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

Study Description

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Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Unverricht-Lundborg Disease	Drug: Brivaracetam 25 mg; Drug: Brivaracetam 50 mg; Other: Placebo	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	50 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Quadruple (Participant, Care Provider, 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
Study Start Date :	November 2006
Actual Primary Completion Date :	October 2007
Actual Study Completion Date :	October 2007

Arms and Interventions

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Arm	Intervention/treatment
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

Outcome Measures

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Primary Outcome Measures :

1. 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 Measures :

1. 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) ]
2. 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) ]
3. 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) ]
4. 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) ]

Eligibility Criteria

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Ages Eligible for Study:	16 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
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

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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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Kälviäinen R, Genton P, Andermann E, Andermann F, Magaudda A, Frucht SJ, Schlit AF, Gerard D, de la Loge C, von Rosenstiel P. Brivaracetam in Unverricht-Lundborg disease (EPM1): Results from two randomized, double-blind, placebo-controlled studies. Epilepsia. 2016 Feb;57(2):210-21. doi: 10.1111/epi.13275. Epub 2015 Dec 15.

Responsible Party:	UCB Pharma SA
ClinicalTrials.gov Identifier:	NCT00357669 History of Changes
Other Study ID Numbers:	N01187; 2006-000169-12 ( EudraCT Number )
First Posted:	July 27, 2006
Last Update Posted:	May 18, 2015
Last Verified:	May 2015

Keywords provided by UCB Pharma ( UCB Pharma SA ):

Unverricht-Lundborg disease; Baltic myoclonus; progressive myoclonic epilepsies; myoclonus; brivaracetam

Additional relevant MeSH terms:

Unverricht-Lundborg Syndrome; Myoclonic Epilepsies, Progressive; Epilepsies, Myoclonic; Epilepsy; Brain Diseases; Central Nervous System Diseases	Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Brivaracetam; Anticonvulsants