Phase 1b Study of DISC-3405 Safety and Effects in Adults With Sickle Cell Disease

What this Trial Is About

Researchers are conducting an open-label, multicenter study to evaluate DISC-3405 in adults with sickle cell disease SCD. The study examines up to three different dose levels of DISC-3405 to assess its safety, tolerability, pharmacokinetics PK, and pharmacodynamics PD. This Phase 1b trial focuses on participants with SCD, including those with certain complications related to the disease. In this study, DISC-3405 is given by subcutaneous injection in a within-participant dose-escalation design, meaning each participant may receive increasing doses over time. The study examines up to three dose levels sequentially to evaluate how the body processes the drug and how the drug affects blood markers related to SCD. The treatment period can last up to 36 weeks, during which safety and PD measures are closely monitored. Participants will be involved throughout the treatment period with regular assessments including blood tests to measure hemoglobin, hematocrit, reticulocyte count, red blood cell count, lactate dehydrogenase, bilirubin levels, and drug concentrations in blood. Safety and tolerability are primary concerns, with follow-up evaluations up to 36 weeks. Participants must comply with study procedures and provide informed consent before enrollment, ensuring they understand the studys aims and requirements.

A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)