A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
A Double-blind, Placebo-controlled, Dose-ranging Clinical Study to Evaluate the Safety, Tolerability, and Antiepileptic Activity of Ganaxolone in Treatment of Patients With Infantile Spasms
1 other identifier
interventional
57
1 country
15
Brief Summary
The study consists of cohorts where participants are randomized, in a 2:1 ratio, to 1 of 2 sequences, A and B. In each cohort, Sequence A, comprised of participants, who will receive ascending doses of ganaxolone and ascending doses of placebo. Sequence B, comprised of participants, who will receive ascending doses of placebo and ascending doses of ganaxolone. The dosing level in each subsequent cohort will be based upon experience gained from previous cohorts.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jan 2007
Shorter than P25 for phase_2
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2007
CompletedFirst Submitted
Initial submission to the registry
February 27, 2007
CompletedFirst Posted
Study publicly available on registry
March 1, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2008
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2008
CompletedResults Posted
Study results publicly available
June 2, 2023
CompletedJune 2, 2023
May 1, 2023
1.3 years
February 27, 2007
August 18, 2022
May 5, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Frequency of Spasm Clusters at Day 10
Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG) at Day 10. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment before study drug infusion.
Baseline (Day 0) and Day 10
Secondary Outcomes (8)
Change From Baseline in Frequency of Spasm Clusters at Day 20
Baseline (Day 0) and Day 20
Number of Participants With Absence of Hypsarrhythmia
Day 10 and Day 20
Number of Participants With Change in Clinical Status on the Investigator's Global Assessment
Baseline (Day 0), Day 10 and Day 20
Number of Participants With Change in Clinical Status on Caregiver's Global Assessment
Baseline (Day 0), Day10 and Day 20
Number of Participants With Spasm-free Durations
Day 10 and Day 20
- +3 more secondary outcomes
Study Arms (2)
ganaxolone
EXPERIMENTALganaxolone
non-active drug
PLACEBO COMPARATORplacebo
Interventions
Eligibility Criteria
You may qualify if:
- Be diagnosed with IS (regardless of etiology- except for a progressive neurologic illness). 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 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 or fewer AEDs.
- If being treated with concomitant AEDs
- Current AEDs have been at a constant daily dose for at least 2 weeks; Note: Subjects with minor dose adjustments may be allowed to enter the study after shorter periods after detailed discussion with the medical monitor.
- Have a stable clinical response/plateau for at least 2 weeks
- Are able to continue treatment with no more than 2 concomitant AEDs (ACTH, corticosteroids, felbamate, and vigabatrin are not allowed concomitantly).
- A ketogenic diet is permitted if it can be maintained for the duration of the study.
- Be a male or female, 4 to 24 months of age (inclusive)
- Have a Parent/Guardian who is properly informed of the nature and risks of the clinical study, who is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study
- Be able to participate for the full term of the clinical study.
You may not qualify if:
- Treatment with corticosteroids, ACTH, vigabatrin, felbamate, or any AED not approved by Regulatory Agencies, 2 weeks prior to randomization.
- Treatment with more than two AEDs at baseline.
- 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 imaging (MRI).
- Have any disease or condition (medical or surgical) at screening that might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, 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 aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin greater than four times the upper limit of normal (ULN) or 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 randomization.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Mattel Children's Hospital at UCLA
Los Angeles, California, 90095, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Miami Children's Hospital, The Brain Institute
Miami, Florida, 33155, United States
Child Neurology Care Center of Northwest Florida
Pensacola, Florida, 32504, United States
Child Neurology Center of Northwest Florida
Pensacola, Florida, United States
University of Chicago Comer Children's Hospital
Chicago, Illinois, 60637, United States
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, 55102, United States
Montefiore Medical Center- Albert Einstein College of Medicine
The Bronx, New York, 10467, United States
Le Bonheur Children's Medical Center
Memphis, Tennessee, 38105, United States
Dallas Pediatric Neurology Associates
Dallas, Texas, 75230, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Virginia Commonwealth University Health System
Richmond, Virginia, 23298, United States
Children's Hospital and Regional Medical Center
Seattle, Washington, 98105, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53201, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Marinus
- Organization
- Marinus Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- 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
May 1, 2008
Study Completion
May 1, 2008
Last Updated
June 2, 2023
Results First Posted
June 2, 2023
Record last verified: 2023-05