E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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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 evaluate if intramuscular injections with Botulinum toxin-A is effective in reducing muscle-related chronic pain in adults with spastic cerebral palsy, compared to placebo control. |
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E.2.2 | Secondary objectives of the trial |
• To evaluate if intramuscular injections with Botulinum toxin-A leads to reduced need for analgesics in adults with spastic cerebral palsy.
• To evaluate if intramuscular injections with Botulinum toxin-A is effective in reducing interference with activities of daily living (ADL) associated with pain in adults with spastic cerebral palsy.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Age ≥ 18 years.
- Spastic Cerebral palsy, according to SCPE guidelines 1
- Chronic pain, with pain localization related to regional spastic muscle. Chronic pain defined as:
* Recurring regional pain for at least three (3) months.
* Pain intensity on average for the last 24 hours ≥3 on Numerical Rating Scale.
- Signed Informed consent.
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E.4 | Principal exclusion criteria |
For reasons of safety:
- Allergy/hypersensitivity to Dysport® or any of its components.
- Pregnancy. All women not passed menopause or not previously surgically sterilized, will need to perform a urine pregnancy test at Visit 2, before treatment.
- Women who breastfeed their children.
For reasons of study consistency:
- Treatment with BoNT-A within the last 5 months.
- If there has been dose changes in any muscle-tone altering medication within two (2) weeks of Screening visit.
- A clear degenerative cause behind the pain as elucidated by the clinical examination (i.e. history of osteoarthritis).
- Intellectual disability and/or communication impairment that disable the individual from answering the questionnaires and giving informed consent.
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of responders derived as reduction of intensity of pain of ≥2 points on the Numerical Rating Scale (NRS) at Visit 3 (six weeks after treatment) compared to baseline. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline and ca six weeks after treatment
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E.5.2 | Secondary end point(s) |
• Categories of change in the use of analgesic treatments, derived as Increase, No change or Decrease at Visit 3 (six weeks after treatment), compared to baseline.
• Proportion of responders derived as a reduction in mean interference score of ≥1 on the Brief Pain Inventory (BPI) at Visit 3 (six weeks after treatment) compared to baseline.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline and ca six weeks after treatment |
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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 | No |
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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 2 |
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 | 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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |