E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Focal upper limb spasticity due to upper motor neuron syndrome including poststroke spasticity. (for the objective of this study patients must also have stable compromised baseline respiratory status) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028335 |
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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 |
To evaluate the pulmonary function safety of patients with stable compromised baseline respiratory status who receive repeat treatments of BOTOX® for focal upper limb spasticity. |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Male or female, 18 years of age or older 2. Written informed consent 3. Written Authorization for Use and Release of Health and Research Study Information has been obtained. 4. Medically stable subjects with stable compromised baseline respiratory status who have spastic muscles in at least the upper limb that manifests as common spasticity induced patterns of the upper motor neuron syndrome 5. History of upper motor neuron syndrome that results in upper limb spasticity of one or both upper limbs (spasticity in lower limb is allowed but will not be treated with study medication) 6. In the opinion of the investigator, the subject has sufficient spasticity in an affected upper limb, to be designated as the study limb, to warrant a dose of 360 U of BOTOX® 7. Stable pulmonary condition defined as no history of acute pulmonary complications within the past 4 months (e.g. hospitalization for exacerbation of chronic obstructive pulmonary disease, pneumonia, obvious reactive airway disease diagnosed by signs and symptoms, abnormal chest x-ray readings, etc.) 8. The largest FEV1 must be 40% to 80% of the predicted value at Visit 1 and Visit 2, and the largest FEV1 value at Visit 2 must not vary more than 20% of the largest value at Visit 1 9. The largest FVC value at Visit 2 must not vary more than 20% of the largest FVC value at Visit 1 10. Ability to perform acceptable and reproducible FEV1 and FVC according to 1995 ATS spirometry guidelines 11. Ability to follow study instructions and likely to complete all required visits |
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E.4 | Principal exclusion criteria |
1. Stroke within 6 months of Visit 2 2. Known allergy or sensitivity to the study medication or its components 3. Previous therapy with BOTOX or any other botulinum toxin for any condition 4. Females who are pregnant, nursing, or planning a pregnancy during the study, or females of childbearing potential, not using a reliable means of contraception 5. Subjects with multiple sclerosis or primary lateral sclerosis where progression of the disease may confound study results 6. Any medical condition that may put the subject at increased risk with exposure to BOTOX® including diagnosed myasthenia gravis, Eaton- Lambert Syndrome, amyotrophic lateral sclerosis, or any other disorder that might interfere with neuromuscular transmission 7. More than one overnight hospitalization or 2 emergency room visits for respiratory related event(s) in the last 12 months 8. Infection or dermatological condition at anticipated injection sites 9. Current treatment with an intrathecal baclofen pump 10. Changes in corticosteroid medication dose/regimen in the last 2 months 11. Current enrollment in an investigational drug or device study or participation in such a study within 30 days of entry into this study 12. Subject has a condition or is in a situation which in investigator's opinion may put the subject at significant risk, may confound the study results, or may interfere significantly with the subject's participation in the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
The pulmonary function test (PFT) assessments FEV1, FVC (both observed and percent predicted values), and the derived variable FEV1/FVC are the primary assessments for this study. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
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) | No |
E.8.2.2 | Placebo | Yes |
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 | 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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 0 |