Open-label Extension to Protocol 1042-0500
Condition: Infantile Spasms
MeSH terms: Spasm; Spasms, Infantile
Intervention: Ganaxolone
Intervention MeSH terms: Ganaxolone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 54.0
Sex: All
Minimum age: 4 Months
Maximum age: 24 Months
Healthy volunteers: No
Keywords: infantile spasms; anticonvulsant; pediatric epilepsy; West Syndrome; epileptic spasms

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

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.