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Blepharospasm Short Interval

Primary Purpose

Blepharospasm

Status
Terminated
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
NT 201
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

Inclusion Criteria:

  • A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
  • Medical need for treatment with shortened injection intervals (< 12 weeks) confirmed by the patient and investigator
  • Blepharospasm Disability Index (BSDI) at baseline visit before injection of > / = 1,6

Exclusion Criteria:

  • Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid)
  • Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert-Eaton syndrome), as well as amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial
  • The previous three injections with Botulinum toxin Type A with more than 50 Units [U] per eye per injection session
  • Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to baseline and during the trial

Sites / Locations

  • Universitaets Augenklinik

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NT 201 (IncobotulinumtoxinA/Xeomin®)

Arm Description

Outcomes

Primary Outcome Measures

The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
The BSDI includes 6 items to be assessed by a 5-point scoring (i.e., 0-4 points per item) ranging from "no impairment, "slight impairment", "moderate impairment", "severe impairment" to "no longer possible due to my illness". The 6 items are "Driving a vehicle", "Reading", "Watching TV", "Shopping", "Walking" and "Doing everyday activities".

Secondary Outcome Measures

Change in Jankovic Rating Scale (JRS) score from baseline over time.
The JRS ranges from 0-8 points and consists of the two items "Severity" and "Frequency" with five rating categories each (0-4 points).
Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit
The PEGR scale is a descriptive subjective 9-point response self-rating scale ranging from "complete abolishment of signs and symptoms" (+4) down to "very marked worsening" (-4).

Full Information

First Posted
July 25, 2007
Last Updated
February 3, 2012
Sponsor
Merz Pharmaceuticals GmbH
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1. Study Identification

Unique Protocol Identification Number
NCT00507637
Brief Title
Blepharospasm Short Interval
Official Title
Prospective, Open-label, Single-arm, Single-center Trial to Investigate the Tolerability of NT 201 and Quality of Life of Patients in the Treatment of Blepharospasm With Shortened Injection Intervals
Study Type
Interventional

2. Study Status

Record Verification Date
February 2012
Overall Recruitment Status
Terminated
Why Stopped
primary efficacy endpoint not attainable.
Study Start Date
August 2007 (undefined)
Primary Completion Date
February 2008 (Actual)
Study Completion Date
February 2008 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The objective of this Phase II study is to show that pre-treated patients with BEB with the need of shortened injection intervals achieve an improved and stable quality of life level if they receive repeated NT 201 injections at short injection intervals. In addition, the tolerability of repeated shortened NT 201 administrations in patients with BEB including the development of neutralizing antibodies will be investigated.

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 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NT 201 (IncobotulinumtoxinA/Xeomin®)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
NT 201
Other Intervention Name(s)
Xeomin®, IncobotulinumtoxinA, "Botulinum toxin type A (150 kiloDalton), free from complexing proteins"
Intervention Description
Injection of up to 50 Units NT 201 (incobotulinumtoxinA/Xeomin®) per eye and injection visit (8 injections visits in total).
Primary Outcome Measure Information:
Title
The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
Description
The BSDI includes 6 items to be assessed by a 5-point scoring (i.e., 0-4 points per item) ranging from "no impairment, "slight impairment", "moderate impairment", "severe impairment" to "no longer possible due to my illness". The 6 items are "Driving a vehicle", "Reading", "Watching TV", "Shopping", "Walking" and "Doing everyday activities".
Time Frame
From baseline up to week 62. Follow up visits include 8 injection visits (V2 to V8) with intervals of at least 6 weeks between injections.
Secondary Outcome Measure Information:
Title
Change in Jankovic Rating Scale (JRS) score from baseline over time.
Description
The JRS ranges from 0-8 points and consists of the two items "Severity" and "Frequency" with five rating categories each (0-4 points).
Time Frame
From baseline up to week 62
Title
Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit
Description
The PEGR scale is a descriptive subjective 9-point response self-rating scale ranging from "complete abolishment of signs and symptoms" (+4) down to "very marked worsening" (-4).
Time Frame
Week 6 up to week 62

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles Medical need for treatment with shortened injection intervals (< 12 weeks) confirmed by the patient and investigator Blepharospasm Disability Index (BSDI) at baseline visit before injection of > / = 1,6 Exclusion Criteria: Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid) Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert-Eaton syndrome), as well as amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial The previous three injections with Botulinum toxin Type A with more than 50 Units [U] per eye per injection session 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
Bettina Wabbels, MD
Organizational Affiliation
Universitaets Augenklinik Bonn
Official's Role
Principal Investigator
Facility Information:
Facility Name
Universitaets Augenklinik
City
Bonn
ZIP/Postal Code
53125
Country
Germany

12. IPD Sharing Statement

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Blepharospasm Short Interval

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