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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00368251
• Date of registration: 23/08/2006
• Prospective Registration: Yes
• Primary sponsor: UCB Pharma
• Public title: Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease (ULD) in Adolescents and Adults
• Scientific title: A Multicenter, 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: 56
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00368251
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Canada, Finland, Former Serbia and Montenegro, France, Israel, Russian Federation, Serbia, Tunisia, United States

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 CSTB gene-
Subjects with moderate to severe myoclonus documented by an Action Myoclonussum 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-
Male/female subjects from 16 years onwards. Subjects under 18 years may only be included
where legally permitted and ethically accepted

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: All

Health Condition(s) or Problem(s) studied

Unverricht-Lundborg Disease

Intervention(s)

Drug: BRV 50 mg

Drug: BRV 2.5 mg

Drug: BRV 25 mg

Other: Placebo

Primary Outcome(s)

Percent Change From Baseline to the End of Treatment Period on the Action Myoclonus Score (Unified Myoclonus Rating Scale (UMRS) Section 4) [Time Frame: From Baseline to End of Treatment Period (Week 14 or Early Discontinuation Visit)]

Secondary Outcome(s)

Percent Change From Baseline to the End of Treatment Period on the Functional Disability Score (Unified Myoclonus Rating Scale (UMRS) Section 5) [Time Frame: Baseline to End of Treatment Period (Week 14 or Early Discontinuation Visit)]

Global Evaluation Score (Investigator) at the End of Treatment Period [Time Frame: End of Treatment Period (Week 14 or Early Discontinuation Visit)]

Percent Change From Baseline to the End of Treatment Period on the Stimulus Sensitivity Score (Unified Myoclonus Rating Scale (UMRS) Section 3) [Time Frame: Baseline to End of Treatment Period (Week 14 or Early Discontinuation Visit)]

Percent Change From Baseline to the End of Treatment Period on the Myoclonus Patient Questionnaire (Unified Myoclonus Rating Scale (UMRS) Section 1) [Time Frame: Baseline to End of Treatment Period (Week 14 or Early Discontinuation Visit)]

Secondary ID(s)

2006-001536-46

N01236

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 13/04/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00368251