Open-Label Extension to Protocol 1042-0500 - Full Text View

Sponsored by:	Marinus Pharmaceuticals
Information provided by:	Marinus Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00442104

Purpose

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

Condition	Intervention	Phase
Infantile Spasms	Drug: Ganaxolone	Phase II

Genetics Home Reference related topics: pyridoxal 5'-phosphate-dependent epilepsy

MedlinePlus related topics: Epilepsy

Drug Information available for: Ganaxolone

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	An Open-Label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.

Further study details as provided by Marinus Pharmaceuticals:

Primary Outcome Measures:

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

Secondary Outcome Measures:

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

Estimated Enrollment:	54
Study Start Date:	January 2007

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

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

Locations

United States, California
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Mattel Children's Hospital at UCLA
Los Angeles, California, United States, 90095
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

information about ganaxolone This link exits the ClinicalTrials.gov site

Study ID Numbers:	1042-0501, Amend 5 (ROW), Amend 6 (US)
Study First Received:	February 27, 2007
Last Updated:	December 8, 2008
ClinicalTrials.gov Identifier:	NCT00442104
Health Authority:	United States: Food and Drug Administration

Keywords provided by Marinus Pharmaceuticals:

infantile spasms
anticonvulsant
pediatric epilepsy
West Syndrome
epileptic spasms

Study placed in the following topic categories:

Spasm
Signs and Symptoms
Spasms, Infantile
West syndrome
Epilepsy

Central Nervous System Diseases
Neurologic Manifestations
Infantile spasms
Brain Diseases
Epilepsy, Generalized

Additional relevant MeSH terms:

Neuromuscular Manifestations
Nervous System Diseases

ClinicalTrials.gov processed this record on January 16, 2009