Phase II Study Comparing Sirolimus Alone or With Cyclosporin A in Chinese Adults With Aplastic Anemia Resistant to Cyclosporin A

What this Trial Is About

Researchers are evaluating the effectiveness and safety of sirolimus alone compared to sirolimus combined with cyclosporin A CsA in Chinese adults with aplastic anemia who do not respond to or cannot tolerate CsA. This phase II, randomized, open-label study focuses on patients who have failed CsA treatment and aims to understand how these treatments impact blood cell counts and patient safety. Participants are randomly assigned in a 13 ratio to receive either sirolimus alone or sirolimus with CsA. Sirolimus is taken orally once daily at doses of 1 to 3 mg, targeting a specific blood concentration. CsA is administered orally every 12 hours, with doses adjusted based on kidney function and blood levels. For those with normal kidney function, the target CsA blood concentration is about 150 ngml, while those with impaired function receive lower doses to help stabilize or improve kidney health. The treatment response and safety are evaluated at 3 and 6 months after starting therapy. During the study, participants will be closely monitored through blood tests to assess response, including hemoglobin and platelet levels without transfusions. Researchers will record the overall response rate at 3 months and assess longer-term outcomes at 6 and 12 months, including any progression to other blood disorders. Safety and adverse events will also be tracked throughout. The entire participation period includes these assessments and monitoring to evaluate how well the treatments work and their safety profile.

Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A