E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
• Cerebral Palsy
• Spastic Paraplegia and Hemiparesis
• Equine and Equinovarus Foot Deformation |
|
E.1.1.1 | Medical condition in easily understood language |
• Cerebral Palsy
• Spastic Paraplegia and Hemiparesis
• Equine and Equinovarus Foot Deformation |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
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 |
To assess the clinical and neurophysiological efficacy of Xeomin® vs. Botox® in children with spastic equine and equinovarus foot deformation in pediatric cerebral palsy |
|
E.2.2 | Secondary objectives of the trial |
To assess the safety of Xeomin® use as compared to Botox® in this patient population |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Children from 2 through 12 years of age, of both sexes, suffering from spastic paraplegia or hemiparesis in pediatric cerebral palsy.
• Equine and equinovarus foot posture.
• Gastrocnemius spasticity of 2 points and greater, by modified Ashworth scale.
• Patient can walk unassisted or with a support.
• Mental development of patients is normal or mildly retarded.
• Previous course of spasticity treatment with BTA products was completed earlier than at 6 months before this trial or never administered before.
• Patient's parents have signed an informed consent, are able and wishing to adhere to procedures described in the trial protocol and to the schedule of visits throughout the entire period of treatment. |
|
E.4 | Principal exclusion criteria |
• Fixed ankle joint contracture.
• Previous denervation of spastic muscles by surgery, phenol or alcohol;
• Athetosis and dystonia in the area of injected muscles.
• Inflammation at the planned injection site.
• Elevated body temperature and acute (infectious and non-infectious) diseases at the time of injection.
• Neuromuscular transmission disorders (myasthenia gravis, Lambert-Eaton syndrome, etc.).
• Decompensated physical diseases potentially affecting the trial findings.
• Acute fever, infection or surgery within 1 month before the trial.
• Use of aminoglycosides or spectinomycin within 1 month before starting the trial.
• Hypersensitivity to any of product ingredients.
• Positive history for allergies (especially with regard to protein-containing products).
• Patient's parents are unable or unwilling to adhere to the trial protocol requirements including signing the informed consent and conforming to the schedule of visits.
• Participation in other clinical trials in the last 4 weeks before inclusion. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Changes from baseline in the degree of spasticity in gastrocnemius according to modified Ashworth scale (AS) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
• Changes from baseline in patient percentage in groups by the degree of gastrocnemius spasticity according to modified Ashworth scale
• Percentage of decrease in M-response magnitude and area recorded from the lateral and medial gastrocnemius heads, from baseline values
• Changes from baseline in the ratio of M-response recorded from the lateral and medial gastrocnemius heads and from tibialis anterior
• Changes from baseline in angles and angle ratio of ankle joints at passive and voluntary extension
• Changes from baseline in motor activity according to Gross Motor Function Classification Systems (GMFCS) criteria
• Changes from baseline in the degree of spasticity in gastrocnemius according to modified Ashworth scale |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
From baseline up to day 90 |
|
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 | 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) | 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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
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 | 5 |
E.8.9.2 | In all countries concerned by the trial days | 0 |