Comparison of Two Botulinum Type A Products in the Treatment of Blepharospasm

Study Overview
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Brief Summary
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This pilot study estimates the treatment effects of two different types of botulinum toxin type A in the treatment of Blepharospasm. Blepharospasm is characterised by excessive contraction of the muscles around the eye and can lead to repetitive blinking or sustained closure of the eyelids.

Conditions
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Blepharospasm

Intervention / Treatment
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* Biological: Botulinum Toxin Type A 900kDa
* Biological: Botulinum Toxin Type A 150kDa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with benign essential blepharospasm Received ≥20U/eye of BOTOX® for at least one visit prior to study entry and required, in the investigators opinion, the same dose at the study injection visit. Combined Jankovic Rating Score of >2 Exclusion Criteria: Female subjects who were pregnant, breastfeeding, or who were of childbirth potential and not practicing birth control. Profound atrophy of the muscles in the target area(s) of injection. Myasthenia Gravis, Lambert-Eaton Syndrome, Amyotrophic Lateral Sclerosis or any other disease that might interfere with neuromuscular function. Known significantly impaired renal and/or hepatic function

Ages Eligible for Study
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Minimum Age: 18 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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br> | Biological: Botulinum Toxin Type A 900kDa<br>* 6 to 16 injections, with maximum of 21, at a dose of ≥20U/eye (≥40U total dose)<br>* Other names: BOTOX®;|
| Active Comparator: 2<br> | Biological: Botulinum Toxin Type A 150kDa<br>* 6 to 16 injections, with a maximum of 21, at a dose of ≥20U/eye (≥40U total dose)<br>* Other names: Xeomin®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Week 4 in Blepharospasm Disability Index | Blepharospasm Disability Index is a validated 5-point scale (0-4) with six items (e.g., reading, driving a vehicle). 0 - no impairment, 1 - mild impairment, 2 - moderate impairment, 3 - severe impairment, 4 - not possible due to disease, N/A - Not applicable. The total score ranged from 0 (no impairment) to 24 (not possible due to disease). A negative change from baseline indicated improvement. | Baseline to Week 4 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Week 8 in Blepharospasm Disability Index | Blepharospasm Disability Index is a validated 5-point (0-4) scale with six items (e.g., reading, driving a vehicle). 0 - no impairment, 1 - mild impairment, 2 - moderate impairment, 3 - severe impairment, 4 - not possible due to disease, N/A - Not applicable. The total score ranged from 0 (no impairment) to 24 (not possible due to disease). A negative change from baseline indicated improvement. | Baseline to Week 8 | 
| Change From Baseline to Week 4 and Week 8 in Total Jankovic Rating Scale (JRS) (Severity and Frequency Measured on a Scale of 0-4) | Jankovic Rating Scale Severity: 0 - None; 1 - Minimal; 2 - Mild; 3 - Moderate; 4 - Severe. Frequency: 0 - None; 1 - Slight increase; 2 - Fluttering duration less than 1 second; 3 - Spasm greater than 1 second and eyes open > 50% of waking time; 4 - Functionally blind. The range of the total score was from 0 (None) to 8 (Severe and Functionally Blind). A negative change from baseline indicated improvement. | Baseline to Week 4 and Week 8 | 
| Changes From Baseline to Week 4 and Week 8 in Patient Global Assessment (PGA) Score | Subjective satisfaction rating: -4: marked worsening, -3: moderate worsening, -2: marked worsening in symptoms, -1: mild worsening in symptoms, 0: no effect +1: mild improvement in symptoms, +2: moderate improvement in symptoms, +3: mild improvement, +4: marked improvement. A positive change from baseline indicated improvement. | Baseline to Week 4 and 8 | 
| Duration of Action | Median Duration for decision to reinject | Interval between initial injection (Week 0) and final visit (Week 11 through Week 14) |