Beach
country: United States
facility: Collaborative NeuroScience Network, LLC
state: California
city: Newport Beach
country: United States
facility: Hoag Memorial Hospital Presbyterian
state: California
city: Newport Beach
country: United States
facility: Brain and Spine Surgeons of Orange County
state: California
city: Sacramento
country: United States
facility: Drug Shipment
state: California
city: Sacramento
country: United States
facility: Sacramento Comprehensive Epilepsy Program
state: California
city: Sacramento
country: United States
facility: Sutter Cancer Center Specialty Clinic
state: California
city: Sacramento
country: United States
facility: Sutter Institute for Medical Research
state: California
city: Sacramento
country: United States
facility: Northern California Cardiology
state: California
city: Bradenton
country: United States
facility: Bradenton Research Center, Inc.
state: Florida
city: Clearwater
country: United States
facility: Morton Plant Hospital Epilepsy Clinic
state: Florida
city: Clearwater
country: United States
facility: Morton Plant Hospital Pharmacy
state: Florida
city: Gulf Breeze
country: United States
facility: Child Neurology Center of NorthWest Florida
state: Florida
city: Gulf Breeze
country: United States
facility: NorthWest Florida Clinical Research Group, LLC
state: Florida
city: Lighthouse Point
country: United States
facility: Emery Neuroscience Center
state: Florida
city: Loxahatchee
country: United States
facility: Pediatric Neurology and Epilepsy Center
state: Florida
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Orlando
country: United States
facility: EKG only
state: Florida
city: Orlando
country: United States
facility: Pediatric Neurology, PA
state: Florida
city: Tallahassee
country: United States
facility: AMO Corp
state: Florida
city: Tallahassee
country: United States
facility: Tallahassee Neurological Clinic
state: Florida
city: Tampa
country: United States
facility: Pediatric Epilepsy & Neurology Specialists
state: Florida
city: Tampa
country: United States
facility: Willsey Research Inc
state: Florida
city: Atlanta
country: United States
facility: Child Neurology Associates, PC
state: Georgia
city: Savannah
country: United States
facility: Savannah Neurology, PC
state: Georgia
city: Boise
country: United States
facility: Consultants in Epilepsy and Neurology, PLLC
state: Idaho
city: Chicago
country: United States
facility: Comer Children's Hospital
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Hospital Pharmacy
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center (UCMC) Center for Advanced Medicine (CAM)
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medical Center (UCMC)
state: Illinois
city: Louisville
country: United States
facility: University Neurologists PSC Pediatric Division
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Ambulatory Care Building Clinic
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Clinical Trials Unit
state: Kentucky
city: Burlington
country: United States
facility: Lahey Clinic Medical Center
state: Massachusetts
city: St Cloud
country: United States
facility: Neurology Clinic of St Cloud
state: Minnesota
city: Chesterfield
country: United States
facility: The Comprehensive Epilepsy Care Center For Children and Adults
state: Missouri
city: Kansas City
country: United States
facility: MRI Location
state: Missouri
city: Kansas City
country: United States
facility: St Luke's Hospital Neurological Consultants
state: Missouri
city: Kansas City
country: United States
facility: St Luke's Hospital
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center
state: New Hampshire
city: Gibbsboro
country: United States
facility: Clinical Research Center of New Jersey
state: New Jersey
city: New Brunswick,
country: United States
facility: University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School
state: New Jersey
city: New Brunswick
country: United States
facility: University of Medicine and Dentistry of New Jersey Robert Wood Johnson Medical Group
state: New Jersey
city: Buffalo
country: United States
facility: Elmwood Clinic
state: New York
city: Buffalo
country: United States
facility: Millard Fillmore Gates Hospital/Comprehensive Epilepsy Center
state: New York
city: Buffalo
country: United States
facility: Women and Children's Hospital of Buffalo
state: New York
city: New York
country: United States
facility: NYU Medical Center, Comprehensive Epilepsy Center
state: New York
city: Rochester
country: United States
facility: Strong Memorial Hospital
state: New York
city: Rochester
country: United States
facility: University of Rochester, Strong Epilepsy Center
state: New York
city: Greensboro
country: United States
facility: Cone Health Child Neurology
state: North Carolina
city: Greensboro
country: United States
facility: Guilford Neurologic Associates
state: North Carolina
city: Raleigh
country: United States
facility: Raleigh Neurology Associates, PA
state: North Carolina
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Cleveland
country: United States
facility: The Cleveland Clinic Health Systems
state: Ohio
city: Columbus
country: United States
facility: Ohio State University Neurology Clinic
state: Ohio
city: Columbus
country: United States
facility: Ohio State University University Hospital
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: North Pacific Epilepsy Research/The Northrup Center
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christopher's Hospital for Children
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christopher's Hospital for Children
state: Pennsylvania
city: York
country: United States
facility: Wellspan Neurosciences
state: Pennsylvania
city: York
country: United States
facility: Apple Hill Medical Center (EKG & Lab Draw)
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina Hospitals and Clinics
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina/Department of Pharmacy Service
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Orangeburg
country: United States
facility: The Neurology and Pain Clinic
state: South Carolina
city: Germantown
country: United States
facility: Mid-South Physicians Group, PLLC
state: Tennessee
city: Memphis
country: United States
facility: University of Tennessee Lebonheur Pediatric Specialists Inc.
state: Tennessee
city: Nashville
country: United States
facility: Access Clinical Trial, Inc.
state: Tennessee
city: Nashville
country: United States
facility: CMC - Physician's Park
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt Epilepsy Clinic
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Hospital Pharmacy
state: Tennessee
city: Dallas
country: United States
facility: Neurological Clinic of Texas, PA
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Physcian Network
state: Texas
city: Ft Worth
country: United States
facility: Cook Children's Medical Center Office of Grants and Research
state: Texas
city: San Antonio
country: United States
facility: Alamo City Clinical Research, LLC
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University Health System
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University Division of Child Neurology
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University Ambulatory Care Center/Department of Neurology
state: Virginia
city: Seattle
country: United States
facility: Harborview Medical Center
state: Washington
city: Seattle
country: United States
facility: University of Washington Regional Epilepsy Center, Harborview Medical Center
state: Washington
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: 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.