E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Muscle pain in the extremities due to spasticity in children with cerebral palsy
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E.1.1.1 | Medical condition in easily understood language |
Muscle pain in the extremities due to spasticity in children with cerebral palsy |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028322 |
E.1.2 | Term | Muscle pain |
E.1.2 | System Organ Class | 100000004859 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10041418 |
E.1.2 | Term | Spasticity muscle |
E.1.2 | System Organ Class | 100000004852 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021745 |
E.1.2 | Term | Infantile cerebral palsy, unspecified |
E.1.2 | System Organ Class | 100000004850 |
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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 examine the effect of botulinum toxin injections in regards to pain and quality of life in children with cerebral Palsy |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Diagnosed with spastic cerebral palsy
- GFMCS 1-5.
- Age 2-18
- Having a pain response by the revised and individualized Face Legs Activity Cry and Consolability (rFLACC) for muscles with spasticity (MAS score ≥ 1 and a spasticity grade of 1-4 on the Tardieu scale) during the passive range of motion by the treating physician.
- Requiring BonTA injection for their spasticity
- having had a washout period of at least 6 month from last injection of botulinum toxin
- Dynamic Myocontracture /spasticity in the painful limb (MAS score ≥ 1 and a spasticity grade of 1-4 on the Tardieu scale) |
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E.4 | Principal exclusion criteria |
- Fixed myocontracture (Reduced and static passive range of motion)
- Severe athetoid/dystonic movements in the targeted leg(s)
Relative exclusion criteria
Prior and recent surgery that might influence the pain evaluation is a contraindication and will be evaluated on an individual basis for the specific test subject by the principle investigator.
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E.5 End points |
E.5.1 | Primary end point(s) |
The revised Face, Legs Activity, Cry, Consolability Scale (r-FLACC):
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1-2 weeks pre injection and 4, 12, 28 weeks post injection |
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E.5.2 | Secondary end point(s) |
Secondary endpoints
Different pain scores:
Paediatric Pain Profile (PPP)
Wong-Baker pain scale (3-8)
Visual analogue Scale (VAS) (8y+):
Quality of life questionaire: CPchild, CPQQL
Tertiary endpoints :
Spasticity (Tardieu scale and modified Ashworth scale) and Passive range of motion of the muscles being treated
Goal attainment scale
Modified House Classification (MHC)
Bilateral Hand function
Thumb in palm
Measurement of spasticitet - dynamic component using the NeuroFlexor
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1-2 weeks pre injection and 4, 12, 28 weeks post injection |
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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 | No |
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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
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 | 6 |
E.8.9.1 | In the Member State concerned days | |