Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease (ULD) in Adolescents and Adults
Condition: Unverricht-Lundborg Disease
MeSH terms: Unverricht-Lundborg Syndrome
Intervention: Placebo; BRV 2.5 mg; BRV 25 mg; BRV 50 mg
Intervention description: Pharmaceutical Form: Tablet
Concentration: 2.5 mg, 25 mg and 50 mg
Route of Administration: Oral use; Pharmaceutical Form: Tablet
Concentration: 2.5 mg
Route of Administration: Oral use; Pharmaceutical Form: Tablet
Concentration: 25 mg
Route of Administration: Oral use; Pharmaceutical Form: Tablet
Concentration: 50 mg
Route of Administration: Oral use
Intervention MeSH terms: Brivaracetam
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 56.0
Sex: All
Minimum age: 16 Years
Healthy volunteers: No
Keywords: Unverricht-Lundborg Disease; Baltic Myoclonus; Progressive Myoclonic Epilepsies; Myoclonus; Brivaracetam

Summary:

      The study will compare the efficacy and safety of Brivaracetam with placebo in patients with
      Unverricht- Lundborg Disease (ULD).
    

Eligibility 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