Actively Recruiting
Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A
Led by Peking Union Medical College Hospital · Updated on 2025-06-04
40
Participants Needed
1
Research Sites
100 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosed with acquired aplastic anemia, excluding congenital aplastic anemia
- Hemoglobin level below 100 g/L, or platelet count below 50 x 10⁹/L, or neutrophil count below 1.0 x 10⁹/L at enrollment
- Either cyclosporin A treatment ineffective after at least 3 months without partial response or disease relapse, or intolerance to standard dose cyclosporin A due to side effects or underlying conditions
- No active infections
- Not pregnant or breastfeeding
- Willing to sign the consent form
- Eastern Cooperative Oncology Group (ECOG) performance status score between 0 and 2
You will not qualify if you...
- Pancytopenia caused by conditions other than aplastic anemia, such as myelodysplastic syndrome
- Evidence of clonal bone marrow diseases like myelodysplastic syndrome or acute myeloid leukemia
- Paroxysmal nocturnal hemoglobinuria clone 50% or greater
- History of hematopoietic stem cell transplantation before enrollment
- Previous use of sirolimus or allergy to sirolimus
- Severe adverse events from cyclosporin A making reuse unsuitable
- Uncontrolled infection or bleeding despite standard treatment
- Active infections with HIV, hepatitis C virus, or hepatitis B virus, liver cirrhosis, or portal hypertension
- Any cancer in the past 5 years except localized basal cell carcinoma of the skin
- History of blood clots, heart attack, stroke, or current use of blood thinners
- Pregnant or breastfeeding women
- Participation in other clinical trials within the last 3 months
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
Research Team
Z
Ziwei Liu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here