E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
ALS patient according with the El-Escorial criteria |
|
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 |
Evaluation of the impact of Litio with a level between 0,4 and 0,8 mEq/L per os for 15 months on the disability of the ALS |
|
E.2.2 | Secondary objectives of the trial |
Evaluation of the drug tolerability and safety |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
a)Diagnosis of clinically definitive, probable or probabile laboratory-supported, possible ALS b)Adult males and females aged 18 to 75 years. c)Onset of disease within 36 months. d)Value more than 2 at the point Swallowing in the ALS-FRS-R scale, value more than 2 at the point walking in the ALS-FRS-R scale, value more than 3 at the point respiratory function at the ALS-FRS- R scale e)Progression of disease in the last 3 months. f)Able to understand the protocol and to give written informed consent. g)Concomitant standard Riluzole therapy 50 mg twice daily for at least 2 months or without standard Riluzole therapy during the period of the study. |
|
E.4 | Principal exclusion criteria |
a.pregresse poliomielite b.other motoneuron diseases different from ALS (progressive bulbar paralysis, progressive muscular atrophy, primary lateral sclerosis) c. other neurological diseases (sensory, extrapiramidal, cerebellar) d.A clinical relevant presence of cardiovascular, arterious hypertension, renal, hepatic and endocrine diseases. e.Creatininemie> 1,6 mg/dl f.A clinical relevant medical history of cardiovascular, brain vascular diseases. g.A history of presence of malignancy h.Important cognitive disorders i.Low compliance in past therapy j.Female with intention of pregnancy or breast feeding . the non-pregnancy will confirmed by pregnancy test a week before the onset of treatment. The absence of pregnancy risk is indicated by (1) menopause state, (2) total hysterectomy, (3) adequate method of contraception, (4) sexual inactivity |
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E.5 End points |
E.5.1 | Primary end point(s) |
Death or tracheostomy, or almoust 2 of the 3 following value at the ALS-FRS-R: 1 at the point swallowing, 1 at the point walking, 2 at the point respiratory failure. |
|
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 | No |
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 | Yes |
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 | Yes |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
- same IMP used at different dosage |
|
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
| |
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 | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |