E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | Spasticity of the Gastrocnemius Muscle due to Cerebral Palsy | |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.0 | E.1.2 | Level | LLT | E.1.2 | Classification code | 10024132 | |
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 determine whether BoNT-A treatment of the gastrocnemius muscle is more effective in the reduction of spasticity if it is administered by multiple injections (test) or the standard double injection technique (reference). This will be determined by analysing the change in gait 4 weeks after each treatments using the physician's rating index. | |
E.2.2 | Secondary objectives of the trial | - Comparison of the change in gait at all other assessment timepoints. - Comparison of patient, parent and nurse pain scores (using FACES and VAS scales) for the two injection techniques. - Comparison of the safety of the two injection techniques. | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria | 1. The patient’s legal parent/guardian must give written (personally signed and dated) informed consent, and the patient must give informed assent before completing any study-related procedure, which means any assessment or evaluation that would not have formed part of their normal medical care. 2. The patient must be between 4 and 12 years of age inclusive. 3. The patient must have a diagnosis of hemiplegic or diplegic cerebral palsy. 4. The patient must be able to walk with or without the use of aids. 5. The patient must have spasticity of the Gastrocnemius muscle on one or both sides, as measured by an inability to achieve heel strike due to lower limb spasticity predominantly affecting the calf muscles. 6. The patient and his/her parent/guardian must be able and willing to comply with the requirements of the protocol. | |
E.4 | Principal exclusion criteria | 1. The patient has calf muscle contractures preventing dorsiflexion of the foot to less than the neutral position with the knee straight. 2. The patient also needs treatment to the adjacent Soleus or Tibialis Posterior muscles. 3. The patient needs treatment to the hamstring muscles on the same side as the treated Gastrocnemius muscle. 4. The patient has had prior lower limb orthopaedic surgery in the previous year. 5. The patient has severe athetoid movements of the lower limbs. 6. Previous injection with Botulinum toxin within 6 months prior to inclusion in the study. 7. The patient has a known allergy or hypersensitivity to any of the test compounds or materials. 8. The patient has received any investigational drug therapy within 30 days prior to the study, or is scheduled to receive such a drug during the study period. 9. The patient has previously entered this study. | |
E.5 End points |
E.5.1 | Primary end point(s) | Change in gait (assessed using the Physician's rating Index) 4 weeks after each treatment. A clinically relevant difference between the multiple injection (test) technique and the double injection (reference) technique, would be an additional 1 point improvement in the Physician's rating index for the test intervention over the reference intervention. | |
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 | Information not present in EudraCT |
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 | 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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description | |
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.8.5 | 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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | The last visit of the last subject undergoing the trial. | |
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 | 0 |
E.8.9.1 | In the Member State concerned days | |