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

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Merz Pharmaceuticals GmbH
Sponsor:
Information provided by (Responsible Party):
Merz Pharmaceuticals GmbH
ClinicalTrials.gov Identifier:
NCT01896895
First received: July 8, 2013
Last updated: October 28, 2014
Last verified: October 2014
  Purpose

This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blephorasm (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.


Condition Intervention Phase
Bilateral Blepharospasm (BEB)
Drug: IncobulinumtoxinA (Xeomin), 25 Units
Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units
Drug: Placebo
Drug: IncobotulinumtoxinA (Xeomin), 35 Units
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Prospective, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multi-center Study With an Open-label Extension Period to Investigate the Efficacy and Safety of Two Different Doses of NT 201 in Botulinum Toxin Treatment-naïve Subjects With Blepharospasm

Resource links provided by NLM:


Further study details as provided by Merz Pharmaceuticals GmbH:

Primary Outcome Measures:
  • Change from baseline in Jankovic rating scale (JRS) severity subscore determined at V4 (Week 6) [ Time Frame: Baseline to week 6 ] [ Designated as safety issue: No ]
    The Jankovic Rating Scale (JRS) is used for classification of the patient's individual symptoms of blepharospasm and for determination of the therapeutic efficacy of study medication. The JRS-Severity sub-score is used, which ranges from 0 (=absence of severity) to 4 (=maximum severity).


Secondary Outcome Measures:
  • Change from baseline in Blepharospasm Disability Index (BSDI) score at Visit 4 (week 6) [ Time Frame: Baseline to week 6 ] [ Designated as safety issue: No ]
    The Blepharospasm Disability Index is a disease specific scale for subject functional self assessment of impairment of specific activities of daily living caused by blepharospasm. The BSDI consists of six items (driving a vehicle; reading; watching TV; shopping; getting about on foot (walking); doing everyday activities), each ranges from 0 (=no impairment) to 4 (=no longer possible due to illness). The BSDI will be assessed before rating the JRS for unbiased information by subject

  • Patient Evaluation of Global Response [PEGR] at final visit of Main Period (V5 -up to week 20) [ Time Frame: Up to week 20 ] [ Designated as safety issue: No ]
    The PEGR is a descriptive subjective 9-point response scale ranging from "complete abolishment of signs and symptoms" (value=+4) down to "very marked worsening" (value=-4).


Estimated Enrollment: 60
Study Start Date: November 2013
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: IncobotulinumtoxinA (Xeomin) 25U per eye
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.
Drug: IncobulinumtoxinA (Xeomin), 25 Units
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 25 Units per eye.
Other Names:
  • NT 201
  • Xeomin
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins
Drug: IncobotulinumtoxinA (Xeomin), 35 Units

IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).

Open-Label Extension: up to 35 Units per eye.

Other Names:
  • NT 201
  • Xeomin
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins
Experimental: IncobotulinumtoxinA (Xeomin) 12.5U per eye
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.
Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 12.5 Units per eye.
Other Names:
  • NT 201
  • Xeomin
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins
Drug: IncobotulinumtoxinA (Xeomin), 35 Units

IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).

Open-Label Extension: up to 35 Units per eye.

Other Names:
  • NT 201
  • Xeomin
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins
Placebo Comparator: Placebo

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.

Drug: Placebo
Main Period: Placebo to IncobotulinumtoxinA (Xeomin), powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
Drug: IncobotulinumtoxinA (Xeomin), 35 Units

IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).

Open-Label Extension: up to 35 Units per eye.

Other Names:
  • NT 201
  • Xeomin
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins

Detailed 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).

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01896895

Contacts
Contact: Public Disclosure Manager clinicaltrials@merz.de

Locations
Greece
Merz Investigational Site #030001 Recruiting
Athens, Greece, 11526
Merz Investigational Site #030002 Recruiting
Athens, Greece, 11521
Merz Investigational Site #030004 Recruiting
Ioannina, Greece, 45500
Merz Investigational Site #030003 Withdrawn
Thessaloniki, Greece, 56429
Malaysia
Merz Investigational Site #060004 Recruiting
Kota Kinabalu, Sabah, Malaysia, 88586
Merz Investigational Site #060002 Recruiting
Kuala Lumpur, Malaysia, 56000
Merz Investigational Site #060006 Recruiting
Kuala Lumpur, Malaysia, 50586
Merz Investigational Site #060003 Recruiting
Selangor, Malaysia, 43000
Sri Lanka
Merz Investigational Site #094004 Withdrawn
Anuradhapura, Sri Lanka, 50000
Merz Investigational Site #094001 Recruiting
Colombo, Sri Lanka, 07
Merz Investigational Site #094003 Withdrawn
Colombo, Sri Lanka, 10
Merz Investigational Site #094005 Recruiting
Colombo, Sri Lanka, 10350
Merz Investigational Site #094006 Recruiting
Kurunegala, Sri Lanka, 60000
Merz Investigational Site #094002 Recruiting
Nugegoda, Sri Lanka, 10250
Sponsors and Collaborators
Merz Pharmaceuticals GmbH
Investigators
Study Director: Merz Medical Expert Merz Pharmaceuticals GmbH
  More Information

No publications provided

Responsible Party: Merz Pharmaceuticals GmbH
ClinicalTrials.gov Identifier: NCT01896895     History of Changes
Other Study ID Numbers: MRZ60201_3074_1, 2012-004821-26
Study First Received: July 8, 2013
Last Updated: October 28, 2014
Health Authority: Malaysia: Ministry of Health
Sri Lanka: Ministry of Healthcare & Nutrition
Greece: National Organization of Medicines
United States: Food and Drug Administration

Additional relevant MeSH terms:
Blepharospasm
Eye Diseases
Eyelid Diseases
Botulinum Toxins
Botulinum Toxins, Type A
Anti-Dyskinesia Agents
Central Nervous System Agents
Neuromuscular Agents
Peripheral Nervous System Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014