Aplastic AnaemiaPhase 2NCT06802055
Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A
A Phase 2 study testing Sirolimus (Rapamune®) and Cyclosporin A (CsA) in people with Aplastic Anaemia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anaemia
- What is being tested?
- Sirolimus (Rapamune®) and Cyclosporin A (CsA)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 25 Jan 2025to Jan 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- No active infections;
- Not pregnant or breastfeeding;
- Willing to sign the consent form;
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Ziwei Liu, +8613811615392, liuzw10@163.com
About this study
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
- Aplastic Anaemia
- Being tested
- Sirolimus (Rapamune®), Cyclosporin A (CsA)
- Keywords
- Sirolimus, Relapsed/refractory aplastic anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sirolimus (Rapamune®)Drug
Sirolimus 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml.
Cyclosporin A (CsA)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus + cyclosporin A | Experimental | Not described Receives: Drug: Sirolimus (Rapamune®); Drug: Cyclosporin A (CsA) |
| Sirolimus | Placebo comparator | Not described Receives: Drug: Sirolimus (Rapamune®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR) at 3 months
Overall Response Rate (ORR) is defined as the number of participants who meet the criteria of either complete response (CR) or partial response (PR)
Measured: Week 12
Secondary outcomes (5)
Overall response rate (ORR) at 6 months
ORR will be evaluated after 6 months of treatment by measuring platelet, reticulocyte, hemoglobin, neutrophil and transfusion independence.
Measured: Week 24
Changes in Hemoglobin in the Absence of Red Blood Cells Transfusion
The change in hematology values (hemoglobin) were evaluated
Measured: Week 12
Changes in Platelet in the Absence of Platelet Transfusion
The change in hematology values (platelet) were evaluated
Measured: Week 12
Duration of hematologic response
Time from the date of the start of the first response to the date of first relapse defined as again meeting criteria for aplastic anemia
Measured: by 6 months (all patients), at 12 months (responders only)
Percentage of patients with clonal evolution to myelodysplasia, PNH, and acute leukemia
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2025
- Study start25 Jan 2025
- Primary completionJan 2026 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnaemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06802055
- Sponsor's protocol ID
- I-25PJ0215
- Status last verified
- May 2025
- First submitted
- 25 Jan 2025
- Last updated
- 4 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data would be accepted upon request
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- Acronym / sponsor ID
- I-25PJ0215
- Conditions
- Aplastic Anaemia
- Keywords
- Sirolimus, Relapsed/refractory aplastic anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 25 Jan 2025 (actual)
- Primary completion
- Jan 2026 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 30 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sirolimus + cyclosporin A | Experimental | Drug: Sirolimus (Rapamune®), Drug: Cyclosporin A (CsA) |
| Sirolimus | Placebo comparator | Drug: Sirolimus (Rapamune®) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sirolimus (Rapamune®)Sirolimus 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng/ml. | Drug | Sirolimus, Sirolimus + cyclosporin A |
| 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. | Drug | Sirolimus + cyclosporin A |
Primary outcome
Overall response rate (ORR) at 3 months
Time frame: Week 12
Overall Response Rate (ORR) is defined as the number of participants who meet the criteria of either complete response (CR) or partial response (PR)
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2025.
- Individual participant dataSharing planned: Yes. Individual participant data would be accepted upon request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus (Rapamune®) and Cyclosporin A (CsA) in people with Aplastic Anaemia, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ziwei Liu (+8613811615392, liuzw10@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) at 3 months (time frame: Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2025.