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Open-label Extension to Protocol 1042-0500

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ClinicalTrials.gov Identifier: NCT00442104
Recruitment Status : Terminated (ganaxolone did not significantly reduce spasms after an 8-day placebo control period)
First Posted : March 1, 2007
Last Update Posted : November 2, 2009
Sponsor:
Information provided by:
Marinus Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE February 27, 2007
First Posted Date  ICMJE March 1, 2007
Last Update Posted Date November 2, 2009
Study Start Date  ICMJE January 2007
Actual Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 28, 2009)
Number of subjects who are free of spasms measured by 24-hour vEEG [ Time Frame: Week 96 ]
Original Primary Outcome Measures  ICMJE
 (submitted: February 28, 2007)
Number of subjects who are free of spasms measured by 24-hour vEEG
Change History Complete list of historical versions of study NCT00442104 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 28, 2009)
  • Spasm frequency measured by diary. [ Time Frame: throughout whole study ]
  • Cessation of hypsarrythmia, as determined by vEEG. [ Time Frame: week 96 ]
  • Physician and caregiver global assessment of seizure severity and response to treatment. [ Time Frame: throughout whole study ]
  • The number of subjects spasm-free and seizure-free (for at least 24 hours) at each visit. [ Time Frame: throughout whole study ]
  • Responders (≥ 50% decrease in spasm frequency) at each visit. [ Time Frame: throughout whole study ]
  • Reduction of other seizure types at each visit. Parents/caregivers will maintain a spasm/seizure diary for clinical study subjects. [ Time Frame: throughout whole study ]
  • Investigators and caregivers assessment of the presence and absence of spasms in each subject following treatment. [ Time Frame: throughout whole study ]
  • Developmental assessment [ Time Frame: throughout whole study ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 28, 2007)
  • Spasm frequency measured by diary.
  • Cessation of hypsarrythmia, as determined by vEEG.
  • Physician and caregiver global assessment of seizure severity and response to treatment.
  • The number of subjects spasm-free and seizure-free (for at least 24 hours) at each visit.
  • Responders (≥ 50% decrease in spasm frequency) at each visit.
  • Reduction of other seizure types at each visit. Parents/caregivers will maintain a spasm/seizure diary for clinical study subjects.
  • Investigators and caregivers assessment of the presence and absence of spasms in each subject following treatment.
  • Developmental assessment
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-label Extension to Protocol 1042-0500
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Infantile Spasms
Intervention  ICMJE Drug: Ganaxolone
Study Arms  ICMJE Experimental: ganaxolone
Intervention: Drug: Ganaxolone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: February 28, 2007)
54
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE March 2009
Actual Primary Completion Date March 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Months to 24 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00442104
Other Study ID Numbers  ICMJE 1042-0501
Amend 5 (ROW)
Amend 6 (US)
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Julia Tsai/ Director Clinical Operations, Marinus Pharmaceutical, Inc
Study Sponsor  ICMJE Marinus Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Marinus Pharmaceuticals
Verification Date October 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP