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

NCT ID: NCT00368251

Last Updated: 2023-05-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 56 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-11-30
• Study Completion Date: 2008-01-31

Brief Summary

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

Conditions

Unverricht-Lundborg Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

Placebo Placebo twice a day (bid), 14 weeks (2 week Up-Titration Period + 12 week Maintenance Period)

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

• Pharmaceutical Form: Tablet
• Concentration: 2.5 mg, 25 mg and 50 mg
• Route of Administration: Oral use

Brivaracetam 5 mg/day

Brivaracetam (BRV) 5 mg/day 5 mg twice a day (bid) using 2.5 mg tablets for 12 weeks (after 2 week Up- Titration Period)

Group Type: EXPERIMENTAL

BRV 2.5 mg

Intervention Type: DRUG

• Pharmaceutical Form: Tablet
• Concentration: 2.5 mg
• Route of Administration: Oral use

Brivaracetam 150 mg/day

Brivaracetam (BRV) 150 mg/day 150 mg twice a day (bid) using 25 mg and 50 mg tablets for 12 weeks (after 2 week Up-Titration Period)

Group Type: EXPERIMENTAL

BRV 25 mg

Intervention Type: DRUG

• Pharmaceutical Form: Tablet
• Concentration: 25 mg
• Route of Administration: Oral use

BRV 50 mg

Intervention Type: DRUG

• Pharmaceutical Form: Tablet
• Concentration: 50 mg
• Route of Administration: Oral use

Interventions

Placebo

• Pharmaceutical Form: Tablet
• Concentration: 2.5 mg, 25 mg and 50 mg
• Route of Administration: Oral use

Intervention Type: OTHER

BRV 2.5 mg

• Pharmaceutical Form: Tablet
• Concentration: 2.5 mg
• Route of Administration: Oral use

Intervention Type: DRUG

BRV 25 mg

• Pharmaceutical Form: Tablet
• Concentration: 25 mg
• Route of Administration: Oral use

Intervention Type: DRUG

BRV 50 mg

• Pharmaceutical Form: Tablet
• Concentration: 50 mg
• Route of Administration: Oral use

Intervention Type: DRUG

Other Intervention Names

ucb34714

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

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UCB Pharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

UCB Clinical Trial Call Center

Role: STUDY_DIRECTOR

+1 877 822 9493 (UCB)

Locations

135

San Francisco, California, United States

133

Gainesville, Florida, United States

132

New York, New York, United States

131

Charlottesville, Virginia, United States

151

Vancouver, British Columbia, Canada

150

Montreal, Quebec, Canada

152

Québec, Quebec, Canada

100

Helsinki, , Finland

122

Bron, , France

121

Lille, , France

120

Paris, , France

170

Tel Aviv, , Israel

141

Moscow, , Russia

142

Saint Petersburg, , Russia

143

Samara, , Russia

161

Belgrade, , Serbia

162

Belgrade, , Serbia

180

Manouba, , Tunisia

Countries

United States; Canada; Finland; France; Israel; Russia; Serbia; Tunisia

References

Ben-Menachem E, Baulac M, Hong SB, Cleveland JM, Reichel C, Schulz AL, Wagener G, Brandt C. Safety, tolerability, and efficacy of brivaracetam as adjunctive therapy in patients with focal seizures, generalized onset seizures, or Unverricht-Lundborg disease: An open-label, long-term follow-up trial. Epilepsy Res. 2021 Feb;170:106526. doi: 10.1016/j.eplepsyres.2020.106526. Epub 2020 Dec 4.

Reference Type: DERIVED

PMID: 33461041 (View on PubMed)

Kalviainen 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.

Reference Type: DERIVED

PMID: 26666500 (View on PubMed)

Related Links

https://www.briviact.com/briviact-PI.pdf?v=1479491757

Product Information

http://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm

FDA Safety Alerts and Recalls

Other Identifiers

2006-001536-46

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

N01236

Identifier Type: -

Identifier Source: org_study_id