Linperlisib in the Treatment of aPRCA

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · Jun 12, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medicine called Linperlisib to treat a rare blood condition called acquired pure red cell aplasia (aPRCA). In aPRCA, the body stops making enough red blood cells, which can cause anemia and tiredness. Current treatments sometimes work but can have problems like relapses or take a long time to help. Linperlisib is a new type of drug that targets a specific part of the immune system thought to be involved in causing aPRCA. Researchers want to see if Linperlisib is safe and effective for people whose aPRCA has not improved after trying at least two other treatments.

People who might be eligible for this study are adults aged 18 or older who have been diagnosed with aPRCA and have not responded well to previous treatments. They need to have low hemoglobin levels (a measure related to red blood cells) and be willing to follow the study rules, including stopping some other medicines before joining. The trial is not yet recruiting participants, but those who join can expect their response to Linperlisib to be closely monitored for both benefits and side effects. It’s important for potential participants to discuss with their doctors whether joining this study fits their health needs.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Age ≥ 18 years;
• • Patients meeting the diagnostic criteria for acquired PRCA;
• • Patients who are relapsed or refractory after at least two lines of prior therapy. Refractory is defined as failure to achieve partial remission after 3 months of treatment at a stable dose of immunosuppressive agents.
• • Hb ≤ 100 g/L;
• • Patients with complete clinical data, demonstrated good treatment compliance, and who have signed the informed consent form;
• • If taking glucocorticoids, must have discontinued them or been on a stable low maintenance dose (prednisone ≤ 15 mg/day) for at least 2 weeks prior to enrollment, and continue this dose;
• • If taking immunosuppressive agents such as cyclosporine or sirolimus, must have been on a stable dose for at least 3 months, and discontinue them upon enrollment.
• Exclusion Criteria:
• • Patients with organ dysfunction (e.g., heart, liver, or lung) or acute renal insufficiency;
• • Patients who have used PI3Kδ inhibitors within the past 6 months;
• • Patients with severe infectious diseases;
• • Patients with malignant tumors;
• • Patients with psychiatric disorders or cognitive impairment;
• • Pregnant or lactating women;
• • Patients who have participated in other clinical trials within the past 3 months.