Infantile spasms MedDRA version: 9.1 Level: LLT Classification code 10021750 Term: Infantile spasms
Conditions
Interventions
Product Name: Ganaxolone
Pharmaceutical Form: Oral suspension
CAS Number: 38398-32-2
Current Sponsor code: CCD 1042
Other descriptive name: Ganaxolone
Concentration unit: mg/kg milligram(s)/kilogram
C
Sponsors
Marinus Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects accepted for this clinical study must: (a) Be diagnosed with IS (regardless of etiology- except for a progressive neurologic illness, see protocol Section 9.3.3 Exclusion Criteria c). 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). (b) Have a vEEG recording confirming the diagnosis of IS. (c) Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS. (d) Have been previously treated with 3 or fewer AEDs. (e) If being treated with concomitant AEDs (also see protocol Section 9.2.2) 1) 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. 2) Have a stable clinical response/plateau for at least 2 weeks 3) Are able to continue treatment with no more than 2 concomitant AEDs (ACTH, corticosteroids, felbamate, and vigabatrin are not allowed concomitantly). 4) A ketogenic diet is permitted if it can be maintained for the duration of the study. (f) Be a male or female, 4 to 24 months of age (inclusive). (g) 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. (h) Be able to participate for the full term of the clinical study. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Any of the following conditions are cause for exclusion from the clinical study: (a) Treatment with corticosteriods, ACTH, vigabtrin, felbamate, or any AED not approved by Regulatory Agencies 2 weeks prior to randomization. Up to 0.5 mg/kg/day of prednisone (or equivalent) is allowed at baseline if subjects are being tapered off prednisone following ACTH or corticosteroid treatment. Subjects being tapered on prednisone (up to 0.5 mg/kg/day at baseline) may be randomized. (b) Treatment with more than two AEDs at baseline. (c) 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). (d) 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. (e) 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. (f) History of recurrent status epilepticus. (g) Have been exposed to any other investigational drug within 30 days prior to randomization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety, tolerability, and antiepileptic activity of variable ascending doses of ganaxolone and to determine the therapeutic dose of ganaxolone in subjects (ages 4 to 24 months) with infantile spasms (IS).;Secondary Objective: To determine the PK profile of ganaxolone in subjects 4 to 24 months of age.;Primary end point(s): The primary efficacy variable is the spasm frequency as measured from 24-hour vEEG by a central reader on Visit 5. | — |
Countries
Czech Republic
Outcome results
None listed