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AzTrials

Autoimmune Hemolytic Anemia (AIHA)sirolimusNCT07518277

A Multicenter, Prospective, Randomized Controlled Study Comparing Glucocorticoid Combined With Sirolimus With Monotherapy of Glucocorticoid in the Treatment of Newly Diagnosed Mild Autoimmune Hemolytic Anemia

A study testing Glucocorticoids in people with Autoimmune Hemolytic Anemia (AIHA), run by Chen Miao at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

This study is a prospective, multicenter, randomized controlled trial. A total of 216 adult patients with newly diagnosed wAIHA were planned to be included and randomly assigned in a 1:1 ratio to the experimental group (glucocorticoid combined with sirolimus) or the control group (glucocorticoid monotherapy). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Autoimmune Hemolytic Anemia (AIHA)
What is being tested?
Glucocorticoids
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
216 participants
Where
1 siteChina
Study dates
1 Apr 2026to 1 Apr 2028
Phase
Interventional
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years old, gender not limited;
  • Diagnosed as newly diagnosed wAIHA and requiring systemic immunosuppressive therapy (those with short-term hormone exposure of ≤2 weeks before enrollment can be included)
  • After a comprehensive rheumatology and immunology assessment, there is no evidence of clinical organ involvement in SLE or other connective tissue diseases (only serological abnormalities can be included).
  • Positive Coombs test (IgG type, IgG+C3d type, or only C3d type with a condensed agglutinin titer of 1:64);
  • Active hemolytic anemia, hemoglobin (HGB) ≤ 100 g/L;
  • Liver and kidney function: Alanine aminotransferase (ALT) < 3 times the upper limit of the normal value (ULN), isolated elevation of aspartate aminotransferase (AST) (normal ALT) is acceptable; Serum creatinine ≤2×ULN;
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  • Eastern Cooperative Oncology Group (ECOG) Performance status score ≤2 points;
  • Those with a history of malignant tumors must meet the following conditions:
  • Radical treatment has been achieved; The disease-free survival period complies with the protocol provisions (such as ≥1 year for basal cell carcinoma, ≥5 years for early solid tumors, ≥5 years for curable lymphoma, etc.); There is currently no evidence of recurrence. Those who do not meet the above conditions (such as CLL, advanced solid tumors, and active tumors) are not included.
  • Voluntarily sign a written informed consent form.

Who cannot join

  • Pregnant or lactating patients;
  • Clinically confirmed SLE or other definite connective tissue diseases;
  • Secondary AIHA secondary to lymphoproliferative diseases, other hematological malignancies, solid tumors, infections, and drugs;
  • Cold-resistant type or hybrid type AIHA;
  • Received before group enrollment:
  • Other immunosuppressants (such as rituximab, cyclosporine, etc., regardless of the duration); Glucocorticoid treatment for more than 2 weeks;
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  • Severe cardiac insufficiency (NYHA grade III/IV, or LVEF < 40%);
  • Currently active malignant tumors, or previous tumors that do not meet the requirements of Article 8 of the inclusion criteria;
  • Chronic active infections (active tuberculosis, active hepatitis B/C, etc.);
  • Severe immunodeficiency diseases, HIV infection (CD4⁺ < 200/μL), currently using other potent immunosuppressants, and using targeted biological agents within the last 3 months;
  • Have a history of severe allergy to sirolimus, glucocorticoids or related excipients;
  • Other circumstances where the researcher deems it inappropriate to participate.

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

  • Peking union medical college hospital

    Beijing, Shuangfuyuan, NO I., 100730

Other trials in China

About this study

This study is a prospective, multicenter, randomized controlled trial. A total of 216 adult patients with newly diagnosed wAIHA were planned to be included and randomly assigned in a 1:1 ratio to the experimental group (glucocorticoid combined with sirolimus) or the control group (glucocorticoid monotherapy). The initial dose of sirolimus in the experimental group was 1mg/d, adjusted according to the blood drug concentration. The target concentration was 4-12ng/mL, and the treatment course was 6 months. Both groups of hormones were gradually reduced according to the standard protocol. All patients were followed up for 24 months, and the differences between the two groups at endpoints such as the hormone-free sustained response rate at the 12th month were compared.

Read the detailed description

This study was a randomized controlled study. Two groups of patients were randomly assigned in a 1:1 ratio to receive the following treatment regimens:

  1. Glucocorticoid monotherapy group Prednisone at a dose of 1-2 mg/kg/d (or an equivalent dose of methylprednisolone) is taken orally for 2-3 weeks, and then the dosage is reduced regularly. The total course of treatment is approximately 3-6 months. The reduction plan was formulated by the researchers based on clinical practice.
  2. Glucocorticoid combined with sirolimus group Glucocorticoids: The usage, dosage and reduction regimen are the same as those in the monotherapy group. | Sirolimus: Starting dose 1 mg/d, orally, once a day. After reaching a steady state 7 to 14 days of medication, monitor the trough concentration. The target trough concentration range is 4 to 12 ng/mL. Adjust the dosage based on the results of blood drug concentration monitoring. The total course of treatment for sirolimus is 6 months. If grade ≥3 related adverse events or trough concentration \&gt occur; 12 ng/mL. Consider reducing the dosage or suspending the administration
Being tested
Glucocorticoids
Keywords
autoimmune hemolytic anemia
Medical subject headings
Anemia, Hemolytic, Autoimmune

