E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Lower limb spasticity in children with dynamic equinus foot deformity due to cerebral palsy |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10024132 |
E.1.2 | Term | Leg spasticity |
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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 |
The primary study objective is to assess the long term safety of repeated treatments with Dysport used in the treatment of lower limb spasticity in children with dynamic equinus foot deformity due to CP. |
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E.2.2 | Secondary objectives of the trial |
The secondary study objective is to assess the long term efficacy of repeated treatments with Dysport, which will include changes in the following efficacy parameters for the affected lower limb(s): • Modified Ashworth Scale (MAS) at the ankle joint. • Physician’s global assessment (PGA) of treatment response, • Goal Attainment Scale (GAS), • Tardieu Scale (TS), • Observational Gait Scale (OGS), • Lower limb pain, • Duration of effects, • Time intervals between treatments, and • Quality of life (QoL). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All subjects must fulfil the following inclusion criteria to be eligible for the study: (1) Completion of the double blind study (Study 141) up to the Week 12, Week 16, Week 22 or Week 28 follow up visit. (2) Without any major protocol deviations and/or any ongoing AEs, either of which, in the opinion of the Investigator would pose an unacceptable risk to the subject were he/she to continue receiving treatment in this open label extension study. (3) Written informed consent obtained from the child’s parent(s)/guardian(s) for this study, and assent from the child, when and where applicable. |
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E.4 | Principal exclusion criteria |
Subjects are to be excluded if any of the following apply: (1) Fixed myocontracture as defined by a passive range of motion (Xv1) of 10° or less in ankle dorsiflexion. (2) Unwillingness or inability to comply with the protocol. (3) Current need for surgery of the muscles of any affected limb. (4) Treatment with any drug that interferes either directly or indirectly with neuromuscular function (e.g. aminoglycoside antibiotics or neuroblocking agents used during surgery e.g. curare) within the last 30 days prior to study medication or a planned treatment with such drugs. (5) Be pregnant and/or lactating. (6) Not willing to use contraceptive measures throughout the course of the study if post pubertal and sexually active. (7) An infection at the injection site(s). (8) Planned treatment with any new investigational drug or device during the study period. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety will be assessed through collection of AEs, vital signs (blood pressure (BP) and heart rate (HR)), clinical laboratory parameters (haematology and clinical chemistry (including alkaline phosphatase - total and bone isoenzyme, and blood glucose), development of antibodies against BTX-A (BTX-A-Abs), 12-lead electrocardiograms (ECGs). Use of prior and concomitant medications and prior lower limb surgeries will be recorded. |
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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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | No |
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 | No |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |