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Phase 3 N=61 Randomized Quadruple-blind Treatment

Efficacy and Safety Study of Botulinum Toxin Type A Against Placebo to Treat Abnormal Contraction or Twitch of the Eyelid

Bilateral Blepharospasm (BEB)

Enrolled (actual)
61
Serious AEs
3.0%
Results posted
Mar 2018
Primary outcome: Primary: Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4) — -0.6; -1.0; -1.8 score on a scale — p==0.0004

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
IncobulinumtoxinA (Xeomin), 25 Units (Drug); IncobotulinumtoxinA (Xeomin), 12.5 Units (Drug); Placebo (Drug); IncobotulinumtoxinA (Xeomin), 35 Units (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Merz Pharmaceuticals GmbH
Primary completion
Apr 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4)
-0.6; -1.0; -1.8 =0.0004 sig
SECONDARY
Double-blind MP: Change From Baseline in Blepharospasm Disability Index (BSDI) at Day 43 (Visit 4)
-0.4; -0.5; -0.7
SECONDARY
Double-blind MP: Patient Evaluation of Global Response (PEGR) at Final Visit (Day 43-Day 141)
1.3; 1.8; 2.2

Summary

This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blepharospasm (BEB) who are BTX treatment-naïve. In this study, BTX treatment-naïve subjects are defined as those who have not received BTX treatment within the last 12 months for the treatment of BEB. This definition aims to avoid bias by comparison of treatment effects in the subject's assessments. Furthermore, this study will substantiate the existing efficacy and safety database for the indication BEB.

Eligibility Criteria

Inclusion Criteria

  • Male or female out-patients age ≥ 18 and ≤ 80 years.
  • A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of orbicular oculi muscles.
  • A need for injection of BTX defined as a Jankovic Rating Scale [JRS] severity subscore ≥ 2.
  • Treatment-naïve subject defined as at least 12 months without BTX of any serotype for the treatment of BEB before administration of IP.

Exclusion Criteria

  • Subject with any previous unsuccessful treatment with BTX of any serotype for the treatment of BEB.
  • Atypical variant of BEB (e.g., apraxia of the eyelid opening) caused by inhibition of levator palpebrae muscle.
  • Neuroleptic-induced blepharospasm.
  • Myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation, spinal cord stimulation) and surgery in the upper face.
  • Generalized disorders of muscles activity (e.g., myasthenia gravis in particular ocularis, Lambert-Eaton-Syndrome, amyotrophic lateral sclerosis) or any other significant neuromuscular dysfunction which might interfere with the study.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01896895). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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