Unverricht-Lundborg DiseasePhase 3NCT00357669
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults
A Phase 3 study testing Brivaracetam 25 mg and Brivaracetam 50 mg in people with Unverricht-Lundborg Disease, run by UCB Pharma SA at 13 sites in 7 countries.
Trial summary
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Unverricht-Lundborg Disease
- What is being tested?
- Brivaracetam 25 mg and Brivaracetam 50 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 13 sitesin 7 countries
- Study dates
- Nov 2006to Oct 2007
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
Italy4 sites
Study site
Bologna
Study site
Messina
Study site
Milan
Study site
Naples
About this study
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
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
- Conditions
- Unverricht-Lundborg Disease
- Being tested
- Brivaracetam 25 mg, Brivaracetam 50 mg, Placebo
- Keywords
- Unverricht-Lundborg disease, Baltic myoclonus, progressive myoclonic epilepsies, myoclonus, brivaracetam
- Medical subject headings
- Unverricht-Lundborg Syndrome, Myoclonic Epilepsies, Progressive, Myoclonus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Brivaracetam 25 mgDrug
Also called ucb34714
- Active Substance: Brivaracetam
- Pharmaceutical Form: Tablet
- Concentration: 25 mg
- Route of Administration: Oral use
Brivaracetam 50 mgDrug
Also called ucb34714
- Active Substance: Brivaracetam
- Pharmaceutical Form: Tablet
- Concentration: 50 mg
- Route of Administration: Oral use
PlaceboOther
- Active Substance: Placebo Pharmaceutical Form: Tablet
- Concentration: 25 mg and 50 mg
- Route of Administration: Oral use
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Other: Placebo |
| Brivaracetam 50 mg/day | Experimental | BRV 50 mg/day Receives: Drug: Brivaracetam 25 mg; Drug: Brivaracetam 50 mg |
| Brivaracetam 150 mg/day | Experimental | BRV 150 mg/day Receives: Drug: Brivaracetam 25 mg; Drug: Brivaracetam 50 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent reduction from baseline on the Action Myoclonus score (Unified Myoclonus Rating Scale (UMRS) Section 4) at the end of the Treatment Period
Measured: End of treatment period (Week 14 or early discontinuation visit)
Secondary outcomes (4)
Percent reduction from baseline on the functional disability score (UMRS Section 5) at the end of the Treatment Period
Measured: End of treatment period (week 14 or early discontinuation visit)
Percent reduction from baseline on the stimulus sensitivity score (UMRS Section 3) at the end of the Treatment Period
Measured: End of treatment period (week 14 or early discontinuation visit)
Percent reduction from baseline on the myoclonus patient questionnaire (UMRS Section 1) at the end of the Treatment Period
Measured: End of treatment period (week 14 or early discontinuation visit)
Global Evaluation Scale by Investigator (I-GES) at the end of the Treatment Period
Measured: End of treatment period (week 14 or early discontinuation visit)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2006 (estimated)
- Study startNov 2006
- Primary completionOct 2007
- Study completionOct 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Unverricht-Lundborg DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Pharma SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Clinical Trial Call Center, Study director, +1 877 822 9493 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00357669
- Sponsor's protocol ID
- N01187
- Other IDs
- 2006-000169-12
- Status last verified
- May 2015
- First submitted
- 25 Jul 2006
- Last updated
- 18 May 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 33461041Kalviainen 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.
PubMed 26666500
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00357669
- Acronym / sponsor ID
- N01187
- Conditions
- Unverricht-Lundborg Disease
- Keywords
- Unverricht-Lundborg disease, Baltic myoclonus, progressive myoclonic epilepsies, myoclonus, brivaracetam
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- UCB Pharma SAIndustry (pharma, biotech, devices)
- Study start
- Nov 2006
- Primary completion
- Oct 2007 (actual)
- Study completion
- Oct 2007 (actual)
- First posted
- 27 Jul 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: Placebo |
| Brivaracetam 50 mg/day | Experimental | Drug: Brivaracetam 25 mg, Drug: Brivaracetam 50 mgBRV 50 mg/day |
| Brivaracetam 150 mg/day | Experimental | Drug: Brivaracetam 25 mg, Drug: Brivaracetam 50 mgBRV 150 mg/day |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (11 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Brivaracetam 25 mgAlso: ucb34714* Active Substance: Brivaracetam * Pharmaceutical Form: Tablet * Concentration: 25 mg * Route of Administration: Oral use | Drug | Brivaracetam 150 mg/day, Brivaracetam 50 mg/day |
| Brivaracetam 50 mgAlso: ucb34714* Active Substance: Brivaracetam * Pharmaceutical Form: Tablet * Concentration: 50 mg * Route of Administration: Oral use | Drug | Brivaracetam 150 mg/day, Brivaracetam 50 mg/day |
| Placebo* Active Substance: Placebo Pharmaceutical Form: Tablet * Concentration: 25 mg and 50 mg * Route of Administration: Oral use | Other | Placebo |
Primary outcome
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)
4 secondary outcomes
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)
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)
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)
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2015.
- Derived from this study: PubMed 33461041Ben-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.
- Derived from this study: PubMed 26666500Kalviainen 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.
Related trials for Unverricht-Lundborg Disease
RecruitingObservational study
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)
Progressive Myoclonus Epilepsy Type 1, EPM1
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Clinical Trial Call Center study director
+1 877 822 9493 (UCB)
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Brivaracetam 25 mg and Brivaracetam 50 mg in people with Unverricht-Lundborg Disease, run by UCB Pharma SA at 13 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Italy, Finland, France, Netherlands, Reunion and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00357669, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2015.