BlepharospasmPhase 3NCT00406367
IncobotulinumtoxinA (Xeomin) Versus Placebo in the Treatment of Blepharospasm
A Phase 3 study testing incobotulinumtoxinA (Xeomin) in people with Blepharospasm, run by Merz Pharmaceuticals GmbH at 2 sites in 2 countries.
Trial summary
Patients received one injection with incobotulinumtoxinA (Xeomin) or placebo at baseline. Thereafter, all patients who entered the Open-Label Extension Period (OLEX) received up to five injection sessions of incobotulinumtoxinA (Xeomin) during the OLEX period. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Blepharospasm
- What is being tested?
- incobotulinumtoxinA (Xeomin)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 109 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Oct 2006to Jul 2009
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Main Inclusion Criteria:
- Male or female pretreated outpatients between ages 18 and 80 years (inclusive)
- A clinical diagnosis of bilateral blepharospasm (BEB) characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
- A need for injection of Botulinum toxin (defined by a Jankovic Rating Scale (JRS) severity subscore >= 2)
- On a stable dose of other medications (if any) used for focal dystonia treatment (e.g. anticholinergics and benzodiazepines) for at least 3 months prior to and expected throughout the Main Period
- Source documentation of the last two consecutive injection sessions with BOTOX® and a stable satisfactory therapeutic response directly prior to trial entry
- At least 10 weeks must have been passed between the last injection with BOTOX® for BEB and Baseline
Main Exclusion Criteria:
- Atypical variant of BEB caused by inhibition of levator palpebrae muscle
- Myotomy or denervation surgery in the affected muscles (e.g. peripheral denervation and/or spinal cord stimulation)
- The previous two injections with BOTOX® with more than 50 Units per eye
- Hypersensitivity to human serum albumin, sucrose, or Botulinum toxin A
- Neuroleptic induced blepharospasm
- Diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease which might interfere with the trial
- Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to Baseline and during the trial
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: Prospective, Double-blind, Placebo-controlled, Randomized, Multi-center Trial With an Open-label Extension Period to Investigate the Efficacy and Safety of incobotulinumtoxinA (Xeomin) in the Treatment of Blepharospasm
Patients received one injection with incobotulinumtoxinA (Xeomin) or placebo at baseline. Thereafter, all patients who entered the Open-Label Extension Period (OLEX) received up to five injection sessions of incobotulinumtoxinA (Xeomin) during the OLEX period.
- Conditions
- Blepharospasm
- Being tested
- incobotulinumtoxinA (Xeomin), Placebo
- Medical subject headings
- Blepharospasm
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
incobotulinumtoxinA (Xeomin)Drug
incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, up to 50 Units per eye; Mode of administration: intramuscular injection
PlaceboDrug
Placebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection
| Study group | Role | What they receive |
|---|---|---|
| incobotulinumtoxinA (Xeomin) | Experimental | incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), up to 50 Units per eye; Open-Label Extension Period: up to 5 injections, up to 50 Units per eye per injection session; Mode of administration: intramuscular injection Receives: Drug: incobotulinumtoxinA (Xeomin) |
| Placebo | Placebo comparator | Placebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by a Blinded Independent Rater)
The Jankovic Rating Scale (JRS) is used for classification of the patient's individual symptoms of blepharospasm and for determination of the therapeutic efficacy of study medication. The JRS sumscore is the sum of the two components of the scale:
- JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity)
- JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Measured: Baseline, week 6
Secondary outcomes (3)
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by Subject Diary)
The Jankovic Rating Scale (JRS) is used for classification of the patient's individual symptoms of blepharospasm and for determination of the therapeutic efficacy of study medication. The JRS sumscore is the sum of the two components of the scale:
- JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity)
- JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Measured: Baseline, week 6
Blepharospasm Disability Index (BSDI) Change From Baseline in the BSDI at Week 6 After Injection
The Blepharospasm Disability Index is a scale for the assessment of impairment of specific activities of daily living caused by blepharospasm. The BSDI consists of six items (driving a vehicle; reading; watching TV; shopping; getting about on foot (walking); doing everyday activities), each ranges from 0 (=no impairment) to 4 (=no longer possible due to illness). The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Measured: Baseline, week 6
Patient Evaluation of Global Response (PEGR) at Final Visit
The PEGR is a descriptive subjective 9-point response scale ranging from "complete abolishment of signs and symptoms" (value=+4) down to "very marked worsening" (value=-4).
Measured: Final visit (up to week 20 after injection of the Main Period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2006 (estimated)
- Study startOct 2006
- Primary completionApr 2008
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for BlepharospasmStudies 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 109 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merz Pharmaceuticals GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joseph Jankovic, Prof., Study chair, Houston, Texas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00406367
- Sponsor's protocol ID
- MRZ 60201-0433
- Status last verified
- Mar 2013
- First submitted
- 30 Nov 2006
- Last updated
- 15 Mar 2013
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Jankovic J, Comella C, Hanschmann A, Grafe S. Efficacy and safety of incobotulinumtoxinA (NT 201, Xeomin) in the treatment of blepharospasm-a randomized trial. Mov Disord. 2011 Jul;26(8):1521-8. doi: 10.1002/mds.23658. Epub 2011 Apr 22.
PubMed 21520284Truong DD, Gollomp SM, Jankovic J, LeWitt PA, Marx M, Hanschmann A, Fernandez HH; Xeomin US Blepharospasm Study Group. Sustained efficacy and safety of repeated incobotulinumtoxinA (Xeomin((R))) injections in blepharospasm. J Neural Transm (Vienna). 2013 Sep;120(9):1345-53. doi: 10.1007/s00702-013-0998-9. Epub 2013 Feb 23.
PubMed 23435927
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Double-blind, Placebo-controlled, Randomized, Multi-center Trial With an Open-label Extension Period to Investigate the Efficacy and Safety of incobotulinumtoxinA (Xeomin) in the Treatment of Blepharospasm
- ClinicalTrials.gov ID
- NCT00406367
- Acronym / sponsor ID
- MRZ 60201-0433
- Conditions
- Blepharospasm
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 109 participants (actual)
- Lead sponsor
- Merz Pharmaceuticals GmbHIndustry (pharma, biotech, devices)
- Study start
- Oct 2006
- Primary completion
- Apr 2008 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 4 Dec 2006 (estimated)
- Results
- Posted 16 Nov 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients received one injection with incobotulinumtoxinA (Xeomin) or placebo at baseline. Thereafter, all patients who entered the Open-Label Extension Period (OLEX) received up to five injection sessions of incobotulinumtoxinA (Xeomin) during the OLEX period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| incobotulinumtoxinA (Xeomin) | Experimental | Drug: incobotulinumtoxinA (Xeomin)incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), up to 50 Units per eye; Open-Label Extension Period: up to 5 injections, up to 50 Units per eye per injection session; Mode of administration: intramuscular injection |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| incobotulinumtoxinA (Xeomin)incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, up to 50 Units per eye; Mode of administration: intramuscular injection | Drug | incobotulinumtoxinA (Xeomin) |
| PlaceboPlacebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection | Drug | Placebo |
Primary outcome
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by a Blinded Independent Rater)
Time frame: Baseline, week 6
The Jankovic Rating Scale (JRS) is used for classification of the patient's individual symptoms of blepharospasm and for determination of the therapeutic efficacy of study medication. The JRS sumscore is the sum of the two components of the scale: * JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity) * JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
3 secondary outcomes
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by Subject Diary)
Time frame: Baseline, week 6
Blepharospasm Disability Index (BSDI) Change From Baseline in the BSDI at Week 6 After Injection
Time frame: Baseline, week 6
Patient Evaluation of Global Response (PEGR) at Final Visit
Time frame: Final visit (up to week 20 after injection of the Main Period)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21520284Jankovic J, Comella C, Hanschmann A, Grafe S. Efficacy and safety of incobotulinumtoxinA (NT 201, Xeomin) in the treatment of blepharospasm-a randomized trial. Mov Disord. 2011 Jul;26(8):1521-8. doi: 10.1002/mds.23658. Epub 2011 Apr 22.
- Results publication: PubMed 23435927Truong DD, Gollomp SM, Jankovic J, LeWitt PA, Marx M, Hanschmann A, Fernandez HH; Xeomin US Blepharospasm Study Group. Sustained efficacy and safety of repeated incobotulinumtoxinA (Xeomin((R))) injections in blepharospasm. J Neural Transm (Vienna). 2013 Sep;120(9):1345-53. doi: 10.1007/s00702-013-0998-9. Epub 2013 Feb 23.
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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.
Joseph Jankovic, Prof. study chair
Houston, Texas
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing incobotulinumtoxinA (Xeomin) in people with Blepharospasm, run by Merz Pharmaceuticals GmbH at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 15 Mar 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Canada. 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?
Yes. Results are posted in the registry (first posted 16 Nov 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by a Blinded Independent Rater) (time frame: Baseline, week 6). 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 NCT00406367, 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 15 Mar 2013.