Prospective, single-arm, multicenter trial to investigate the efficacy and safety of NT 201 (botulinum neurotoxin type A free of complexing proteins) and the duration of treatment effect after one injection session and in long-term treatment in patients with cervical dystonia
- Conditions
- Cervical dystonia, predominantly rotational spasmodic torticollisMedDRA version: 10.0Level: LLTClassification code 10064124Term: <Manually entered code. Term in E.1.1>
- Registration Number
- EUCTR2006-003410-18-DE
- Lead Sponsor
- Merz Pharmaceuticals GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- Not specified
- Willingness of the patient to participate as documented by written informed consent
- Male or female patient aged = 18 and < 76 years
- A clinical diagnosis of cervical dystonia, i.e., spasmodic torticollis, of predominantly rotational form with a need for injection. Cervical dystonia is characterized by involuntary, inappropriate neuromuscular hyperactivity in neck and shoulder muscles leading to abnormal head movements and postures
- Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS, see protocol) – total score at baseline = 25
- TWSTRS – severity score = 10
- TWSTRS – disability score = 3
- Patient must be on a stable dose of other medications (if any) used for focal dystonia treatment (e.g., anticholinergics and benzodiazepines) for = 3 months prior to and throughout the Main Period and if possible, throughout the Extension Period
- Negative pregnancy test at trial entry for female of childbearing potential (premenopausal female or postmenopausal female with last menses less than 12 months ago)
- Woman of childbearing potential 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)
- For pre-treated patients only: Source documentation of the last two consecutive injection sessions with botulinum neurotoxin type A and stable treatment response directly prior to trial entry.
- For pre-treated patients only: At least 10 weeks must have passed between the last injection session with botulinum neurotoxin type A for cervical dystonia and the time of the baseline visit.
- For pre-treated patients only: The most recent injection session with botulinum neurotoxin for cervical dystonia must have been at a dose of = 300 units of Botox® or Xeomin® or = 1,200 units Dysport®
For the longterm extension period:
- Negative pregnancy test for female of childbearing potential (premenopausal female or postmenopausal female with last menses less than 12 months ago)
- Woman of childbearing potential 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)
- At least 10 weeks must have been passed since the last injection session with botulinum neurotoxin type A for cervical dystonia
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
- Pre-treatment with botulinum neurotoxin type B (Neurobloc®) at any time prior to study entry
- Concomitant treatment with botulinum neurotoxin of any serotype for any other indication (including aesthetic indications) and for any body region during the trial
- Traumatic torticollis and tardive torticollis
- Previous or planned myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation and/or spinal cord stimulation)
- Hypersensitivity to human serum albumin, sucrose or botulinum neurotoxin type A
- Diagnosis of myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial
- Severe swallowing disorder of any origin (Dysphagia Scale score = 3 = severe, with swallowing difficulties and requiring a change in diet)
- Predominantly marked limitation on passive range of motion that clinically suggests fixed structures, e.g., cervical contractures or cervical spine syndrome
- Present or pre-existing blood coagulation disorders including therapeutic or prophylactic anticoagulation (e.g., heparin, phenprocoumon)
- Infection in the area of the planned injection sites or systemic infection presenting a hazard for local injections into the neck muscles
- Non-authorized concomitant treatment (see protocol section Prior and Concomitant Therapy)
- Current participation in another investigational clinical trial or participation in a clinical trial within the past 3 months
- Inability of patient to give informed consent
- Nursing mother
- Any severe or uncontrolled systemic disease (e.g., cardiac, renal, pulmonary, hepatic, or gastrointestinal), malignant tumor, or known HIV infection in medical history
- Planned elective surgery under general anesthesia during the trial
- Known current alcoholism or other drug abuse/dependence
- Being an employee or direct relative of an employee of the involved CRO, the Study Center or Merz Pharmaceuticals
For the long-term extension period:
- Concomitant treatment with botulinum neurotoxin of any serotype for any other indication (including aesthetic reasons) and for any body region during the trial
- Planned myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation and/or spinal cord stimulation)
- Severe swallowing disorder of any origin (Dysphagia Scale score ³ 3 = severe, with swallowing difficulties and requiring a change in diet
- Predominantly marked limitation on passive range of motion that clinically suggests fixed structures, e.g., cervical contractures or cervical spine syndrome
- Present or pre-existing blood coagulation disorder including therapeutic or prophylactic anticoagulation (e.g., heparin, phenprocoumon)
- Infection in the area of the planned injection sites or systemic infection presenting a hazard for local injections into the neck muscles
- Non-authorized concomitant treatment (see protocol section Prior and Concomitant Therapy9.4.7)
- Nursing mother
- Any severe or uncontrolled systemic disease (e.g., cardiac, renal, pulmonary, hepatic, or gastrointestinal), malignant tumor, or medical history of HIV infection
- Planned elective surgery under general anesthesia during the trial
- Known current alcoholism or other drug abuse/dependence
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 confirm the safety and efficacy of NT 201 after one injection session in the treatment of patients with cervical dystonia. ;Secondary Objective: As secondary objectives the efficacy and safety profile and the duration of treatment effect of NT 201 in long-term treatment with repeated injection sessions will be determined. In addition, the usefulness of a patient diary for achievement of optimal treatment during this period in clinical practice will be investigated.<br>As part of the safety evaluations during the Main Period and the Extension Period, the incidence of adverse events (AEs) and the formation of antibodies will be investigated.<br>;Primary end point(s): TWSTRS – total score : Change from baseline to week 4 after initial injection session
- Secondary Outcome Measures
Name Time Method