Autoimmune AnemiaPhase 4NCT04324411
Sirolimus Combined With ATRA for the Treatment of Auto-Immune Anemia
A Phase 4 study testing sirolimus and ATRA and sirolimus in people with Autoimmune Anemia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
Autoimmune anemia (AIA), including autoimmune hemolytic anemia (AIHA), EVENs' syndrome (ES), acquired pure red aplastic anemia (PRCA), is a kind of anemia disease mediated by autoimmunity, which can be primary or secondary to other diseases including autoimmune disease, malignant tumor, infection, etc. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Autoimmune Anemia
- What is being tested?
- sirolimus and ATRA and sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteChina
- Study dates
- 20 Apr 2020to 1 Sep 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosed as autoimmune anemia (autoimmune hemolytic anemia, pure red aplastic anemia, events syndrome) without organ complications;
- ineffective, relapsed or intolerant patients who have been treated with at least one kind of sufficient current conventional drug (steroids, CsA, CD20 monoclonal antibody, tacrolimus and others) ;
- normal cardiac function, liver function (ALT/AST ≤ 3.0 × ULN) and renal function (serum creatinine ≤ 1 × ULN);
- no secondary disease;
- unable to accept hematopoietic stem cell transplantation;
- ECoG score ≤ 2;
- able to sign the informed consent form.
Who cannot join
- failure to make a definite diagnosis;
- AIA secondary to known diseases such as systemic lupus erythematosus, rheumatoid arthritis, tumor or other inflammatory diseases;
- severe hepatorenal insufficiency (creatinine, transaminase more than 3 times of the upper limit of normal value);
- uncontrollable systemic infection or other serious diseases;
- pregnant or lactating women;
- patients with mental disease who are unable to sign the informed consent;
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- taking other AIA drugs or stopping the drugs for less than 3 months;
- allergic to the study drug;
- participation in other clinical studies;
- patients in any other circumstances considered unsuitable by the investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Sirolimus Combined With All Trans Retinoic Acid for the Treatment of Auto-Immune Anemia
Autoimmune anemia (AIA), including autoimmune hemolytic anemia (AIHA), EVENs' syndrome (ES), acquired pure red aplastic anemia (PRCA), is a kind of anemia disease mediated by autoimmunity, which can be primary or secondary to other diseases including autoimmune disease, malignant tumor, infection, etc. Glucocorticoid is the first-line treatment. However, the recurrence rate is very high and some patients may not response to steroids, the latter defined as refractory autoimmune anemia (RAIA). Second-line therapies include cyclosporine A (CSA), cyclophosphamide, 6-mercaptopurine, CD20 monoclonal antibody, anti human lymphocyte immunoglobulin (ATG), and even splenectomy. Cyclosporine A is easy to accept while some patients may have side effects such as renal function damage, gingival hyperplasia, hypertension and so on. Other second-line drugs also have many problems, such as low effective rate, slow onset, expensive price, and large side effects, and some patients do not response to these treatments. The refractory/relapsed AIA patients have increased cardiovascular events, increased opportunities for infections, decreased quality of life, and even death. At present, there is still no effective treatment for these patients. Our previous retrospective study showed that sirolimus was effective in cyclosporine refractory PRCA with an effective rate of 70% and slight side effects. In addition, we used sirolimus in refractory AIHA and ES, with an effective rate of 60-70%. However, there are still some non-responsive patients. Recently, it has been reported that all trans retinoic acid (ATRA) combined with danazol was effective in the treatment of refractory immune thrombocytopenic purpura (ITP). Therefore, we plans to combine sirolimus and ATRA in the treatment of refractory AIA to improve the efficacy. Since both sirolimus and ATRA are cheap and have slight side effects, this combination may reduce the economic burden of patients and reduce the side effects related to treatment.
- Conditions
- Autoimmune Anemia
- Being tested
- sirolimus and ATRA, sirolimus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sirolimus and ATRADrug
sirolimus (serum concentration 4-10ng/ml) and ATRA 20mg bid
sirolimusDrug
Since ATRA is not available in China for a variety of reasons, it was only used for less than 6 months. Then, sirolimus (serum concentration 4-10ng/ml) monotherapy was used.
