E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amiotrophic Lateral Sclerosis |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002026 |
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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 assess if the addition of EPO to the conventional therapy can improve the therapeutic outcome in patients affected by ALS. Safety will be assessed for all subjects, for the entire duration of the study. |
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E.2.2 | Secondary objectives of the trial |
Secondary parameters will include the Neuro-Phisiological index NI , FVC , the SF-36 quality of life rating scale and the number of patients showing an improvement 15 in ALSFRS-R slope as compared to the lead-in period responders . |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
- Caucasian male or female outpatients - aged 18 to 75 years inclusive - diagnosis of probable or definite ALS according to the El Escorial revised criteria Brooks et al., 2000 - first symptoms of ALS by no more than 2 years - in treatment with steady regimen of Riluzole and Vitamin E by at least 3 months, and desiring its continuation - FVC 75 of predicted - no conditions known to be contraindications to the use of EPO -written informed consent |
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E.4 | Principal exclusion criteria |
- subjects who underwent tracheostomy - subjects with signs of conduction blocks of motor nerves, sensory nerves or both on nerve conduction study - subjects with clinical signs of dementia - history of myocardial infarction or thrombotic vascular events such as stroke, pulmonary emboli, deep vein thrombosis, TIA - clinically evident cardiac disease ischemic heart disease or congestive heart failure - uncontrolled hypertension - active malignancy - polycytemia - myeloproloferative disorders - hypercoagulabily disorders - porphyria - known hypersensitivity to human albumin - pregnant and lactating women - subjects who received an experimental drug or have participated in a clinical trial within 3 months prior to screening - employees of the Investigator or study centre with direct involvement in the study or in other sudies under the direction of the Investgator or study center |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary evaluation parameter will be the ALSFRS-R Cederbaum et al, 1999 according to the consensus conference on designing and implementing clinical trials in ALS Leigh et al., 2004 . Primary endpoint will be the ALSFRS-R slope during the treatment period as compared to the lead-in period. Safety evaluation Incidence, severity and type of adverse events,; changes in clinical laboratory findings. |
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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 | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | Information not present in EudraCT |
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 | Information not present in EudraCT |
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 | 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 | 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.8.3 |
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.8.5 | 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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |