ClinicalTrials.gov record NCT07518277
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 Multicenter, Prospective, Randomized Controlled Study Comparing Glucocorticoid Combined With Sirolimus With Monotherapy of Glucocorticoid in the Treatment of Newly Diagnosed Mild Autoimmune Hemolytic Anemia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This randomized trial plans to enroll adults with newly diagnosed warm autoimmune hemolytic anemia who need systemic immunosuppressive treatment. Participants are assigned to standard glucocorticoid therapy or glucocorticoid plus sirolimus (sirolimus starting 1 mg/day with blood-level monitoring, target trough 4–12 ng/mL for 6 months). All patients are followed for 24 months; the main outcome is sustained remission at 12 months (off glucocorticoids or on a very low dose, prednisone <=5 mg/day) and safety is recorded.
What it is studying. The study compares adding sirolimus to standard glucocorticoid treatment versus glucocorticoid alone, measuring the rate of sustained remission at 12 months, which can include people who have stopped glucocorticoids or are taking only a low maintenance dose (prednisone ≤5 mg/day or equivalent) and assessing safety outcomes.
What the researchers hope to learn. To compare the 12-month sustained remission rate between the two groups — the study’s remission endpoint allows glucocorticoids to have been stopped or reduced to a low maintenance dose (prednisone ≤5 mg/day or equivalent) and to assess safety (adverse events, serious adverse events, infections, metabolic problems, and treatment discontinuation related to side effects).
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Glucocorticoids
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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;
- 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.
Exclusion criteria
- 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;
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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:
- 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.
- 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 \> occur; 12 ng/mL. Consider reducing the dosage or suspending the administration
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomly assigned to one of two oral treatment plans: prednisone (1–2 mg/kg/day for 2–3 weeks, then a taper over about 3–6 months) alone, or the same prednisone regimen plus sirolimus (starting 1 mg once daily). If assigned sirolimus, blood will be drawn to measure trough drug levels about 7–14 days after starting (target 4–12 ng/mL) and periodically thereafter; dose adjustments or temporary stopping may occur for high levels or serious side effects. Study follow-up includes safety checks and outcome assessments over 24 months, with closer safety reviews at months such as 1, 3, 6 and 12; blood tests and clinical visits are used to monitor response and adverse events. Severe adverse events may lead to medication dose reduction or discontinuation per the study protocol.
How the study is structured. Interventional, randomized, open-label (no masking), parallel-group drug trial. Two treatment arms: glucocorticoid monotherapy versus glucocorticoid plus oral sirolimus with dose adjusted by trough blood levels. Sirolimus treatment is planned for 6 months; participants are followed for up to 24 months with scheduled safety and response assessments.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Apr 1, 2026 (estimated)
- Primary completion
- Apr 1, 2028 (estimated)
- Study completion
- Apr 1, 2028 (estimated)
- Target enrollment
- 216 participants (estimated)
- Last updated on registry
- Apr 8, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Chen Miao
Participating sites (1)
- Peking union medical college hospital
Beijing, Shuangfuyuan, NO I., 100730 · China
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- 1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- glucocorticoid
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- control group
- The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
- endpoints
- Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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