Blepharospasm Short Interval

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Blepharospasm

Intervention / Treatment
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* Drug: NT 201


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NT 201 (IncobotulinumtoxinA/Xeomin®)<br> | Drug: NT 201<br>* Injection of up to 50 Units NT 201 (incobotulinumtoxinA/Xeomin®) per eye and injection visit (8 injections visits in total).<br>* Other names: Botulinum toxin type A (150 kiloDalton), free from complexing proteins;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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). | 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). | Week 6 up to week 62 |