Anti-CD38 Antibody Treating Evans Syndrome — 2023-D-ES  

Treatment Clinical Trial

Official title: A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-CD38 Antibody in the Treatment of Evans Syndrome

Status Recruiting

Clinical Trial Summary

A single-center, open-label, off-label use investigator-initiated clinical study with safety run-in to explore the clinical activity and safety of Anti-CD38 Antibody in adult ES patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including immunosuppressive agents, Anti-CD20 Antibody and/or TPO-RA, or those in whom no other second-line treatment options are suitable.

Clinical Trial Description

Evans' syndrome (ES) is defined as the concomitant or sequential association of warm auto-immune haemolytic anaemia (AIHA) with immune thrombocytopenia (ITP), and less frequently autoimmune neutropenia. ES is a rare situation that represents up to 7% of AIHA and around 2% of ITP. Due to the rarity of the disease, the treatment of ES is mostly extrapolated from what is recommended for isolated auto-immune cytopenia (AIC) and mostly relies on corticosteroids, rituximab, splenectomy, and supportive therapies.Despite continuous progress in the management of AIC and a gradual increase in ES survival, the mortality due to ES remains higher than the ones of isolated AIC, supporting the need for an improvement in ES management.

A branch of pathogenesis for ES has been revealed that plasma cells secrete pathogenic antibodies directed against platelet and red blood cell antigens. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. In those refractory cases, persistent autoreactive long-lived plasma cells in the bone marrow could explain treatment failure.

Anti-CD38 antibody, such as Daratumumab, has been developed to target tumoral plasma cells in multiple myeloma, was recently found to be effective in antibody-mediated diseases, such as autoimmune cytopenia following hematopoietic stem cell transplantation, systemic lupus and also ES.

This study will evaluate the safety and biologic activity of Anti-CD38 antibody in r/r primary ES who fail to respond to at least one previous second-line therapy or those who cannot chose suitable second-line therapy. The study will enroll approximately 10 participants. This trial will be conducted in China. All participants will be followed for at least 16 weeks after the 8 weeks of treatment. ;

Related Conditions & MeSH terms

• Anemia, Hemolytic, Autoimmune
• Evan Syndrome
• Syndrome
• Thrombocytopenia
• Treatment

• NCT number: NCT06014775
• Study type: Interventional
• Source: Institute of Hematology & Blood Diseases Hospital, China
• Contact: Ting Sun, M.D
• Phone: +86 022-23909009
• Email: sunting@ihcams.ac.cn
• Status: Recruiting
• Phase: Phase 2
• Start date: December 2023
• Completion date: August 2025