E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cervical dystonia (CD), one of the most common forms of focal dystonia, is characterised by an altered head posture, pain in the the neck and shoulder region and hypertrophy of the concerned muscles. Over the past 15 years local injections of botulinum toxin into various neck muscles have become the first line therapeutic approach. |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The results of a dose finding study have shown that 500 units Dysport® are the optimal starting dose for the treatment of patients with rotational torticollis. Although Dysport is an established first-line neurological treatment for cervical dystonia since its registration in Germany in 1995, there are only little data available on dose, efficacy and safety in the treatment of heterogeneous forms of cervical dystonia.
The aim of this multicentre open study is to demonstrate the equivalent efficacy and safety in the treatment of the two most frequent forms of cervical dystonia (predominantly rotational torticollis and predominantly laterocollis) with the standard initial dose of 500 units Dysport.
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E.2.2 | Secondary objectives of the trial |
Quality of life in week 0, 4 and 12 (disease specific craniocervical dystonia questionnaire, CDQ-24, Müller et al., 2004), efficacy assessment by patient and investigator, safety / tolerance / pain assessment by patient and investigator, patient diary (weekly documentation of function in every day life activities, pain and pain duration), documentation of dose and injection protocol of the following treatment after end of the study, documentation of adverse events / adverse drug reactions, descriptive analyses of cervical dystonia subgroups, Tsui-Score over week 0, 4 and 12 (while patient sitting), optional: additional Tsui-Score rating while patient walking, comparison Tsui-Score patient sitting and patient walking |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. De novo patients with cervical dystonia, but not pure retro-or antecollis 2. Ambulant patients 3. Age 18 and older 4. Written consent to participate in the study
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E.4 | Principal exclusion criteria |
1. Pre-treatment of cervical dystonia with botulinum toxin 2. Pre-treatment with botulinum toxin in any other indication than cervical dystonia within the past 12 months 3. Known allergy or antibodies against botulinum toxin 4. Pure retro- or antecollis 5. Pure tremor capitis 6. Pure shift (sagittal or lateral) 7. Dysphagia 8. Serious respiratory tract diseases in need of treatment 9. Ingestion of neuroleptics or hidden neuroleptics, e.g. Paspertin
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E.5 End points |
E.5.1 | Primary end point(s) |
Tsui-Score in weeks 4 vs. baseline in week 0 (rating while patient sitting).
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The study ends with the last visit (in week 12) of the last recruited patient. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |