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IncobotulinumtoxinA (Xeomin) Versus Placebo in the Treatment of Blepharospasm

Primary Purpose

Blepharospasm

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
incobotulinumtoxinA (Xeomin)
Placebo
Sponsored by
Merz Pharmaceuticals GmbH
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Blepharospasm

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Main Inclusion Criteria:

  • Male or female pretreated outpatients between ages 18 and 80 years (inclusive)
  • A clinical diagnosis of bilateral blepharospasm (BEB) characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
  • A need for injection of Botulinum toxin (defined by a Jankovic Rating Scale (JRS) severity subscore >= 2)
  • On a stable dose of other medications (if any) used for focal dystonia treatment (e.g. anticholinergics and benzodiazepines) for at least 3 months prior to and expected throughout the Main Period
  • Source documentation of the last two consecutive injection sessions with BOTOX® and a stable satisfactory therapeutic response directly prior to trial entry
  • At least 10 weeks must have been passed between the last injection with BOTOX® for BEB and Baseline

Main Exclusion Criteria:

  • Atypical variant of BEB caused by inhibition of levator palpebrae muscle
  • Myotomy or denervation surgery in the affected muscles (e.g. peripheral denervation and/or spinal cord stimulation)
  • The previous two injections with BOTOX® with more than 50 Units per eye
  • Hypersensitivity to human serum albumin, sucrose, or Botulinum toxin A
  • Neuroleptic induced blepharospasm
  • Diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease which might interfere with the trial
  • Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to Baseline and during the trial

Sites / Locations

  • Baylor College of Medicine
  • David King, MD - Private Practice

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

incobotulinumtoxinA (Xeomin)

Placebo

Arm Description

incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), up to 50 Units per eye; Open-Label Extension Period: up to 5 injections, up to 50 Units per eye per injection session; Mode of administration: intramuscular injection

Placebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection

Outcomes

Primary Outcome Measures

Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by a Blinded Independent Rater)
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 sumscore is the sum of the two components of the scale: JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity) JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.

Secondary Outcome Measures

Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by Subject Diary)
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 sumscore is the sum of the two components of the scale: JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity) JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Blepharospasm Disability Index (BSDI) Change From Baseline in the BSDI at Week 6 After Injection
The Blepharospasm Disability Index is a scale for the 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 change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Patient Evaluation of Global Response (PEGR) at Final Visit
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).

Full Information

First Posted
November 30, 2006
Last Updated
March 13, 2013
Sponsor
Merz Pharmaceuticals GmbH
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1. Study Identification

Unique Protocol Identification Number
NCT00406367
Brief Title
IncobotulinumtoxinA (Xeomin) Versus Placebo in the Treatment of Blepharospasm
Official Title
Prospective, Double-blind, Placebo-controlled, Randomized, Multi-center Trial With an Open-label Extension Period to Investigate the Efficacy and Safety of incobotulinumtoxinA (Xeomin) in the Treatment of Blepharospasm
Study Type
Interventional

2. Study Status

Record Verification Date
March 2013
Overall Recruitment Status
Completed
Study Start Date
October 2006 (undefined)
Primary Completion Date
April 2008 (Actual)
Study Completion Date
July 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Merz Pharmaceuticals GmbH

4. Oversight

5. Study Description

Brief Summary
Patients received one injection with incobotulinumtoxinA (Xeomin) or placebo at baseline. Thereafter, all patients who entered the Open-Label Extension Period (OLEX) received up to five injection sessions of incobotulinumtoxinA (Xeomin) during the OLEX period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Blepharospasm

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
109 (Actual)

