Blepharospasm
Conditions
Brief 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. In addition, the tolerability of repeated shortened NT 201 administrations in patients with BEB including the development of neutralizing antibodies will be investigated.
Interventions
DRUGNT 201
Injection of up to 50 Units NT 201 (incobotulinumtoxinA/Xeomin®) per eye and injection visit (8 injections visits in total).
Sponsors
Merz Pharmaceuticals GmbH
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Jankovic Rating Scale (JRS) score from baseline over time. | From baseline up to week 62 | The JRS ranges from 0-8 points and consists of the two items Severity and Frequency with five rating categories each (0-4 points). |
| Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit | Week 6 up to week 62 | 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). |
Countries
Germany
Outcome results
None listed