An open-label clinical study to evaluate the safety and antiepileptic activity of ganaxolaone in treatment of patients diagnosed with infantile spasms.

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2006-004286-33-CZ
• Date of registration: 30/03/2007
• Prospective Registration: Yes
• Primary sponsor: Marinus Pharmceuticals, Inc.
• Public title: An open-label clinical study to evaluate the safety and antiepileptic activity of ganaxolaone in treatment of patients diagnosed with infantile spasms.
• Scientific title: An open-label clinical study to evaluate the safety and antiepileptic activity of ganaxolaone in treatment of patients diagnosed with infantile spasms.
• Date of first enrolment: 11/05/2007
• Target sample size: 54
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2006-004286-33
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Czech Republic

Key inclusion & exclusion criteria

Inclusion criteria:
a) 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.
b) Be diagnosed with IS (regardless of etiology- except for a progressive neurologic illness, see Protocol Section 9.3.3 Exclusion Criteria). 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).
c) Have a 24-hour vEEG recording confirming the diagnosis of IS.
d) Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.
e) Have been previously treated with 3 AEDs or fewer.
f) 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.

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:
a) Current treatment with more than 2 concomitant AEDs.
b) 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.
c) 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.
d) 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.
e) History of recurrent status epilepticus.
f) Have been exposed to any other investigational drug within 30 days prior to enrollment.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Infantile spasms
MedDRA version: 9.1 Level: LLT Classification code 10021750 Term: Infantile spasms

Intervention(s)

Product Name: Ganaxolone
Product Code: SPT3162
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
Concentration type: range
Concentration number: 3 mg/kg-18 mg/kg

Primary Outcome(s)

Main Objective: To evaluate the safety and antiepileptic activity of long-term treatment with ganaxolone in subjects with Infantile spasms (IS) who have completed the double blind controlled trial (Protocol 1042-0500).

Primary end point(s): The primary efficacy variable is the number of subjects who are free of spasms measured by 24-hour vEEG read at the site at the Visit 7 (Week 32).

Secondary Objective: To provide further information about the therapeutic dose of ganaxolone in subjects with IS and drug-drug interactions between ganaxolone and other AEDs in infants with IS.

Secondary ID(s)

1042-0501