E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10008129 |
E.1.2 | Term | Cerebral palsy |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary aim of this study is to provide evidence for the effect of ITB treatment on the level of activities in dystonic CP patients. The primary outcome measurement is the effect on daily functioning and daily care measured by Goal Attainment Scaling. |
|
E.2.2 | Secondary objectives of the trial |
Secondary outcome measurements include dystonia, spinal muscle activity and spasticity. Side effects will be monitored and we will study whether patient characteristics influence outcome. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Dystonic cerebral palsy
• GMFCS IV or V
• Lesions on MRI (cerebral white matter, basal ganglia, central cortex)
• Aged 4 to 25 years old
• Able and willing to complete study protocol
• Consensus about inclusion
|
|
E.4 | Principal exclusion criteria |
• Contra-indications for general anaesthesia
• Contra-indications for baclofen
• Inadequate knowledge of Dutch language
• Deep brain stimulation
• Ventriculoperitoneal drain
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
goal attainment scaling : attainment of individual goals |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
baseline, 3 months and 12 months |
|
E.5.2 | Secondary end point(s) |
pain
comfort
dystonia
spasticity
PEDI |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
baseline, 3 months, 12 months |
|
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | 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 | 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 | 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
|
Interim analysis will be done by a blinded annalist after 10 and 20 subjects. If this shows that subjects in one of the groups have significant disadvantages compared to the other group, this could lead to premature termination of the study. This will result in termination of the blinded treatment part of this study but continuation of the second part of the study in which all subjects receive ITB treatment. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
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 |