A controlled clinical trial to look at the effects of treatment with rufinamide on the body, thought process development and its safety in children aged 1 to 4 years with poorly controlled Lennox Gastaut Syndrome.

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2010-023505-36-FR
• Date of registration: 19/05/2011
• Prospective Registration: Yes
• Primary sponsor: Eisai Ltd
• Public title: A controlled clinical trial to look at the effects of treatment with rufinamide on the body, thought process development and its safety in children aged 1 to 4 years with poorly controlled Lennox Gastaut Syndrome.
• Scientific title: A Multicenter, Randomized, Controlled, Open-label Study to Evaluate the Cognitive Development Effects and Safety, and Pharmacokinetics of Adjunctive Rufinamide Treatment in Pediatric Subjects 1 to less than 4 years of age with Inadequately Controlled Lennox-Gastaut Syndrome
• Target sample size: 75
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-023505-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: yes Other specify the comparator: Investigators Choice of Approved AED Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

France, Greece, India, Italy, United States

Contacts

• Name: Medical Information
• Affiliation: Eisai Ltd

• Name: Medical Information
• Affiliation: Eisai Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
Age = 1 and < 4 years

Clinical diagnosis of LGS at screening, which might include the presence of a slow background EEG rhythm, slow spikes-waves pattern (less than 3 Hz), the presence of polyspikes; care should be taken not to include benign myoclonic epilepsy of infancy, subjects with a diagnosis of atypical benign partial epilepsy (pseudo-Lennox syndrome), or continuous spike-waves of slow sleep (CSWS)

On a fixed dose of one to three concomitant regionally approved antiepileptic drugs (AEDs) for a minimum of 8 weeks prior to randomization with an inadequate response to treatment

Consistent seizure documentation (i.e., no uncertainty of the presence of seizures) and AED treatment documentation during the 8 week prerandomization period

Written informed consent provided by parent(s)/legal representative(s)

Are able to comply with all aspects of the protocol
Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
Familial short QT syndrome

Prior treatment with rufinamide

Evidence of clinically significant disease (e.g., cardiac, respiratory, gastrointestinal,
renal disease) that in the opinion of the investigator(s) could affect the subject’s safety or study conduct

Subject with hypersensivity to rufinamide and /or triazole or any of the excipients

Any history of or concomitant medical condition that, in the opinion of the
Investigator, would compromise the subject’s ability to safely complete the study

Scheduled for surgery during the projected course of the study.


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

Health Condition(s) or Problem(s) studied

Lennox Gastaut Syndrome
MedDRA version: 13.1 Level: PT Classification code 10048816 Term: Lennox-Gastaut syndrome System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: rufinamide oral suspension
Product Code: rufinamide oral suspension
Pharmaceutical Form: Oral suspension
INN or Proposed INN: RUFINAMIDE
CAS Number: 106308-44-5
Current Sponsor code: E2080
Other descriptive name: Rufinamide oral suspension
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-

Trade Name: Investigators choice of approved AEDs
Pharmaceutical Form:
INN or Proposed INN: Investigators Choice of Approved AED
CAS Number: N/A
Current Sponsor code: N/A
Other descriptive name: N/A

Primary Outcome(s)

Secondary Objective: N/A

Primary end point(s): Primary efficacy variable:

Change from baseline in CBCL Total Problems Score to the end of the 2-year (106 weeks) treatment period

Main Objective: To compare the effect of 2 drug regimens consisting of either rufinamide or any other approved AED of the investigator’s choice as an add-on to the subject’s existing regimen of 1-3 AEDs on the overall safety and tolerability of rufinamide in subjects aged 1 to less than 4 years of age with inadequately controlled LGS

To characterize the age group specific pharmacokinetics of rufinamide in a pediatric population, 1 to less than 4 years of age, with inadequately controlled LGS, using
the population approach

To evaluate the effect of rufinamide as adjunctive treatment on the cognitive development and behavioral effects in a pediatric population, 1 to less than 4 years of age, with inadequately controlled LGS.

Timepoint(s) of evaluation of this end point: Baseline to end of 2 year treatment period. Physical and neurological examinations (the latter only if possible) will be
performed at screening, baseline, Visits 3, 4, 5, 7, 10, 12, 13, 14, and follow up/final visit.

Secondary Outcome(s)

Secondary end point(s): N/A

Timepoint(s) of evaluation of this end point: N/A

Secondary ID(s)

E2080-G000-303

Source(s) of Monetary Support

Eisai Ltd