Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-LRx
Status:
Recruiting
Trial end date:
2023-10-01
Target enrollment:
Participant gender:
Summary
The purpose is to evaluate the efficacy, safety, tolerability, pharmacokinetics and
pharmacodynamics of IONIS TMPRSS6-LRx administered subcutaneously to participants with
non-transfusion dependent β-Thalassemia Intermedia.