The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
IMP will be provided as a solution to be injected by qualified clinical staff in patient's abdomen, upper arms or front of the thighs.
MHAT Dr. Nikola Vasiliev AD
Kyustendil, Bulgaria
UMHAT Dr. Georgi Stranski AD
Pleven, Bulgaria
Medical Center COMAC MEDICAL
Sofia, Bulgaria
UMHAT Sv. Ivan Rilski
Sofia, Bulgaria
Hammersmith Hospital
# of participants with all AEs as assessed by CTCAE V4.0 included injection site reaction, will be measured from baseline to post dose follow up
Time frame: Up to two months
12 lead electrocardiogram parameters included PR, QTcF and QTcB will be measured from baseline to post dose follow up
Time frame: Up to two months
clinical chemistry, haematology, urinalysis, liver function tests (included ALT, AST, GGT) will be measured from baseline to post dose follow up
Time frame: Up to two months
height, weight, blood pressure, heart rate, respiration rate and temperature will be measured from baseline to post dose follow up
Time frame: Up to two months
Biomarkers will be measured from baseline to post dose follow up
serum hepcidin level (ng/ml), ferritin level (ug/L), Transferrin saturation (%), iron level (umol/L), haemoglobin and reticulocyte count will be analysed by central laboratory.
Time frame: Up to two months
Pharmacokinetic of SLN124 in plasma from baseline to post dose follow up
Peak Plasma Concentration (Cmax) will be analysed by central laboratory.
Time frame: Up to two months
Pharmacokinetic of SLN124 in plasma from baseline to post dose follow up
Area under the plasma concentration versus time curve (AUC) will be analysed by central laboratory.
Time frame: Up to two months
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London, United Kingdom