E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotophic lateral sclerosis |
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MedDRA Classification |
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 tolerability of Anakinra in combination with Riluzol in amyotrophic lateral sclerosis |
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E.2.2 | Secondary objectives of the trial |
Evaluation of clinical efficacy of Anakinra in amyotrophic lateral sclerosis |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients between 18 and 80 years of age - Clinical diagnosis of amyotrophic lateral sclerosis with predominant affection of the lower motor neuron or the clinical ALS variant of progressive muscular atophy (PMA) - Clinical signs of lower motor neuron degeneration in at least one anatomic region beyond the brain stem - Sporadic and familial ALS - Onset of paresis six months to four years before study inclusion - Treatment with riluzol 100mg/d at least 3 month before study inclusion |
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E.4 | Principal exclusion criteria |
- Diagnosis of amyotrophic lateral sclerosis with predominant affection or the upper motor neuron without clinical signs of a concurrent affection of the lower motor neuron in at least one anatomic region beyond the brain stem (spastic ALS) - Diagnosis of primary lateral sclerosis (PLS) - Patients with known intolerance to anakinra, riluzol or one of the additives - Clinically severe hypoventilation syndrome with vital capacity < 50% - Pregnancy or breastfeeding - Continuous non-invasive ventilation with ventilator-free time < 2 hours - Tracheotomy and mechanical ventilation - Laboratory parameters outside the normal range that correspond to a clinically severe cardiovascular, pulmological, hematological, hepatological, metabolic or renal disease - Malignancies - Severe renal insufficiency (creatinine clearance < 30 ml/min) - History of recurrent infections or a disease that may predispose to infections - Severe neutropenia (absolute neutrophil count < 1.5 x 109/l) - Monoclonal gammopathy of unknown significance - Infections including infections with tubercolosis, HIV and hepatitis B and C - Dementia and unable to give informed consent - History of epilepsy and epileptic seizures - Contraindication to E coli-derived proteins, anakinra or any components of the product - Concurrent therapy of anakinra and etanercept or other TNF blocking agents
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E.5 End points |
E.5.1 | Primary end point(s) |
An investigation of number and severity of adverse events (AE), serious adverse events (SAE), adverse drug reactions (ARD), unexpected adverse drug reactions (UADR), serious adverse drug reactions (SADR), suspected unexpected serious adverse reaction (SUSAR) and pathological laboratory parameters |
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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 | No |
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 | Yes |
E.8.1.1 | Randomised | No |
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 | No |
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 |
explorative trial for tolerability and safety |
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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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
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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• Unjustifiable risk and toxicity in risk-benefit analysis (decision taken by principal Investigator). • New scientific evidence provided during the study that could affect the patient’s safety (benefit-risk analysis no longer positive). • Request of the data monitoring committee, the sponsor or regulatory agency. |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |
E.8.9.2 | In all countries concerned by the trial days | 0 |