E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic Lateral Sclerosis |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002026 |
E.1.2 | Term | Amyotrophic lateral sclerosis |
|
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 |
Safety and efficacy evaluation of Lithium carbonate combined with Riluzole in ALS. Primary endpoint is patient survival at month 18. |
|
E.2.2 | Secondary objectives of the trial |
Evaluation of necessity of PAV or tracheostomy and measure of the rate of functional deterioration over 18 month measured by change in the slope of ALSFRS-R, FVC, MRC an McGill quality of life. Evaluation of safety of the treatment in patiens with ALS |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Probable, probable-laboratory supported or definite ALS according to the revised version of the El Escorial World Fed. Neurology criteria with disease duration ≥ 6 and ≤ 36 months 2. Age ≥18 years and ≤ 75 years 3. Capable of understanding the information given and giving full informed consent 4.Continuously treated with Riluzole for at least 30 days and stabilized at 100 mg/day ( 50 mg bid) without significant drug reactions 5. FVC ≥ 60% of predicted 6. Female with child bearing potential, the patients must use adequate contraceptive measures and must not be pregnant or breastfeeding 7. Creatinine ≤ 1.5 mg/dl |
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E.4 | Principal exclusion criteria |
1. Presence of a medical condition contra-indicative to the use of Lithium or known hypersensitivity to the study drugs 2. Patients already taking Lithium in any form 3. Participation in other therapeutic study within the preceding 30 days or use of other investigational drugs or agents 4.Presence the following medical conditions: a. assumption of abuse drugs; renal insufficiency, hepatic insufficiency, heart disease, endocrine or hematological disorders; infective diseases including HIV; cancer; b. evidence of major psychiatric disorders or clinically evident dementia; c. plasmatic TSH > 20% with respect to normal values; d. use of diuretics 5. Abnormal ECG 6. Any medical conditions known to have an association with other neurological systems 7 Tracheostomy or assisted ventilation of any type during the preceding 3 months |
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E.5 End points |
E.5.1 | Primary end point(s) |
Patient`s survival in the two groups at 18 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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | 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
| 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 | 16 |
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
|
durata dello studio complessiva 30 mesi: 4 mesi di preparazione - 6 mesi arruolamento - 18 mesi trattamento - 2 mesi analisi statistica e stesura del rapporto finale |
|
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 |