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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
UCB Pharma ( UCB Pharma SA )
ClinicalTrials.gov Identifier:
NCT00357669
First received: July 25, 2006
Last updated: May 15, 2015
Last verified: May 2015
  Purpose

The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.


Condition Intervention Phase
Unverricht-Lundborg Disease
Drug: Brivaracetam 25 mg
Drug: Brivaracetam 50 mg
Other: Placebo
Phase 3

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
Official 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

Resource links provided by NLM:


Further study details as provided by UCB Pharma:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]

Enrollment: 50
Study Start Date: November 2006
Study Completion Date: October 2007
Primary Completion Date: October 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo Other: Placebo
  • Active Substance: Placebo Pharmaceutical Form: Tablet
  • Concentration: 25 mg and 50 mg
  • Route of Administration: Oral use
Experimental: Brivaracetam 50 mg/day
BRV 50 mg/day
Drug: Brivaracetam 25 mg
  • Active Substance: Brivaracetam
  • Pharmaceutical Form: Tablet
  • Concentration: 25 mg
  • Route of Administration: Oral use
Other Name: ucb34714
Drug: Brivaracetam 50 mg
  • Active Substance: Brivaracetam
  • Pharmaceutical Form: Tablet
  • Concentration: 50 mg
  • Route of Administration: Oral use
Other Name: ucb34714
Experimental: Brivaracetam 150 mg/day
BRV 150 mg/day
Drug: Brivaracetam 25 mg
  • Active Substance: Brivaracetam
  • Pharmaceutical Form: Tablet
  • Concentration: 25 mg
  • Route of Administration: Oral use
Other Name: ucb34714
Drug: Brivaracetam 50 mg
  • Active Substance: Brivaracetam
  • Pharmaceutical Form: Tablet
  • Concentration: 50 mg
  • Route of Administration: Oral use
Other Name: ucb34714

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00357669

Locations
Finland
Kuopio, Finland
Tampere, Finland
France
Marseille, France
Montpellier Cedex 5, France
Italy
Bologna, Italy
Messina, Italy
Milano, Italy
Napoli, Italy
Netherlands
Heemstede, Netherlands
Heeze, Netherlands
Réunion
St Pierre Cedex, Réunion
Sweden
Goteborg, Sweden
Tunisia
Tunis, Tunisia
Sponsors and Collaborators
UCB Pharma SA
Investigators
Study Director: UCB Clinical Trial Call Center +1 877 822 9493 (UCB)
  More Information

No publications provided

Responsible Party: UCB Pharma ( UCB Pharma SA )
ClinicalTrials.gov Identifier: NCT00357669     History of Changes
Other Study ID Numbers: N01187, 2006-000169-12
Study First Received: July 25, 2006
Last Updated: May 15, 2015
Health Authority: European Union: European Medicines Agency

Keywords provided by UCB Pharma:
Unverricht-Lundborg disease
Baltic myoclonus
progressive myoclonic epilepsies
myoclonus
brivaracetam

Additional relevant MeSH terms:
Unverricht-Lundborg Syndrome
Brain Diseases
Central Nervous System Diseases
Epilepsies, Myoclonic
Epilepsy
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Myoclonic Epilepsies, Progressive
Nervous System Diseases
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on May 27, 2015