E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Progressive myoclonus epilepsy of the Unverricht–Lundborg disease (ULD) type |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10054895 |
E.1.2 | Term | Baltic myoclonic epilepsy |
|
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 objective of the current study is to investigate the efficacy and sarfety of add-on dopaminergic medication (ropininirole hydrochloride) on the symptoms of ULD, especially on myoclonic jerks and on general disability. |
|
E.2.2 | Secondary objectives of the trial |
To investigate the efficacy of add-on dopaminergic medication (ropininirole hydrochloride) on electrophysiological determinants on disease activity (EEG and sensory evoked potentials) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) ULD diagnosed by an experienced neurologist, and confirmed with molecular diagnostics 2) Stimulus-activated myoclonus as the most prominent symptom of ULD 3) Age 18-60 years
|
|
E.4 | Principal exclusion criteria |
1) Current medicated psychiatric disorder 2) Previous severe head trauma 3) History of alcohol/substance abuse 4) Pregnancy or lactation 5) Known hypersensitivity to ropinirole hydrochloride or to other ingredients of the product 6) Concomitant participation in another drug study 7) Severe renal or hepatic insufficiency
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Clinical assessments/endpoints Unified myoclonus rating scale (UMRS) is the main clinical assessment of the efficacy of the ropinirole hydrochloride in the study on myoclonus severity and overall disability. UMRS questionnaire includes structural assessment sections on 1) patient questionnaire of myoclonus, 2) moclonus at rest, 3) stimulus sensitivity, 4) myoclonus with action, 5) functional tests, 6) global scores of disability, 7) presence of negative myoclonus and 8) severity of negative myoclonus.
Electrophysiological assessments/endpoints The electrophysiological evaluation including nerve conduction, multi-modality evoked potentials including visual evoked potential (VEP), somatosensory evoked potential (SSEP) and brainstem auditory evoked potential (BAEP) and electroencephalography (EEG) will be determined and evaluated. All the electrophysiological evaluations are carried out using standard protocols. |
|
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 | 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 | 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 | Yes |
E.8.2.3 | Other | No |
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 | Information not present in EudraCT |
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.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |