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Tests treatment safety and results for Aplastic Anaemia
Official title Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A
ClinicalTrials.gov ID: NCT06802055
What this study is testing
What is Sirolimus (Rapamune®)?
Sirolimus (Rapamune®) is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for aplastic anaemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- No active infections;
You likely can't join if
- 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;
See the full eligibility 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...
- No active infections;
- Not pregnant or breastfeeding;
- Willing to sign the consent form;
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.