This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c. doses and multiple doses in healthy male and female subjects. Up to 7 cohorts of 56 patients with Thalassaemia and up to 7 cohorts of 56 patients with MDS will be enrolled. Each subject will receive single or multiple doses of SLN124 or placebo given by subcutaneous (s.c) injection.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
44
SLN124 for subcutaneous (s.c.) injection
Sodium chloride for s.c. injection
Universitaetsklinikum Duesseldorf
Düsseldorf, Germany
Universitat Leipzig
Leipzig, Germany
Rambam Health Care Campus
Haifa, Israel
Sheba Medical Center
Ramat Gan, Israel
Bar-Ilan University - Faculty of Medicine
Safed, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
AUSL della Romagna - Ospedale di Ravenna
Ravenna, Italy
Azienda Unità Sanitaria Locale - IRCCS di Reggio Emilia
Reggio Emilia, Italy
Jordan University Hospital
Amman, Jordan
King Hussein Cancer Center
Amman, Jordan
...and 10 more locations
Incidence of treatment-emergent adverse events
safety and tolerability will be reported separately following single-dose administration.
Time frame: Day 84
Incidence of treatment-emergent adverse events
safety and tolerability will be reported separately following multi-dose administration.
Time frame: Day 140
Pharmacokinetic: peak plasma concentration (Cmax)
Will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacokinetic: area under the plasma concentration (AUC)
Will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacokinetic: apparent total clearance from plasma after s.c injection (CL/F)
Will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacodynamic biomarkers: Change in TSAT after s.c injection.
safety and tolerability will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacodynamic biomarkers: Change in hepcidin after s.c injection.
safety and tolerability will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacodynamic biomarkers: Change in serum iron after s.c injection.
safety and tolerability will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
Pharmacodynamic biomarkers: Change in haemoglobin after s.c injection.
safety and tolerability will be reported separately following single-dose and multiple-dose administration.
Time frame: Day 84 and Day 140
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