E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic lateral sclerosis |
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E.1.1.1 | Medical condition in easily understood language |
Motor Neuron disease (Lou Gehrig´s disease) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002026 |
E.1.2 | Term | Amyotrophic lateral sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
Safety: To assess an absence of complications at the site of intrathecal infusion and no new neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the disease.
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E.2.2 | Secondary objectives of the trial |
Efficacy: To assess progress of the disease measured according to ALS functional rating scale (ALSFRS) and Norris scale and spirometry (FVC) after the AMSC treatment. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To participate in this trial, patients will have to meet following criteria:
1. established diagnosis of definite ALS according to El Escorial kriteria and data available from detailed neurologic observation (including ALSFRS, Norris scale, spirometry, brain and spinal cord MRI) for at least 6 months prior to the study entrance,
2. riluzole naive or at stable dose for at least 2 months,
3. patients between 18–65 years, both sexes, life expectancy more than 2 years,
4. patients able to provide written informed consent.
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E.4 | Principal exclusion criteria |
Patients meeting any of the following criteria will be excluded from the participation in this study:
1. FVC less than 70% ,
2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,
3. less than 15 points on Norris spinal scale,
4. pregnancy, breastfeeding
5. coagulopathy,
6. skin infection at the site of bone marrow aspiration or application of the cell product,
7. gastrostomy,
8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, reccurent thromboembolic disease .....),
9. alcohol or drug abuse,
10. cancer,
11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners,
12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube). |
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Complication at the site of intrathecal infusion
2) New neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributable to the natural progression of the disease. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
ad 1: at Visits IV, V, and VI (1 day to 3 months after treatment administration)
ad 2: at Visits IV, V, VI and IX (1 day to 12 months after treatment administration) |
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E.5.2 | Secondary end point(s) |
ALS disease progression (assessment of functional deterioration milestones) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At 3, 6, 9, 12 and 18 months following treatment administration. |
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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 | 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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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.8.5 | 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 | No |
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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The end of the trial is defined by 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 | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |