Anti-CD38 Antibody Treating Evans Syndrome

NCT06014775 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2023036
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Evan Syndrome; Treatment

Outcome Measures

Primary Outcomes (2)

• Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks

  Overall response rate defined as improvement in any cytopenias by at least one grade, without worsening any other cytopenias or stable disease at least once within 8 weeks after the first dose.

  8 weeks

• Safety of Anti-CD38 antibody treatment

  Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment

  24 weeks

Secondary Outcomes (15)

• Evaluation of stable sustained response after Anti-CD38 antibody treatment at week 8

  8 weeks

• Number of Participants With ES Response to Anti-CD38 antibody treatment

  24 weeks

• Measurements of platelet count at each visit time point

  24 weeks

• Measurements of Hb value at each visit time point

  24 weeks

• Measurements of hemolytic marker reticulocyte count at each visit time point

  24 weeks

Study Arms (1)

Intervention（Anti-CD38 antibody）

EXPERIMENTAL

10 enrolled subjects : once a week x 8 doses

Drug: Anti-CD38 antibody Injection

Interventions

Anti-CD38 antibody Injection DRUG

intravenous Anti-CD38 antibody administration This study adopts a prospective, single arm, open design method. Ten subjects were enrolled in the study and were treated with Anti-CD38 antibody (16mg/kg/w) for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg Anti-CD38 antibody once a week for 8 weeks to observe the safety and efficacy during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Anti-CD38 antibody after treatment.

Intervention（Anti-CD38 antibody）

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female aged ≥18 years.
• Prior to enrollment, a clinical diagnosis of primary Evans syndrome was made.
• Platelet count \< 30×10\^9/L or Hb \< 100g/L or symptomatic anemia within 48 hours before the first administration of study drug;
• Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy or those who cannot chose other second-line therapy;
• If receiving emergency care for ES, treatment should be stopped \>2 weeks before first dose.
• DAT positive (IgG+, with or without C3+).
• The patient need to be in the state of active hemolysis.
• With normal hepatic and renal functions.
• ECOG performance status ≤2.
• Cardiac function: New York Heart Association functional class ≤2.
• For patients receiving maintenance treatment, corticosteroids must have a stable dose at least 2 weeks before the first administration, TPO receptor agonists and azathioprine, danazol, cyclosporin A, tacrolimus, sirolimus, etc. must be stopped at least 4 weeks before the first administration; The end of anti-CD20 antibody treatment was\>6 months.The end of alkylating agent treatment was\>2 months.
• Understand the study procedures and voluntarily sign the informed consent form in person.

You may not qualify if:

• Secondary Evans syndrome. Received any treatment of anti-CD38 antibody drug
• Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
• HIV positive;
• Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
• Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
• At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
• Patients with thrombotic diseases such as pulmonary embolism, thrombosis and atherosclerosis;
• Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
• Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
• Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
• Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
• Patients with septicemia or other irregular severe bleeding;
• Patients taking antiplatelet drugs at the same time;
• Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Evans Syndrome

Study Officials

• Lei Zhang, M.D

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ting Sun, M.D

CONTACT

+86 022-23909009; sunting@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2023
• First Posted: August 28, 2023
• Study Start: December 1, 2023
• Primary Completion: August 1, 2025
• Study Completion: August 1, 2025
• Last Updated: February 21, 2025

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)