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GlucocorticoidsDrug

    Glucocorticoids combined with sirolimus

Study groups in NCT07518277
Study groupRoleWhat they receive
GlucocorticoidActive comparatorGlucocorticoid monotherapy group Prednisone at a dose of 1-2 mg/kg/d (or an equivalent dose of methylprednisolone) is taken orally for 2-3 weeks, and then the dosage is reduced regularly. The total course of treatment is approximately 3-6 months. The reduction plan was formulated by the researchers based on clinical practice.
Receives: Drug: Glucocorticoids
Glucocorticoids combined with SirolimusExperimentalGlucocorticoids: The usage, dosage and reduction regimen are the same as those in the monotherapy group. | Sirolimus: Starting dose 1 mg/d, orally, once a day. After reaching a steady state 7 to 14 days of medication, monitor the trough concentration. The target trough concentration range is 4 to 12 ng/mL. Adjust the dosage based on the results of blood drug concentration monitoring. The total course of treatment for sirolimus is 6 months. If grade ≥3 related adverse events or trough concentration \&gt occur; 12 ng/mL. Consider reducing the dosage or suspending the administration.
Receives: Drug: Glucocorticoids

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The 12-month sustained remission rate

    Defined as the proportion of patients who simultaneously met the following conditions at the 12th month after randomization:

    Discontinue glucocorticoids (or only use prednisone ≤5 mg/d or an equivalent dose for maintenance);

    • Has not received any follow-up treatment for wAIHA;
    • Still in complete remission (CR) or partial remission (PR) status.

    Measured: 12 months

Secondary outcomes (1)

  1. Safety

    1. The incidence of adverse events (AE), serious adverse events (SAE), and grade ≥3 AE;
    2. Adverse events of particular concern: infections (especially opportunistic infections), metabolic disorders (new-onset diabetes, dyslipidemia), bleeding events, hormone-related adverse events (Cushing's syndrome, hypertension, osteoporosis, etc.);
    3. The proportion of patients who discontinued medication, reduced dosage or interrupted treatment due to adverse events

    Measured: 1，3，6，12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Apr 2026
  2. Study start1 Apr 2026 (estimated)
  3. Primary completion1 Apr 2028 (estimated)
  4. Study completion1 Apr 2028 (estimated)

Resources

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 N/A
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
216 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07518277
Sponsor's protocol ID
GC vs. GC+Sirolimus
Acronym
sirolimus
Status last verified
Mar 2026
First submitted
30 Mar 2026
Last updated
8 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 1.Birgens H, Frederiksen H, Hasselbalch HC, et al. A phase III randomized trial comparing glucocorticoid monotherapy versus glucocorticoid and rituximab in patients with autoimmune haemolytic anaemia. Br J Haematol. 2013;163(2):244-252.https://doi.org/10.1111/bjh.12541. 2.Kuter DJ. Warm autoimmune hemolytic anemia and the best treatment strategies. Hematology Am Soc Hematol Educ Program. 2022;2022(1):393-402.https://doi.org/10.1182/hematology.2022000405. 3.Murakhovskaya I, Crivera C, Leon A,et al. Healthcare resource utilization of patients with warm autoimmune hemolytic anemia initiating first line therapy of oral corticosteroids with or without rituximab. Ann Hematol. 2024;103(2):521-533.https://doi.org/10.1007/s00277-023-05613-8. 4.Berentsen S. Treatment of autoimmune hemolytic anemia: novel and investigational approaches. Minerva Med. 2025;116(2):145-158.https://doi.org/10.23736/S0026-4806.25.09617-X. 5.Ciudad M, Ouandji S, Lamarthée B, et al. Regulatory T-cell dysfunctions are associated with increase in tumor necrosis factor α in autoimmune hemolytic anemia and participate in polarization. Haematologica. 2023;108(11):3012-3025. https://doi.org/10.3324/haematol.2023.282859. 6.中华医学会血液学分会红细胞疾病（贫血）学组. 中国成人自身免疫性溶血性贫血诊疗指南（2023年版）[J]. 中华血液学杂志,2023,44(01)：12-18. DOI:10.3760/cma.j.issn.0253-2727.2023.01.003

Questions about this trial

What is this clinical trial studying?

A study testing Glucocorticoids in people with Autoimmune Hemolytic Anemia (AIHA), run by Chen Miao 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: opening soon, as of the sponsor's last update on 8 Apr 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.

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?

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?

The 12-month sustained remission rate (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07518277, which also lists 1 linked publication.

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 8 Apr 2026.

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