An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A (AHA2023)

A Multicenter, Single-arm Exploratory Study of CD38 (Daratumumab) Monoclonal Antibody (Daratumumab) Combined Corticosteroid in the Treatment of Acquired Hemophilia A (AHA)

To evaluate the time of response, sustained remission rate and relapse rate of CD38 monoclonal antibody (Daratumumab) combined corticosteroid in the treatment of AHA. To evaluate the safety of CD38 monoclonal antibody in the treatment of AHA.

Study Overview

• Status: Recruiting
• Conditions: Acquired Hemophilia
• Intervention / Treatment: Drug: Daratumumab and corticosteroid treatment

Detailed Description

This is a prospective ,single-arm, multi-center controlled pilot trial of CD38 (Daratumumab) monoclonal antibody combined corticosteroid in the treatment of AHA patients.

Patients will receive Daratumumab and corticosteroid treatment in this trial.

• Study Type: Interventional
• Enrollment (Estimated): 25
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Wei Liu; Phone Number: +8613820261971; Email: liuwei1@ihcams.ac.cn
• Study Contact Backup: Name: Lei Zhang; Email: zhanglei1@ihcams.ac.cn

Study Locations

• China
  • Tianjin, China
    • Recruiting
    • Chinese Academy of Medical Science and Blood Disease Hospital
    • Contact: Shuo Chen; Email: chenshuo@ihcams.ac.cn
    • Contact: Lijun Liu; Email: gcp@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients must meet all enrollment criteria before they can be enrolled:

Diagnosed as AHA; Women are postmenopausal women or women of childbearing age with strict contraception; Patients with good compliance

Exclusion Criteria:

• Patients with any of the following items cannot be enrolled in this study:

Congenital hemophilia with inhibitor; Pregnant and lactating women; Who are positive for hepatitis B surface antigen, hepatitis C antibody, HIV antibody (Ⅰ + Ⅱ) and syphilis antibody; Patients with poor compliance; Who cannot use contraception during the trial; Researchers believe that it is not appropriate for patients to participate in any other condition of this trial; Four weeks before entering the group, the patients received immunosuppressive therapy and the inhibitor showed a progressive decreasing trend (the change was less than 50%).

Acquired von Willebrand disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment arm; Daratumumab and corticosteroid treatment	Drug: Daratumumab and corticosteroid treatment; Daratumumab and corticosteroid treatment; Other Names: Intervention arm

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total response rate to treatment (OR) Total response rate to treatment	The proportion of patients achieving OR including CR and PR	During 8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
time to CR	The time for patients to reach complete remission	During 8 weeks
The proportion of patients remaining in CR during 24 weeks	The proportion of patients who reached CR and remain in CR during 24 weeks of follow -up time	During 24 weeks
The proportion of patients relapse after reaching CR during 24 weeks	The proportion of patients who reached CR and relapse during 24 weeks of follow -up time	During 24 weeks
Safety outcome	Adverse events will be collected	From start of the treatment until the end of 24 weeks in the follow-up period
the proportion of patients with complete remission (CR)	The proportion of patients achieving CR , which is defined as titer FVIII inhibitor lower than 0.6 Bethesda unit, factor VIII level≥ 50%	During 8 weeks
the proportion of patients with partial remission rate (PR)	The proportion of patients achieving PR, which is defined as titer FVIII inhibitor higher than 0.6 Bethesda unit and factor VIII level≥ 50%, and no bleedings will be evaluated.	During 8 weeks

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Investigators: Principal Investigator: Lei Zhang, Chinese Academy of Medical Science and Blood Disease Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 22, 2023
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): October 31, 2027

Study Registration Dates

• First Submitted: April 18, 2023
• First Submitted That Met QC Criteria: May 5, 2023
• First Posted (Actual): May 9, 2023

Study Record Updates

• Last Update Posted (Actual): August 5, 2025
• Last Update Submitted That Met QC Criteria: July 31, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Antineoplastic Agents; Daratumumab
• Other Study ID Numbers: IIT2023013

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Data can be requested from the corresponding authors from 12 months to 36 months after study completion
• IPD Sharing Time Frame: from 12 months to 36 months after study completion
• IPD Sharing Access Criteria: From corresponding author
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes