E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic lateral sclerosis therapy |
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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 |
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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 |
Assessment of the impact of LAC at the dose of 3g/day by oral route for 12 consecutive months on the disability of ALS, in a clinically significant and measurable way |
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E.2.2 | Secondary objectives of the trial |
1)Assessment of the impact of the drug on mortality (all causes); 2)Evaluation of the tolerability and safety of the drug at the given treatment regimen. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Inclusion criteria To be accepted, a case must satisfy all of the following: a. Diagnosis of definite, probable (laboratory-supported) or probable ALS (Sect. 5); b. Age between 40 and 70 years; c. Disease duration 6 < months < 24 months; d. Mild to moderate disability, documented by satisfactory bulbar and spinal function (minimal score of 3 on ALS Functional Rating Scale [ALS-FRS-R] 34 for swallowing, cutting food and handling utensils, and walking), satisfactory respiratory function (forced vital capacity (FVC) > 80% of predicted); e. Documented progression of the disease in the last 3 months; f. Patients able to understand, to comply with the requirements of the entire study, and to give an informed written consent. |
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E.4 | Principal exclusion criteria |
Exclusion criteria The presence of at least one of the following leads to exclusion of the patient: a. Familial ALS; b. Antecedent polio infection; c. Motor neuron disease other than ALS (progressive bulbar palsy; progressive muscular atrophy; primary lateral sclerosis); d. Involvement of other neurological systems (sensory, extra pyramidal, oculomotor, cerebellar, autonomic). e. Antecedent or current exposure to metals (lead, aluminium, mercury, manganese, magnesium, selenium); f. Diabetes, severe clinical conditions, like cardiovascular disorders, arterial hypertension, kidney and liver disorders, dysthyroidism; g. Neoplasms or other diseases reducing life expectancy; h. Severe mental deterioration; i. Poor compliance with previous treatments; l. Neuroradiologic findings documenting lesions which might be responsible of the clinical findings; m. Experimental treatments in the preceding 3 months; n. Women who are pregnant or breast-feeding and/or of childbearing potential for the duration of the study; o. Patient and/or caring physician unwilling to take and/or prescribe riluzole. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary end-point is the proportion of cases becoming non-self supporting during the 12-month follow-up period. |
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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 | 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 | 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 | 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.8.5 | 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
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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 | 9 |
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 |