Open-label Extension to Protocol 1042-0500

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Brief Title

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.

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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Number of subjects who are free of spasms measured by 24-hour vEEG

Secondary Outcome

 Spasm frequency measured by diary.

Condition

Infantile Spasms

Intervention

Ganaxolone

Study Arms / Comparison Groups

 ganaxolone
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

54

Start Date

January 2007

Completion Date

March 2009

Primary Completion Date

March 2009

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.
      

Gender

All

Ages

4 Months - 24 Months

Accepts Healthy Volunteers

No

Contacts

Joseph Hulihan, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00442104

Organization ID

1042-0501

Secondary IDs

Amend 5 (ROW)

Responsible Party

Sponsor

Study Sponsor

Marinus Pharmaceuticals


Study Sponsor

Joseph Hulihan, MD, Study Director, Marinus Pharmaceuticals, Inc.


Verification Date

August 2020