A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SL… (NCT04718844) | Clinical Trial Compass
CompletedPhase 1
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
Germany, Israel44 participantsStarted 2021-04-14
Plain-language summary
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.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* Adult with alpha- or beta-thalassaemia or compound heterozygous haemoglobin E/beta-thalassaemia or adult with very low- or low-risk MDS according to the 2016 revision to the World Health Organisation classification.
* All subjects must agree to adhere to appropriate contraception requirements.
* Subjects must provide written informed consent and be able to comply with all study requirements.
* Body mass index ≥18 kg/m2 and ≤35 kg/m2 at screening.
* At least one of: a) Mean ferritin \>250 μg/L based on a minimum of 2 measurements ≥1 week apart within 20 days before the planned dosing day, in the absence of active significant infection; b) Mean TSAT \>40% measured on a minimum of 2 occasions ≥1 week apart within 20 days before the planned dosing day; c) Liver iron \>3 mg Fe/g dry weight, measured according to local procedures.
* Mean baseline haemoglobin concentration ≥5 g/dL and ≤11 g/dL, based on a minimum of 2 measurements ≥1 week apart, within 20 days before the planned dosing day.
Exclusion criteria
* Adult with haemoglobin S/alpha-thalassaemia or haemoglobin S/beta-thalassaemia or adult with secondary MDS, i.e., MDS that is known to have arisen because of chemical injury or treatment with chemotherapy and/or radiation for another disease.
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc, or intolerance to s.c. injections.
* Known infection with HIV, or active infectious hepatitis A, B, or C virus…