E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Moderate to Severe Cervical Dystonia |
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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 objectives of this study are to evaluate the safety and efficacy of fixed dosages of BOTOX® or Dysport® in the treatment of idiopathic cervical dystonia in subjects who have been previously treated successfully with equivalent doses of BOTOX®. |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects with idiopathic cervical dystonia of the predominantly rotational form (i.e., spasmodic torticollis) of minimum duration eighteen (18) months. 2. Moderate-to-severe symptoms as defined by a minimum Toronto Western Spasmodic Torticollis Scale (TWSTRS) severity scale score of 15, with a rotation score >2 and a duration factor >2. 3. Subjects have received successful treatment as determined clinically by both the investigator and patient with 180 – 220 U BOTOX® in each of the last two (2) treatment sessions. 4. Cervical dystonia symptom severity is considered predictable and successfully responsive to BOTOX® treatment. 5. The most recent treatment with BOTOX® occurred on a date at least 16 weeks prior to study entry, and the subject has received two (2) treatment sessions within the prior 40 weeks with a good clinical response. 6. Outpatient, male or female subjects, of any race, age 18 and older. 7. Screening laboratory values either within the reference ranges as defined by the laboratory or in the investigator’s opinion out of range test results that are not clinically significant. 8. Negative urine pregnancy test on the day of treatment prior to the administration of study medication (for female subjects of childbearing potential). 9. Written informed consent has been obtained. 10. Written Authorization for Use and Release of Health and Research Study Information (as applicable) has been obtained. 11. Written Data Protection Consent (European and Indian sites only) has been obtained. 12. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable. 13. Ability to follow study instructions and likely to complete all required visits.
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E.4 | Principal exclusion criteria |
1. Symptomatic or non-idiopathic cervical dystonia. 2. Current or previous surgery, peripheral denervation, and/or spinal cord stimulation for the management of cervical dystonia. 3. Previous injections of phenol or alcohol for cervical dystonia. 4. History or evidence of secondary non-response to botulinum toxin type A therapy. 5. Presence of significant anterocollis, retrocollis, or head shift as the principal component of cervical dystonia. 6. Profound atrophy of the muscles in the target area(s) of injection. 7. Infection or skin problem at the anticipated injection site. 8. Myasthenia Gravis, Eaton-Lambert Syndrome, Amyotrophic Lateral Sclerosis or any other disease that might interfere with neuromuscular function. 9. Current anticoagulant therapy. 10. Failure to meet prohibited/restricted concomitant medication criteria: 11. Subjects planning inpatient surgery or other elective hospitalization during the study period. 12. Females who are pregnant, nursing, or planning a pregnancy (positive urine pregnancy test). 13. Females of childbearing potential, not using a reliable means of contraception. 14. Any uncontrolled systemic disease. 15. History of treatment with botulinum toxin type A of any brand other than BOTOX® within the past two (2) years. 16. History of treatment with botulinum toxin type B within the past two (2) years. 17. Allergy or sensitivity to any component of the study medication. 18. Recent history of alcohol or drug abuse (as defined by DSM IV criteria). 19. History of poor cooperation, non-compliance with medical treatment, or unreliability. 20. Subjects currently participating in another investigational drug study or who have participated in an investigational drug study within 30 days of the Baseline Visit. 21. Any condition or situation which, in the investigator’s opinion, places the patient at significant risk, could confound the study results, or may interfere significantly with the patient’s participation in the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome variable will be the incidence and severity of the adverse event of dysphagia, evaluated using ratings from a structured questionnaire and from a Dysphagia Interview. |
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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 | No |
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 | No |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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) | Yes |
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.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 1 |