Clinical trial NCT00442104: Open-label Extension to Protocol 1042-0500
Official title: An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Infantile Spasms
Keywords: infantile spasms
anticonvulsant
pediatric epilepsy
West Syndrome
epileptic spasms
Lead sponsor: Marinus Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2007-03-01
Enrollment: 54
Sex: ALL
Minimum age (years): 4
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To allow open-label extension to patients who have completed Protocol 1042-0500

Detailed description: Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.

There will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.

Eligibility criteria: Inclusion Criteria:

* Have completed all scheduled clinical study visits in the previous Protocol 1042 0500 and have been deemed eligible (no SAEs thought to be drug related and had a response to treatment) by the Investigator.
* Be diagnosed with IS regardless of etiology. Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).
* Have a 24 hour vEEG recording confirming the diagnosis of IS.
* Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
* Have been previously treated with 3 AEDs or fewer.
* Have a parent/guardian who is properly informed of the nature and potential risks and benefits of the clinical study, is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study.

Exclusion Criteria:

* Current treatment with more than 2 concomitant AEDs.
* Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain MRI.
* Have any disease or condition (medical or surgical) at Screening that might compromise the hematologic, cardiovascular, pulmonary, renal, GI, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.
* Aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin greater than 4 times the upper limit of laboratory normal or any clinical laboratory value deemed clinically significant by the Investigator.
* History of recurrent status epilepticus.
* Have been exposed to any other investigational drug within 30 days prior to enrollment.

Primary outcomes:
measure: Percentage of Participants Who Were Free of Spasms
timeFrame: Weeks 4 through Week 96
Secondary outcomes:
measure: Change From Baseline in Frequency of Spasm Clusters
timeFrame: Baseline and Week 4 through Week 32
measure: Change From Baseline in Frequency of Individual Spasm
timeFrame: Baseline and Week 4 through Week 32
measure: Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
timeFrame: Week 4 through Week 32
measure: Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
timeFrame: Week 4 through Week 32
measure: Number of Participants With Spasm-free Durations
timeFrame: Week 4 through Week 32
measure: Number of Participants With Absence of Spasms
timeFrame: Week 4 through Week 32
measure: Developmental Assessment Using Denver-II Developmental Test
timeFrame: Week 8 through Week 32
measure: Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.
timeFrame: Weeks 4 through Week 32
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Los Angeles
country: United States
facility: Mattel Children's Hospital at UCLA
state: California
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: Miami Children's Hospital, The Brain Institute
state: Florida
city: Pensacola
country: United States
facility: Child Neurology Center of Nrothwest Florida, P.A.
state: Florida
city: Chicago
country: United States
facility: University of Chicago Comer Children's Hospital
state: Illinois
city: Saint Paul
country: United States
facility: Minnesota Epilepsy Group, P.A.
state: Minnesota
city: Bronx
country: United States
facility: Montefiore Medical Center- Albert Einstein College of Medicine
state: New York
city: Memphis
country: United States
facility: Le Bonheur Children's Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Dallas Pediatric Neurology Associates
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University Health Systems
state: Virginia
city: Seattle
country: United States
facility: Children's Hospital and Regional Medical Center
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
Study officials:
References: