NCT05925023

Brief Summary

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.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
22

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jun 2023

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 21, 2023

Completed
3 days until next milestone

Study Start

First participant enrolled

June 24, 2023

Completed
5 days until next milestone

First Posted

Study publicly available on registry

June 29, 2023

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2025

Completed
Last Updated

August 8, 2025

Status Verified

August 1, 2025

Enrollment Period

2.4 years

First QC Date

June 21, 2023

Last Update Submit

August 6, 2025

Conditions

Warm Autoimmune Hemolytic Anemia

Keywords

warm autoimmune hemolytic anemiaEvans syndromerefractory/relapsedsirolimusefficacy

Outcome Measures

Primary Outcomes (1)

  • Overall response rate (ORR) and complete response rate (CRR)

    ORR defined as the proportion of patients who met the criteria of either complete response (CR) or partial response (PR).

    3,6,12 months

Secondary Outcomes (3)

  • Number of participants with treatment-related adverse events and the number of relapses

    12 months, end of the follow-up

  • Adverse events

    3,6,12 months

  • Relapse rate

    3,6,12 months

Study Arms (1)

Sirolimus on refractory/relapsed wAIHA

EXPERIMENTAL

A prospective research of the sirolimus efficiency on refractory/relapsed primary wAIHA patients. Sirolimus dosage: 1-3 mg/d with plasma concentration 4-15ng/mL. Medication time should last at least 6 months. After reaching the optimal response, responders continue to use sirolimus for 1 year, and then gradually reduce the dosage.

Drug: Sirolimus

Interventions

SirolimusDRUG

Oral administration, 1-3 mg/d, sirolimus plasma concentration: 4-15 ng/mL

Sirolimus on refractory/relapsed wAIHA

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Related Publications (8)

  • Jager U, Barcellini W, Broome CM, Gertz MA, Hill A, Hill QA, Jilma B, Kuter DJ, Michel M, Montillo M, Roth A, Zeerleder SS, Berentsen S. Diagnosis and treatment of autoimmune hemolytic anemia in adults: Recommendations from the First International Consensus Meeting. Blood Rev. 2020 May;41:100648. doi: 10.1016/j.blre.2019.100648. Epub 2019 Dec 5.

    PMID: 31839434BACKGROUND

  • Bass GF, Tuscano ET, Tuscano JM. Diagnosis and classification of autoimmune hemolytic anemia. Autoimmun Rev. 2014 Apr-May;13(4-5):560-4. doi: 10.1016/j.autrev.2013.11.010. Epub 2014 Jan 11.

    PMID: 24418298BACKGROUND

  • Hill QA, Stamps R, Massey E, Grainger JD, Provan D, Hill A; British Society for Haematology. The diagnosis and management of primary autoimmune haemolytic anaemia. Br J Haematol. 2017 Feb;176(3):395-411. doi: 10.1111/bjh.14478. Epub 2016 Dec 22. No abstract available.

    PMID: 28005293BACKGROUND

  • Barcellini W, Fattizzo B. How I treat warm autoimmune hemolytic anemia. Blood. 2021 Mar 11;137(10):1283-1294. doi: 10.1182/blood.2019003808.

    PMID: 33512406BACKGROUND

  • Birgens H, Frederiksen H, Hasselbalch HC, Rasmussen IH, Nielsen OJ, Kjeldsen L, Larsen H, Mourits-Andersen T, Plesner T, Ronnov-Jessen D, Vestergaard H, Klausen TW, Schollkopf C. A phase III randomized trial comparing glucocorticoid monotherapy versus glucocorticoid and rituximab in patients with autoimmune haemolytic anaemia. Br J Haematol. 2013 Nov;163(3):393-9. doi: 10.1111/bjh.12541. Epub 2013 Aug 24.

    PMID: 23981017BACKGROUND

  • Barcellini W, Fattizzo B, Zaninoni A, Radice T, Nichele I, Di Bona E, Lunghi M, Tassinari C, Alfinito F, Ferrari A, Leporace AP, Niscola P, Carpenedo M, Boschetti C, Revelli N, Villa MA, Consonni D, Scaramucci L, De Fabritiis P, Tagariello G, Gaidano G, Rodeghiero F, Cortelezzi A, Zanella A. Clinical heterogeneity and predictors of outcome in primary autoimmune hemolytic anemia: a GIMEMA study of 308 patients. Blood. 2014 Nov 6;124(19):2930-6. doi: 10.1182/blood-2014-06-583021. Epub 2014 Sep 16.

    PMID: 25232059BACKGROUND

  • Bride KL, Vincent T, Smith-Whitley K, Lambert MP, Bleesing JJ, Seif AE, Manno CS, Casper J, Grupp SA, Teachey DT. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016 Jan 7;127(1):17-28. doi: 10.1182/blood-2015-07-657981. Epub 2015 Oct 26.

    PMID: 26504182BACKGROUND

  • Li H, Ji J, Du Y, Huang Y, Gu H, Chen M, Wu R, Han B. Sirolimus is effective for primary relapsed/refractory autoimmune cytopenia: a multicenter study. Exp Hematol. 2020 Sep;89:87-95. doi: 10.1016/j.exphem.2020.08.001. Epub 2020 Aug 6.

    PMID: 32771553BACKGROUND

MeSH Terms

Conditions

Evans SyndromeRecurrence

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Disease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
associate professor

Study Record Dates

First Submitted

June 21, 2023

First Posted

June 29, 2023

Study Start

June 24, 2023

Primary Completion

December 1, 2025

Study Completion

December 1, 2025

Last Updated

August 8, 2025

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)