E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic Lateral Sclerosis (ALS) |
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E.1.1.1 | Medical condition in easily understood language |
Amyotrophic Lateral Sclerosis (ALS) |
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E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
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 |
To study safety and tolerability of subcutaneously administered ILB in patients diagnosed with ALS. |
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E.2.2 | Secondary objectives of the trial |
To evaluate efficacy of subcutaneously administered ILB in patients diagnosed with ALS. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Willing and able to give informed consent for participation in the study.
2. Clinical diagnosis of Amyotrophic Lateral Sclerosis.
3. Male or female patients between 18-80 years of age (inclusive).
4. Forced Vital Capacity (FVC) >/=65 % of predicted value for gender, height and age at screening.
5. Evaluated with ALSFRS-R and Norris clinical rating scales for at least the past 4 weeks before study drug administration.
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E.4 | Principal exclusion criteria |
1. Unable to understand information about the trial or are expected not to collaborate with the study team.
2. Concurrent serious disease, other than ALS.
3. Pregnancy.
4. Patients of child-bearing potential not willing to use adequate double contraception with <1 % failure rate after the screening visit until the last visit.
5. Addiction to drugs or alcohol.
6. Confirmed HIV, Hepatitis B or C.
7. Known bleeding disorders or abnormal bleeding events.
8. Treatment with anticoagulant drugs warfarin and novel oral anticoagulants (NOAC) within the last 14 days prior to screening.
9. Treatment with Riluzole or Lamotrigine within the last 28 days prior to study drug administration.
10. Hypersensitivity to dextran sulfate
11. Poor venous access.
12. Patients with clinically significant abnormal PK-INR, fibrinogen, von Willebrand factor and activated partial thromboplastin time (APTT) at screening |
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate safety by:
* Adverse events.
* Physical exam.
* Vital signs: blood pressure, heart rate, and body temperature.
* ECG assessment.
* Haematology and clinical chemistry lab tests.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At 9 visits over 4 months. |
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E.5.2 | Secondary end point(s) |
To evaluate efficacy by:
* ALSFRS-R functional rating scale.
* Norris functional rating scale.
* Plasma and CSF-analysis for biomarker Neurofilament light chain (NFL).
* Plasma, blood and serum analysis for biomarkers for neurological disorders.
* Respiratory function (FVC).
* Evaluation of quality of Life and autonomous symptoms.
* Levels of hepatocyte growth factor, pharmacokinetics and activated partial thromboplastin time. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At specific timepoints during up to 9 visits over several months. |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | 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 | No |
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 | 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
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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 | 5 |
E.8.9.1 | In the Member State concerned days | |