Open-label Extension to Protocol 1042-0500

This study has been terminated.
(ganaxolone did not significantly reduce spasms after an 8-day placebo control period)
Sponsor:
Information provided by:
Marinus Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00442104
First received: February 27, 2007
Last updated: October 28, 2009
Last verified: October 2009
  Purpose

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


Condition Intervention Phase
Infantile Spasms
Drug: Ganaxolone
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.

Resource links provided by NLM:


Further study details as provided by Marinus Pharmaceuticals:

Primary Outcome Measures:
  • Number of subjects who are free of spasms measured by 24-hour vEEG [ Time Frame: Week 96 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Spasm frequency measured by diary. [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]
  • Cessation of hypsarrythmia, as determined by vEEG. [ Time Frame: week 96 ] [ Designated as safety issue: No ]
  • Physician and caregiver global assessment of seizure severity and response to treatment. [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]
  • The number of subjects spasm-free and seizure-free (for at least 24 hours) at each visit. [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]
  • Responders (≥ 50% decrease in spasm frequency) at each visit. [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Investigators and caregivers assessment of the presence and absence of spasms in each subject following treatment. [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]
  • Developmental assessment [ Time Frame: throughout whole study ] [ Designated as safety issue: No ]

Enrollment: 54
Study Start Date: January 2007
Study Completion Date: March 2009
Primary Completion Date: March 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ganaxolone Drug: Ganaxolone

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

Ages Eligible for Study:   4 Months to 24 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00442104

Locations
United States, California
Mattel Children's Hospital at UCLA
Los Angeles, California, United States, 90095
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
Miami Children's Hospital, The Brain Institute
Miami, Florida, United States, 33155
Child Neurology Center of Nrothwest Florida, P.A.
Pensacola, Florida, United States, 32504
United States, Illinois
University of Chicago Comer Children's Hospital
Chicago, Illinois, United States, 60637
United States, Minnesota
Minnesota Epilepsy Group, P.A.
St. Paul, Minnesota, United States, 55102
United States, New York
Montefiore Medical Center- Albert Einstein College of Medicine
Bronx, New York, United States, 10467
United States, Tennessee
Le Bonheur Children's Medical Center
Memphis, Tennessee, United States, 38105
United States, Texas
Dallas Pediatric Neurology Associates
Dallas, Texas, United States, 75230
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Virginia
Virginia Commonwealth University Health Systems
Richmond, Virginia, United States, 23298
United States, Washington
Children's Hospital and Regional Medical Center
Seattle, Washington, United States, 98105
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53201
Sponsors and Collaborators
Marinus Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: Julia Tsai/ Director Clinical Operations, Marinus Pharmaceutical, Inc
ClinicalTrials.gov Identifier: NCT00442104     History of Changes
Other Study ID Numbers: 1042-0501, Amend 5 (ROW), Amend 6 (US)
Study First Received: February 27, 2007
Last Updated: October 28, 2009
Health Authority: United States: Food and Drug Administration

Keywords provided by Marinus Pharmaceuticals:
infantile spasms
anticonvulsant
pediatric epilepsy
West Syndrome
epileptic spasms

Additional relevant MeSH terms:
Spasm
Spasms, Infantile
Brain Diseases
Central Nervous System Diseases
Epilepsy
Epilepsy, Generalized
Nervous System Diseases
Neurologic Manifestations
Neuromuscular Manifestations
Signs and Symptoms

ClinicalTrials.gov processed this record on October 21, 2014