Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults

• Register: ClinicalTrials.gov
• Last refreshed on: 25 May 2015
• Main ID: NCT00357669
• Date of registration: 25/07/2006
• Prospective Registration: Yes
• Primary sponsor: UCB Pharma SA
• Public title: Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
• Scientific title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Study to Evaluate the Efficacy and Safety of Brivaracetam Used as Adjunctive Treatment for 12 Weeks in Adolescent and Adult Patients (=16 Years) With Genetically Ascertained Unverricht-Lundborg Disease
• Date of first enrolment: November 2006
• Target sample size: 50
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00357669
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Finland, France, Italy, Netherlands, Réunion, Sweden, Tunisia

Contacts

• Name: UCB Clinical Trial Call Center
• Affiliation: [phone] (UCB)

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subjects with diagnosed Unverricht-Lundborg disease (ULD) ascertained by appropriate
genetic testing for a homozygous or compound heterozygous mutation in the Cystatin B
(CSTB) gene

- Subjects with moderate to severe myoclonus documented by an Action Myoclonus sum
score of = 30 (evaluation by investigator)

- Subjects currently being or having been treated with clonazepam up to the maximum
recommended daily dose of 20 mg or up to their individual optimal dose as assessed by
the investigator

- Subjects currently being or having been treated with valproate up to the maximum
recommended daily dose 60 mg/kg or serum levels of 100 mcg/ml or up to their
individual optimal dose as specified by the investigator

Exclusion Criteria:

- Subjects currently on felbamate or having been on felbamate within less than 18
months prior to Visit 1

- Subjects currently treated with phenytoin or having been on phenytoin in the last
month prior to Visit 1

- Subjects currently on vigabatrine. Subjects having been on vigabatrine if no visual
fields examination report available including standard static (Humphrey or Octopus)
or cinetic perimetry (Goldman)

- Subject taking any drug with possible central nervous system (CNS) effects

- Subjects taking any drug that may significantly influence the metabolism of BRV
(CYP2C or CYP3A potent inducers/inhibitors)

- Known clinically significant acute or chronic illness or illness which may impair
reliable participation in the trial, necessitate the use of medication not allowed by
protocol or represent a safety risk in the Investigator's opinion

- Subjects with history of severe adverse hematological reaction to any drug

- Impaired hepatic function: ALAT/SGPT, ASAT/SGOT, alkaline phosphatase, GGT value of
more than three times the upper limit of the reference range

- History of suicide attempt during the last 5 years

- Subject with suicidal ideations within the last year or at risk of suicide attempt
unless cleared by written confirmation from a psychiatrist and approved by the UCB
physician

- Ongoing psychiatric disorder other than mild controlled disorder



Age minimum: 16 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Unverricht-Lundborg Disease

Intervention(s)

Drug: Brivaracetam 25 mg

Drug: Brivaracetam 50 mg

Other: Placebo

Primary Outcome(s)

Percent reduction from baseline on the Action Myoclonus score (Unified Myoclonus Rating Scale (UMRS) Section 4) at the end of the Treatment Period [Time Frame: End of treatment period (Week 14 or early discontinuation visit)]

Secondary Outcome(s)

Percent reduction from baseline on the stimulus sensitivity score (UMRS Section 3) at the end of the Treatment Period [Time Frame: End of treatment period (week 14 or early discontinuation visit)]

Global Evaluation Scale by Investigator (I-GES) at the end of the Treatment Period [Time Frame: End of treatment period (week 14 or early discontinuation visit)]

Percent reduction from baseline on the myoclonus patient questionnaire (UMRS Section 1) at the end of the Treatment Period [Time Frame: End of treatment period (week 14 or early discontinuation visit)]

Percent reduction from baseline on the functional disability score (UMRS Section 5) at the end of the Treatment Period [Time Frame: End of treatment period (week 14 or early discontinuation visit)]

Secondary ID(s)

N01187

2006-000169-12

Source(s) of Monetary Support

Please refer to primary and secondary sponsors