Open-label Extension to Protocol 1042-0500

NCT ID: NCT00442104

Last Updated: 2024-05-28

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-01-31

Study Completion Date

2009-03-31

Brief Summary

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

Detailed Description

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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.

Conditions

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Infantile Spasms

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ganaxolone

Group Type EXPERIMENTAL

Ganaxolone

Intervention Type DRUG

Interventions

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Ganaxolone

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

4 Months

Maximum Eligible Age

24 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Marinus Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

Mattel Children's Hospital at UCLA

Los Angeles, California, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Miami Children's Hospital, The Brain Institute

Miami, Florida, United States

Site Status

Child Neurology Center of Nrothwest Florida, P.A.

Pensacola, Florida, United States

Site Status

University of Chicago Comer Children's Hospital

Chicago, Illinois, United States

Site Status

Minnesota Epilepsy Group, P.A.

Saint Paul, Minnesota, United States

Site Status

Montefiore Medical Center- Albert Einstein College of Medicine

The Bronx, New York, United States

Site Status

Le Bonheur Children's Medical Center

Memphis, Tennessee, United States

Site Status

Dallas Pediatric Neurology Associates

Dallas, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

Virginia Commonwealth University Health Systems

Richmond, Virginia, United States

Site Status

Children's Hospital and Regional Medical Center

Seattle, Washington, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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Amend 5 (ROW)

Identifier Type: -

Identifier Source: secondary_id

Amend 6 (US)

Identifier Type: -

Identifier Source: secondary_id

1042-0501

Identifier Type: -

Identifier Source: org_study_id

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