Open-label Extension to Protocol 1042-0500

NCT00442104 | Terminated Phase 2 Results

• 3 other identifiers: 1042-0501Amend 5 (ROW)Amend 6 (US)
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 14

Brief Summary

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

Conditions

Infantile Spasms

Keywords

infantile spasms; anticonvulsant; pediatric epilepsy; West Syndrome; epileptic spasms

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Were Free of Spasms

  Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.

  Weeks 4 through Week 96

Secondary Outcomes (8)

• Change From Baseline in Frequency of Spasm Clusters

  Baseline and Week 4 through Week 32

• Change From Baseline in Frequency of Individual Spasm

  Baseline and Week 4 through Week 32

• Number of Participants With Change in Clinical Status on Caregiver's Global Assessment

  Week 4 through Week 32

• Number of Participants With Change in Clinical Status on the Investigator's Global Assessment

  Week 4 through Week 32

• Number of Participants With Spasm-free Durations

  Week 4 through Week 32

Study Arms (1)

ganaxolone

EXPERIMENTAL

Drug: Ganaxolone

Interventions

Ganaxolone DRUG

ganaxolone

Eligibility Criteria

• Age: 4 Months - 24 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead

Study Sites (14)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Mattel Children's Hospital at UCLA

Los Angeles, California, 90095, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Miami Children's Hospital, The Brain Institute

Miami, Florida, 33155, United States

Location

Child Neurology Center of Nrothwest Florida, P.A.

Pensacola, Florida, 32504, United States

Location

University of Chicago Comer Children's Hospital

Chicago, Illinois, 60637, United States

Location

Minnesota Epilepsy Group, P.A.

Saint Paul, Minnesota, 55102, United States

Location

Montefiore Medical Center- Albert Einstein College of Medicine

The Bronx, New York, 10467, United States

Location

Le Bonheur Children's Medical Center

Memphis, Tennessee, 38105, United States

Location

Dallas Pediatric Neurology Associates

Dallas, Texas, 75230, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Virginia Commonwealth University Health Systems

Richmond, Virginia, 23298, United States

Location

Children's Hospital and Regional Medical Center

Seattle, Washington, 98105, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53201, United States

Location

MeSH Terms

Conditions

Spasms, Infantile

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Limitations and Caveats

Seizure-free information is not available

Results Point of Contact

• Title: Marinus
• Organization: Marinus Pharmaceuticals, Inc.

clinicaltrials@marinuspharma.com

484-801-4670

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2007
• First Posted: March 1, 2007
• Study Start: January 1, 2007
• Primary Completion: March 1, 2009
• Study Completion: March 1, 2009
• Last Updated: May 28, 2024
• Results First Posted: June 2, 2023

Record last verified: 2024-05

Locations

US (14)