Sirolimus with or without cyclosporin A for Chinese adults with aplastic anemia who are refractory or intolerant to CsA

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This single-center, randomized, open-label phase II trial in Beijing randomizes participants 1:3 to receive sirolimus alone or sirolimus plus cyclosporin A to compare hematologic response and safety. Sirolimus is started at 1–3 mg once daily with a target trough concentration of 4–12 ng/mL, while cyclosporin A is given every 12 hours (typically 25–150 mg) with dose adjustments for renal function and trough levels (≈150 ng/mL for normal renal function). Patients are assessed for hematologic response rates and adverse events at 3 and 6 months after starting treatment. The trial enrolls adults with acquired aplastic anemia who are refractory or intolerant to standard-dose cyclosporin A.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Age ≥ 18 years;
2. Diagnosed with acquired aplastic anemia (AA), excluding congenital AA;
3. At least one of the following criteria met at enrollment: hemoglobin \< 100 g/L, platelets \< 50 × 10⁹/L, or neutrophils \< 1.0 × 10⁹/L;
4. At enrollment, meeting at least one of the following conditions:

   ① Cyclosporine A (CsA) ineffective: (CsA) used for at least 3 months without achieving partial response (PR), or disease relapse occurred;

   ② CsA intolerant: Unsuitable for standard dose CsA treatment due to adverse events or underlying conditions.
5. No active infections;
6. Not pregnant or breastfeeding;
7. Willing to sign the consent form;
8. Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.

Exclusion Criteria:

1. Pancytopenia caused by other reasons, such as myelodysplastic syndrome (MDS);
2. Evidence of clonal hematopoietic system bone marrow diseases (e.g., MDS or acute myeloid leukemia, AML);
3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
4. History of hematopoietic stem cell transplantation (HSCT) before enrollment;
5. Previous use of sirolimus or allergy to sirolimus;
6. Severe adverse events to CsA in the past, making it unsuitable for reuse;
7. Uncontrolled infection or bleeding with standard treatment;
8. Active infections with HIV, HCV, or HBV, liver cirrhosis, portal hypertension;
9. Any concurrent malignancy within the past 5 years, except for localized basal cell carcinoma of the skin;
10. History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid antibody syndrome), or current use of anticoagulants;
11. Pregnant or breastfeeding women;
12. Participation in other clinical trials within the past 3 months.

Where it's running

• Peking Union Medical College Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Ziwei Liu
• Email: liuzw10@163.com
• Phone: +8613811615392

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• BFCA conditioning for Haplo-HSCT in older adults with severe aplastic anemia

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• Adverse effects of ATG/ALG treatment in aplastic anemia

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• Lower-dose immunosuppression plus hetrombopag for severe aplastic anemia in people 65 and older

  This study tests whether a lower-dose immunosuppressive regimen combined with hetrombopag can improve blood counts and safety for people aged 65 and older with severe or very severe aplastic anemia.

  Recruiting Phase 2 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT07010237

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.