ClinicalTrials.gov record NCT05925023

Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

Sirolimus in the Treatment of Refractory/Relapsed Warm Autoimmune Hemolytic Anemia (AIHA)： a Phase 2 Prospective Trial

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Age18 years old to 90 years old
SexAll
LocationChina
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 Phase 2, single-group interventional trial tests oral sirolimus in adults (ages 18–90) with refractory or relapsed warm autoimmune hemolytic anemia or Evans syndrome. Participants take sirolimus 1–3 mg daily with target blood levels of 4–15 ng/mL for at least 6 months; responders may continue treatment for about a year and then taper. The study measures blood response rates and collects safety and relapse data at 3, 6, and 12 months.

What it is studying. Whether sirolimus produces partial or complete blood-count responses and what side effects and relapse rates occur in refractory/relapsed warm AIHA and Evans syndrome.

What the researchers hope to learn. Scientific goals: measure overall and complete response rates (partial or complete responses) at 3, 6, and 12 months; record treatment-related adverse events and count relapses over the 12‑month follow-up.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Autoimmune hemolytic anemia (AIHA) is a rare and heterogeneous disorder characterized by the destruction of red blood cells through warm or cold antibodies. Glucocorticoid (combined with rituximab) is the first-line treatment. However, the recurrence rate is very high and some patients may not respond to steroids. Second-line therapies include cyclosporine A (CsA), cyclophosphamide, rituximab, azathioprine, and even splenectomy. Our previous study of sirolimus in refractory/relapsed AIHA and ES found an effective rate of 80%. Therefore, the investigators plan to explore the efficacy and safety of sirolimus in the treatment of refractory/relapsed wAIHA.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Sirolimus

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.
  • Diagnosed as primary warm autoimmune hemolytic anemia or Evans syndrome (primary or secondary). There is no treatment indication of other systemic involvement in the original disease if secondary.
  • No response to glucocorticoid therapy or recurrence.
  • Baseline liver (ALT, AST) was less than 2 times the normal value.
  • No active infection; Not pregnant or breastfeeding.
  • Agree to sign the consent form.

Exclusion criteria

  • Patients with connective tissue disease or other organs involvement
  • Infection or bleeding that cannot be controlled by standard treatment.
  • Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
  • Progressed uncontrolled malignant tumors and lymphoma
  • Cirrhosis or portal hypertension.
  • Pregnant or breastfeeding.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

Based on the optimal autoantibody-RBC reactivity temperatures, AIHA is classified into warm type, cold type, and mixed type. AIHA can be further classified into primary or secondary in nature. Glucocorticoid (combined with rituximab) is the first-line treatment. However, the recurrence rate is very high and some patients may not respond to steroids. Second-line therapies include cyclosporine A (CsA), cyclophosphamide, rituximab, azathioprine, and even splenectomy. The refractory/relapsed wAIHA patients have increased cardiovascular events, increased opportunities for infections, decreased quality of life, and even death. A prospective multi-institutional trial in autoimmune cytopenia found that 8 of 10 patients with AIHA and Evans syndrome respond to sirolimus. Our previous study of sirolimus in refractory/relapsed AIHA and ES also found an effective rate of approximately 80%. Since sirolimus is cheap and accessible, our findings may reduce the economic burden of patients and be a guide on the selection of second-line treatment drugs in refractory/relapsed wAIHA and Evans syndrome.

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 will take oral sirolimus (1–3 mg per day) aiming for a blood level of 4–15 ng/mL for at least six months; those who respond typically continue treatment for about one year and then gradually reduce the dose. They will attend regular clinic visits with blood tests to check drug levels, treatment response, and side effects, with key assessments around 3, 6, and 12 months; baseline liver tests are required and liver function may be monitored for safety. All adverse events during or after treatment that the investigators consider related to the drug will be recorded. Participants must sign an informed consent form before joining.

How the study is structured. Single-group, open-label Phase 2 interventional study in which all participants receive oral sirolimus (no placebo or comparison arm). Treatment lasts at least 6 months with monitoring of blood counts and safety; key outcomes are assessed at 3, 6, and 12 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • 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

This trial is also listed on this site under Evans syndrome.

Clinical trials49 trials listed for autoimmune hemolytic anemiaEvery trial the registry lists for autoimmune hemolytic anemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in autoimmune hemolytic anemia researchLatest monthly update: September 2026. Includes: Negative DAT cases. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 24, 2023
Primary completion
December 2025 (estimated)
Study completion
December 2025 (estimated)
Target enrollment
22 participants (estimated)
Last updated on registry
Aug 8, 2025
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
Peking Union Medical College Hospital
Principal investigator
Chen Miao, associate professor
Peking Union Medical College Hospital

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

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.
refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
relapsed
A condition that improved with treatment and has since returned or worsened.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
standard treatment
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
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.

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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