Clinical Trial: Blepharospasm Short Interval

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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 S

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.

Detailed Summary:
Sponsor: Merz Pharmaceuticals GmbH

Current Primary Outcome: The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline. [ 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. ]

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".


Original Primary Outcome: Blepharospasm Disability Index [BSDI] measured at baseline visit before first injection, at each day of the following treatment visits before administration of NT 201, at the follow-up visit and at each telephone contact

Current Secondary Outcome:

  • Change in Jankovic Rating Scale (JRS) score from baseline over time. [ Time Frame: From baseline up to week 62 ]
    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 [ Time Frame: Week 6 up to week 62 ]
    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).


Original Secondary Outcome: - JRS sum score recorded at visit V1-V8, at visit V9, and at each telephone contact - Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit

Information By: Merz Pharmaceuticals GmbH

Dates:
Date Received: July 25, 2007
Date Started: August 2007
Date Completion:
Last Updated: February 3, 2012
Last Verified: February 2012