E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
spinal and bulbar muscular atrophy (SBMA) |
atrofia muscolare spinale e bulbare (SBMA) |
|
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 | 15.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10029317 |
E.1.2 | Term | Neuromuscular disorders |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10029205 |
E.1.2 | Term | Nervous system disorders |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10047868 |
E.1.2 | Term | Weakness of limbs |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013969 |
E.1.2 | Term | Dyspnoea at rest |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal 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 main objective of the study is to investigate the safety and tolerability of clenbuterol of the SBMA |
Lo scopo principale di questo studio e' quello di investigare la sicurezza e la tollerabilita' del clembuterolo in SBMA |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Genetically confirmed SBMA male patients with more than one of the following symptoms: muscle weakness, muscle atrophy, bulbar palsy or hand tremor. 2) Patients able to walk independently along a flat corridor with or without the of a cane or similar equipment. 3) Patients who were 30 to 70 years old at the time of informed consent 4) Patients who gave written informed consent. |
1)Pazienti di sesso maschile con SBNA geneticamente confermata.Con piu'di un anno con piu' o uno dei seguenti sintomi:debolezza muscolare,atrofia muscolare,paralisi bulbare o tremore della mano. 2) Pazienti in grado di camminare indipendentemente lungo un corridoio piano con o senza l'uso di un bastone o equipaggiamenti similari. 3) Pazienti di eta'tra 30 e 70 anni al momento del consenso informato scritto. 4) Pazienti che hanno dato il consenso informato scritto. |
|
E.4 | Principal exclusion criteria |
1) documented cardiac disease. 2) documented hypersensibility to beta2 agonists. |
1) malattie cardiache documentate 2) ipersensibilita' ai farmaci beta 2 agonisti. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
change from the baseline of the walk distance (mt) in six minutes (6-min walk test) at 12 months. |
variazione rispetto al valore basale della distanza (mt) percorsa in 6 minuti a 6 mesi. |
|
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 | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |