BlepharospasmPhase 2NCT00507637
Blepharospasm Short Interval
A Phase 2 study testing NT 201 in people with Blepharospasm, run by Merz Pharmaceuticals GmbH at 1 site in Germany.
Trial summary
The objective of this Phase II study is to show that pre-treated patients with BEB with the need of shortened injection intervals achieve an improved and stable quality of life level if they receive repeated NT 201 injections at short injection intervals. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Blepharospasm
- What is being tested?
- NT 201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteGermany
- Study dates
- Aug 2007to Feb 2008
- Phase
- Phase 2
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
- Medical need for treatment with shortened injection intervals (< 12 weeks) confirmed by the patient and investigator
- Blepharospasm Disability Index (BSDI) at baseline visit before injection of > / = 1,6
Who cannot join
- Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid)
- Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert-Eaton syndrome), as well as amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial
- The previous three injections with Botulinum toxin Type A with more than 50 Units [U] per eye per injection session
- Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to baseline and during the trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective, Open-label, Single-arm, Single-center Trial to Investigate the Tolerability of NT 201 and Quality of Life of Patients in the Treatment of Blepharospasm With Shortened Injection Intervals
The objective of this Phase II study is to show that pre-treated patients with BEB with the need of shortened injection intervals achieve an improved and stable quality of life level if they receive repeated NT 201 injections at short injection intervals. In addition, the tolerability of repeated shortened NT 201 administrations in patients with BEB including the development of neutralizing antibodies will be investigated.
- Conditions
- Blepharospasm
- Being tested
- NT 201
- Medical subject headings
- Blepharospasm
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NT 201Drug
Also called Xeomin®, IncobotulinumtoxinA, "Botulinum toxin type A (150 kiloDalton), free from complexing proteins"
Injection of up to 50 Units NT 201 (incobotulinumtoxinA/Xeomin®) per eye and injection visit (8 injections visits in total).
| Study group | Role | What they receive |
|---|---|---|
| NT 201 (IncobotulinumtoxinA/Xeomin®) | Experimental | Not described Receives: Drug: NT 201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
The BSDI includes 6 items to be assessed by a 5-point scoring (i.e., 0-4 points per item) ranging from "no impairment, "slight impairment", "moderate impairment", "severe impairment" to "no longer possible due to my illness". The 6 items are "Driving a vehicle", "Reading", "Watching TV", "Shopping", "Walking" and "Doing everyday activities".
Measured: From baseline up to week 62. Follow up visits include 8 injection visits (V2 to V8) with intervals of at least 6 weeks between injections.
Secondary outcomes (2)
Change in Jankovic Rating Scale (JRS) score from baseline over time.
The JRS ranges from 0-8 points and consists of the two items "Severity" and "Frequency" with five rating categories each (0-4 points).
Measured: From baseline up to week 62
Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit
The PEGR scale is a descriptive subjective 9-point response self-rating scale ranging from "complete abolishment of signs and symptoms" (+4) down to "very marked worsening" (-4).
Measured: Week 6 up to week 62
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2007 (estimated)
- Study startAug 2007
- Primary completionFeb 2008
- Study completionFeb 2008
Why it stopped: primary efficacy endpoint not attainable.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merz Pharmaceuticals GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bettina Wabbels, MD, Principal investigator, Universitaets Augenklinik Bonn
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00507637
- Sponsor's protocol ID
- MRZ 60201-0703/1
- Other IDs
- 2007-000697-23
- Status last verified
- Feb 2012
- First submitted
- 25 Jul 2007
- Last updated
- 6 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Open-label, Single-arm, Single-center Trial to Investigate the Tolerability of NT 201 and Quality of Life of Patients in the Treatment of Blepharospasm With Shortened Injection Intervals
- ClinicalTrials.gov ID
- NCT00507637
- Acronym / sponsor ID
- MRZ 60201-0703/1
- Conditions
- Blepharospasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: primary efficacy endpoint not attainable.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Merz Pharmaceuticals GmbHIndustry (pharma, biotech, devices)
- Study start
- Aug 2007
- Primary completion
- Feb 2008 (actual)
- Study completion
- Feb 2008 (actual)
- First posted
- 26 Jul 2007 (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 objective of this Phase II study is to show that pre-treated patients with BEB with the need of shortened injection intervals achieve an improved and stable quality of life level if they receive repeated NT 201 injections at short injection intervals. In addition, the tolerability of repeated shortened NT 201 administrations in patients with BEB including the development of neutralizing antibodies will be investigated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NT 201 (IncobotulinumtoxinA/Xeomin®) | Experimental | Drug: NT 201 |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
- Medical need for treatment with shortened injection intervals (< 12 weeks) confirmed by the patient and investigator
- Blepharospasm Disability Index (BSDI) at baseline visit before injection of > / = 1,6
Key exclusion criteria (4 listed)
- Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid)
- Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert-Eaton syndrome), as well as amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial
- The previous three injections with Botulinum toxin Type A with more than 50 Units [U] per eye per injection session
- Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to baseline and during the trial
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 |
|---|---|---|
| NT 201Also: Xeomin®, IncobotulinumtoxinA, "Botulinum toxin type A (150 kiloDalton), free from complexing proteins"Injection of up to 50 Units NT 201 (incobotulinumtoxinA/Xeomin®) per eye and injection visit (8 injections visits in total). | Drug | NT 201 (IncobotulinumtoxinA/Xeomin®) |
Primary outcome
The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
Time frame: From baseline up to week 62. Follow up visits include 8 injection visits (V2 to V8) with intervals of at least 6 weeks between injections.
The BSDI includes 6 items to be assessed by a 5-point scoring (i.e., 0-4 points per item) ranging from "no impairment, "slight impairment", "moderate impairment", "severe impairment" to "no longer possible due to my illness". The 6 items are "Driving a vehicle", "Reading", "Watching TV", "Shopping", "Walking" and "Doing everyday activities".
2 secondary outcomes
Change in Jankovic Rating Scale (JRS) score from baseline over time.
Time frame: From baseline up to week 62
Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit
Time frame: Week 6 up to week 62
Results and safety
The study was stopped early (reason given: primary efficacy endpoint not attainable.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2012.
Related trials for Blepharospasm
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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.
Bettina Wabbels, MD principal investigator
Universitaets Augenklinik Bonn
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NT 201 in people with Blepharospasm, run by Merz Pharmaceuticals GmbH at 1 site in Germany.
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 3 inclusion and 4 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: stopped early, as of the sponsor's last update on 6 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: primary efficacy endpoint not attainable.). Results are not available in the source record.
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?
The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline. (time frame: From baseline up to week 62. Follow up visits include 8 injection visits (V2 to V8) with intervals of at least 6 weeks between injections.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Feb 2012.