| Study group | Role | What they receive |
|---|---|---|
| treatment group | Experimental | combined sirolimus(serum concentration to be 4-10ng/ml) and ATRA (20mg bid) for at least 6 months sirolimus Receives: Drug: sirolimus and ATRA; Drug: sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response rate
overall response rate
Measured: 1 year
rate of side effects
rates and types of all side effects
Measured: 1 year
Secondary outcomes (5)
change of HGB concentration
change of HGB concentration
Measured: through study completion, an average of 1 year
frequency of HGB transfusion
frequency of HGB transfusion
Measured: through study completion, an average of 1 year
time to response
time to response
Measured: through study completion, an average of 1 year
response duration
CR/PR duration
Measured: through study completion, an average of 1 year
change of bilirubin concentration
change of bilirubin concentration
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2020
- Study start20 Apr 2020
- Primary completion1 May 2023 (estimated)
- Study completion1 Sep 2023 (estimated)
Why it stopped: The study has not actually been conducted.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune AnemiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04324411
- Sponsor's protocol ID
- sirolimus-3
- Status last verified
- Jul 2026
- First submitted
- 23 Mar 2020
- Last updated
- 7 Jul 2026
- 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 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sirolimus Combined With All Trans Retinoic Acid for the Treatment of Auto-Immune Anemia
- ClinicalTrials.gov ID
- NCT04324411
- Acronym / sponsor ID
- sirolimus-3
- Conditions
- Autoimmune Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study has not actually been conducted.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 20 Apr 2020 (actual)
- Primary completion
- 1 May 2023 (estimated)
- Study completion
- 1 Sep 2023 (estimated)
- First posted
- 27 Mar 2020 (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.
Autoimmune anemia (AIA), including autoimmune hemolytic anemia (AIHA), EVENs' syndrome (ES), acquired pure red aplastic anemia (PRCA), is a kind of anemia disease mediated by autoimmunity, which can be primary or secondary to other diseases including autoimmune disease, malignant tumor, infection, etc. Glucocorticoid is the first-line treatment. However, the recurrence rate is very high and some patients may not response to steroids, the latter defined as refractory autoimmune anemia (RAIA). Second-line therapies include cyclosporine A (CSA), cyclophosphamide, 6-mercaptopurine, CD20 monoclonal antibody, anti human lymphocyte immunoglobulin (ATG), and even splenectomy. Cyclosporine A is easy to accept while some patients may have side effects such as renal function damage, gingival hyperplasia, hypertension and so on. Other second-line drugs also have many problems, such as low effective rate, slow onset, expensive price, and large side effects, and some patients do not response to these treatments. The refractory/relapsed AIA patients have increased cardiovascular events, increased opportunities for infections, decreased quality of life, and even death. At present, there is still no effective treatment for these patients. Our previous retrospective study showed that sirolimus was effective in cyclosporine refractory PRCA with an effective rate of 70% and slight side effects. In addition, we used sirolimus in refractory AIHA and ES, with an effective rate of 60-70%. However, there are still some non-responsive patients. Recently, it has been reported that all trans retinoic acid (ATRA) combined with danazol was effective in the treatment of refractory immune thrombocytopenic purpura (ITP). Therefore, we plans to combine sirolimus and ATRA in the treatment of refractory AIA to improve the efficacy. Since both sirolimus and ATRA are cheap and have slight side effects, this combination may reduce the economic burden of patients and reduce the side effects related to treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment group | Experimental | Drug: sirolimus and ATRA, Drug: sirolimuscombined sirolimus(serum concentration to be 4-10ng/ml) and ATRA (20mg bid) for at least 6 months sirolimus |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- diagnosed as autoimmune anemia (autoimmune hemolytic anemia, pure red aplastic anemia, events syndrome) without organ complications;
- ineffective, relapsed or intolerant patients who have been treated with at least one kind of sufficient current conventional drug (steroids, CsA, CD20 monoclonal antibody, tacrolimus and others) ;
- normal cardiac function, liver function (ALT/AST ≤ 3.0 × ULN) and renal function (serum creatinine ≤ 1 × ULN);
- no secondary disease;
- unable to accept hematopoietic stem cell transplantation;
Key exclusion criteria (10 listed)
- failure to make a definite diagnosis;
- AIA secondary to known diseases such as systemic lupus erythematosus, rheumatoid arthritis, tumor or other inflammatory diseases;
- severe hepatorenal insufficiency (creatinine, transaminase more than 3 times of the upper limit of normal value);
- uncontrollable systemic infection or other serious diseases;
- pregnant or lactating women;
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 and ATRAsirolimus (serum concentration 4-10ng/ml) and ATRA 20mg bid | Drug | treatment group |
| sirolimusSince ATRA is not available in China for a variety of reasons, it was only used for less than 6 months. Then, sirolimus (serum concentration 4-10ng/ml) monotherapy was used. | Drug | treatment group |
Primary outcome
overall response rate
Time frame: 1 year
overall response rate
rate of side effects
Time frame: 1 year
rates and types of all side effects
5 secondary outcomes
change of HGB concentration
Time frame: through study completion, an average of 1 year
frequency of HGB transfusion
Time frame: through study completion, an average of 1 year
time to response
Time frame: through study completion, an average of 1 year
response duration
Time frame: through study completion, an average of 1 year
change of bilirubin concentration
Time frame: through study completion, an average of 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing sirolimus and ATRA and sirolimus in people with Autoimmune Anemia, 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 7 inclusion and 10 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: withdrawn, as of the sponsor's last update on 7 Jul 2026. 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall response rate (time frame: 1 year). The study lists 2 primary outcomes in all. 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 7 Jul 2026.