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

Merz Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Bilateral Blepharospasm (BEB)

Treatments: Drug: IncobotulinumtoxinA (Xeomin), 35 Units; Drug: Placebo; Drug: IncobulinumtoxinA (Xeomin), 25 Units; Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units

Study type: Interventional

Funder types: Industry

Identifiers

NCT01896895

2012-004821-26 (EudraCT Number)

MRZ60201_3074_1

Details and patient eligibility

About

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.

Full description

Subjects to receive one injection with NT 201 or placebo at baseline of the placebo-controlled first cycle. Thereafter, all subjects entering the Open-Label Extension Period (OLEX) to receive a second injection of NT 201 (second injection cycle).

Enrollment: 61 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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61 participants in 3 patient groups, including a placebo group

IncobotulinumtoxinA (Xeomin) 25U per eye

Experimental group

Description:

Main Period: one injection session, 25 Units per eye. Open-Label Extension: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.

Treatment:

Drug: IncobulinumtoxinA (Xeomin), 25 Units

Drug: IncobotulinumtoxinA (Xeomin), 35 Units

IncobotulinumtoxinA (Xeomin) 12.5U per eye

Experimental group

Description:

Main Period: one injection session, 12.5 Units per eye. Open-Label Extension Period: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.

Treatment:

Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units

Drug: IncobotulinumtoxinA (Xeomin), 35 Units

Placebo

Placebo Comparator group

Description:

Main Period: Placebo to IncobotulinumtoxinA (Xeomin)(12.5 or 25U/eye), one injection session. Open-Label Extension: IncobotulinumtoxinA (Xeomin), one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.

Treatment:

Drug: Placebo

Drug: IncobotulinumtoxinA (Xeomin), 35 Units