E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Early stage of amyotrophic lateral sclerosis (ALS) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy, tolerability and safety of once-daily subcutaneous injections of 40 mg glatiramer acetate in subjects with ALS. |
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E.2.2 | Secondary objectives of the trial |
Time to death / tracheostomy/permanent assisted ventilation |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1. Male and female subjects with definite, probable or probable laboratory-supported ALS according to the World Federation of Neurology El Escorial criteria. ALS may be familial or sporadic. 2. Subjects must have experienced their first ALS symptoms within three years prior to the screening visit. 3. Subjects must have a slow VC score equal to or greater than 70% of the predicted value for gender, height and age at the screening and baseline visits. 4. The sum of the three respiratory items from the ALSFRS-R must total at least ten points at the screening and baseline visits. 5. Subjects taking riluzole must be on a stable dose for at least eight weeks prior to the screening visit and their serum AST and ALT levels should not exceed three times the upper normal limit. 6. Subjects must be between 18 and 70 years of age (inclusive). 7. Subjects and caregivers must be willing and able to provide written, informed consent. |
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E.4 | Principal exclusion criteria |
1. The use of invasive or non-invasive ventilation. 2. Subjects having undergone a gastrostomy. 3. Subjects with any clinically significant or unstable medical or surgical condition including cardiovascular, hepatic, pulmonary, renal, autoimmune, endocrine, metabolic or malignancy or any other condition that, in the investigator's opinion, places the subject at undue risk by participating in the study. 4. HBV or HCV seropositive subjects. 5. Subjects having used within the specified time prior to screening any of the following: + glatiramer acetate (any previous use) + mecasermin (within four weeks prior to screening) + rasagiline (within three weeks prior to screening) + minocycline (within three weeks prior to screening) + a dosage regimen of more than 600 mg/day coenzyme Q10 (within four weeks prior to screening) + any other marketed drug not approved for ALS (within twelve weeks prior to screening), excluding symptomatic drugs for ALS and supplements 6. Subjects participating in any other clinical trial for ALS (within twelve weeks prior to screening or thereafter). 7. Women who are pregnant or nursing at the time of screening or intend to be during the study period. Women of child-bearing potential who do not practice medically acceptable methods of contraception. 8. Subjects with current alcohol or drug abuse. 9. Subjects with known sensitivity to mannitol. 10. Subjects without a caregiver or healthcare professional to inject the study medication if unable to do so themselves. 11. Subjects unable at the time of the screening and baseline visits to comply with the planned schedule of study visits and study procedures. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline to each visit in ALSFRS-R score (slope). |
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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 | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Yes |
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 | 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 | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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
|
30 days post last dose 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 | |
E.8.9.1 | In the Member State concerned months | |
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 |