Prospective, single-arm, single-center, open-label trial to investigate the tolerability of NT 201 and quality of life of patients in the treatment of blepharospasm with shortened injection intervals

• Conditions: Blepharospasm; MedDRA version: 9.1Level: LLTClassification code 10005159Term: Blepharospasm

• Registration Number: EUCTR2007-000697-23-DE
• Lead Sponsor: Merz Pharmaceuticals GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-06-01
• Last Update Posted: 29 April 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 58

Inclusion Criteria

- Willingness of the patient to participate as documented by signed informed consent
- Male or female pretreated outpatient between 18 and 80 years of age (inclusively)
- 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 patient and investigator
- Botulinum toxin Type A treatment effect after last injection before study entry according to patient’s self-assessment
- Blepharospasm Disability Index (BSDI) before injection =1,6
- Source documentation of the last three consecutive injection sessions with Botulinum toxin Type A
- 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 visit V1 and expected throughout the study
- Negative pregnancy test at trial entry for female of childbearing potential (premenopausal female or postmenopausal female with less than 12 months after last menses)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid)(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
- Frontalis suspension surgery in medical history
- Known hypersensitivity to human serum albumin, sucrose or Botulinum toxin Type A
- Known current alcoholism or other drug abuse / dependence
- Ongoing severe or uncontrolled systemic disease (e.g., cardiac, renal, pulmonary, hepatic, or gastrointestinal)
- Uncured malignant tumor or known HIV infection
- Present or pre-existing blood coagulation disorders including therapeutic or prophylactic anticoagulation (e.g. heparin, phenprocoumon)
- Infections in the area of the planned injection points or systemic infections presenting a hazard for local injections
- Treatment with non-authorized medications (see section Prior and Concomitant Therapy)
- Planned elective surgery under general anesthesia during the trial
- Nursing woman or woman of childbearing potential not using highly effective methods of birth control, defined as those which result in a low failure rate (i.e., less than 1% per year) when used constantly and correctly such as hormonal contraception or intrauterine contraceptive device or combination of two barrier methods (e.g. condom or diaphragm plus spermicidal cream)
- Current participation or participation within the past 90 days in another clinical trial
- Previous participation in this clinical study
- Employees or direct relatives of an employee of the CRO, the Study Center or Merz Pharmaceuticals

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to show that patients with BEB pre-treated with BTX-A who show a short duration of efficacy can achieve a stable quality of life by treatment with NT201 administered in shortened injection intervals (.i.e. < 12 weeks).. <br><br>;Secondary Objective: The secondary objective is to investigate the tolerability of NT 201 and the development of neutralizing antibodies of NT 201 administered in shortened injection intervals.<br>;Primary end point(s): The primary efficacy endpoint is the proportion of responders showing an improved and stable quality of life level over the course of the study according to the definition provided in the statistical section of the protocol. A two-sided confidence interval at the 95 % confidence level will be calculated for the overall responder rate.