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Recruiting NCT06802055

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

Phase II Interventional Aplastic Anaemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Sirolimus (Rapamune®), Cyclosporin A (CsA).
Who it may be relevant to
Registry conditions: Aplastic Anaemia. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

This is a single center, randomized, open-label, phase II study to compare the efficacy of sirolimus combined with cyclosporin A (CsA) to sirolimus alone in Chinese subjects with aplastic anemia refractory/intolerant to CsA. The safety would also be evaluated. Patients would be randomized to receive sirolimus alone or sirolimus combined with CsA at a 1:3 ratio. Treatment with sirolimus will be started at 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml. CsA will be given at 25-150 mg orally every 12 hours, with the dose adjusted based on renal function and trough concentration. For patients with normal renal function, the target trough concentration is approximately 150 ng/ml. For patients with impaired renal function, the cyclosporine A dose is reduced to 25-50 mg every 12 hours, aiming for recovery or stabilization of renal function. The hematological response rate and safety will be recorded and compared at 3 and 6 months after starting the study treatment (Week 13 and 25).

Interventions

  • Drug Sirolimus (Rapamune®)
    Sirolimus 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml.
  • Drug Cyclosporin A (CsA)
    Cyclosporine A 25-150 mg orally every 12 hours, with the dose adjusted based on renal function and trough concentration. For patients with normal renal function, the target trough concentration is approximately 150 ng/ml. For patients with impaired renal function, the cyclosporine A dose is reduced to 25-50 mg every 12 hours, aiming for recovery or stabilization of renal function.

Primary outcome measures

  • Overall response rate (ORR) at 3 months [Time frame: Week 12]
Secondary outcome measures (5)
  • Overall response rate (ORR) at 6 months [Time frame: Week 24]
  • Changes in Hemoglobin in the Absence of Red Blood Cells Transfusion [Time frame: Week 12]
  • Changes in Platelet in the Absence of Platelet Transfusion [Time frame: Week 12]
  • Duration of hematologic response [Time frame: by 6 months (all patients), at 12 months (responders only)]
  • Percentage of patients with clonal evolution to myelodysplasia, PNH, and acute leukemia [Time frame: 12 months]

Eligibility criteria

Inclusion criteria

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

  • No active infections;
  • Not pregnant or breastfeeding;
  • Willing to sign the consent form;
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.

Exclusion criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Peking Union Medical College Hospital — Beijing

Identifiers

NCT: NCT06802055 · I-25PJ0215

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