8. Arms, Groups, and Interventions

Arm Title
incobotulinumtoxinA (Xeomin)
Arm Type
Experimental
Arm Description
incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), up to 50 Units per eye; Open-Label Extension Period: up to 5 injections, up to 50 Units per eye per injection session; Mode of administration: intramuscular injection
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection
Intervention Type
Drug
Intervention Name(s)
incobotulinumtoxinA (Xeomin)
Intervention Description
incobotulinumtoxinA (Xeomin, also known as "NT 201" or "Botulinum toxin type A (150 kiloDalton), free from complexing proteins") (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, up to 50 Units per eye; Mode of administration: intramuscular injection
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo to incobotulinumtoxinA (Xeomin) powder for solution for injection dose (Main Period only): one injection session of solution, prepared by reconstitution of powder with 0.9% Sodium chloride (NaCl), placebo volume corresponding to up to 50 Units per eye; Mode of administration: intramuscular injection
Primary Outcome Measure Information:
Title
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by a Blinded Independent Rater)
Description
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 sumscore is the sum of the two components of the scale: JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity) JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Time Frame
Baseline, week 6
Secondary Outcome Measure Information:
Title
Jankovic Rating Scale (JRS) Change From Baseline in the JRS Severity Subscore at Week 6 After Injection (Assessed by Subject Diary)
Description
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 sumscore is the sum of the two components of the scale: JRS-Severity score which ranges from 0 (=absence of severity) to 4 (=maximum severity) JRS-Frequency score which ranges from 0 (=no frequency) to 4 (=maximum frequency) The change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Time Frame
Baseline, week 6
Title
Blepharospasm Disability Index (BSDI) Change From Baseline in the BSDI at Week 6 After Injection
Description
The Blepharospasm Disability Index is a scale for the 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 change from baseline was calculated as the score at the corresponding visit minus the baseline score.
Time Frame
Baseline, week 6
Title
Patient Evaluation of Global Response (PEGR) at Final Visit
Description
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).
Time Frame
Final visit (up to week 20 after injection of the Main Period)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria: Male or female pretreated outpatients between ages 18 and 80 years (inclusive) A clinical diagnosis of bilateral blepharospasm (BEB) characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles A need for injection of Botulinum toxin (defined by a Jankovic Rating Scale (JRS) severity subscore >= 2) On a stable dose of other medications (if any) used for focal dystonia treatment (e.g. anticholinergics and benzodiazepines) for at least 3 months prior to and expected throughout the Main Period Source documentation of the last two consecutive injection sessions with BOTOX® and a stable satisfactory therapeutic response directly prior to trial entry At least 10 weeks must have been passed between the last injection with BOTOX® for BEB and Baseline Main Exclusion Criteria: Atypical variant of BEB caused by inhibition of levator palpebrae muscle Myotomy or denervation surgery in the affected muscles (e.g. peripheral denervation and/or spinal cord stimulation) The previous two injections with BOTOX® with more than 50 Units per eye Hypersensitivity to human serum albumin, sucrose, or Botulinum toxin A Neuroleptic induced blepharospasm Diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease which might interfere with the trial Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to Baseline and during the trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joseph Jankovic, Prof.
Organizational Affiliation
Houston, Texas
Official's Role
Study Chair
Facility Information:
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
David King, MD - Private Practice
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3J 3T1
Country
Canada

12. IPD Sharing Statement

Citations:
PubMed Identifier
21520284
Citation
Jankovic J, Comella C, Hanschmann A, Grafe S. Efficacy and safety of incobotulinumtoxinA (NT 201, Xeomin) in the treatment of blepharospasm-a randomized trial. Mov Disord. 2011 Jul;26(8):1521-8. doi: 10.1002/mds.23658. Epub 2011 Apr 22.
Results Reference
result
PubMed Identifier
23435927
Citation
Truong DD, Gollomp SM, Jankovic J, LeWitt PA, Marx M, Hanschmann A, Fernandez HH; Xeomin US Blepharospasm Study Group. Sustained efficacy and safety of repeated incobotulinumtoxinA (Xeomin((R))) injections in blepharospasm. J Neural Transm (Vienna). 2013 Sep;120(9):1345-53. doi: 10.1007/s00702-013-0998-9. Epub 2013 Feb 23.
Results Reference
result

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IncobotulinumtoxinA (Xeomin) Versus Placebo in the Treatment of Blepharospasm

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