A Study of Elranatamab for Inhibitor Eradication in Patients With Hemophilia A

NCT07788469 | Not Yet Recruiting

• 1 other identifier: IIT2026076
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective, single-arm, open-label, exploratory clinical study designed to evaluate the safety and efficacy of Elranatamab for inhibitor eradication in adults with moderate-to-severe hemophilia A with inhibitors. A total of 15-20 patients aged ≥18 years will be enrolled. Elranatamab will be administered with a stepwise dose-escalation regimen of 12 mg on Day 1 and 32 mg on Day 4 during Week 1, followed by the target dose of 38 mg once weekly during Weeks 2-3. Patients will then enter a 4-week follow-up period. If the inhibitor titer decreases by \<20% from baseline at Week 3, two additional weekly doses of 38 mg may be administered before follow-up. Treatment response will be assessed according to changes in inhibitor titers following treatment.

Conditions

Hemophilia A With Inhibitors

Keywords

Hemophilia A; Inhibitors; Elranatamab

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events (AES)

  AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.

  1 year

Secondary Outcomes (1)

• The proportion of patients who achieve successful inhibitor eradication

  1 year

Study Arms (1)

hemophilia A with inhibitors receiving Elranatamab at a stepwise dose-escalation regimen

EXPERIMENTAL

Elranatamab will be administered with a stepwise dose-escalation regimen

Drug: Elranatamab

Interventions

Elranatamab DRUG

Patients receive Elranatamab at 12 mg on Day 1 and 32 mg on Day 4 during Week 1, followed by the target dose of 38 mg once weekly during Weeks 2-3. Patients will then enter a 4-week follow-up period. If the inhibitor titer decreases by \<20% from baseline at Week 3, two additional weekly doses of 38 mg may be administered before follow-up. If the inhibitor titer decreases by \>20% from baseline at Week 3, no further treatment will be given, and patients will proceed directly to follow-up.

hemophilia A with inhibitors receiving Elranatamab at a stepwise dose-escalation regimen

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 patients with moderate-to-severe hemophilia A (factor VIII activity \<2%).
• Aged 18 to 65 years, inclusive.
• Positive factor VIII inhibitor detected on at least two consecutive occasions (inhibitor titer \>0.6 BU/mL).
• Baseline factor VIII inhibitor titer \>10 BU/mL at the time of enrollment.

You may not qualify if:

• Known hypersensitivity to Elranatamab or any of its excipients.
• Presence of other autoimmune diseases or a need for immunosuppressive therapy for reasons unrelated to this study.
• History of malignancy within 5 years prior to screening, with the exception of adequately treated carcinoma in situ of the cervix or non-metastatic cutaneous squamous cell carcinoma or basal cell carcinoma.
• History of severe recurrent or chronic infections, or acute infection requiring systemic treatment with antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungal agents within 4 weeks before the first dose or during the screening period, or superficial skin infection requiring systemic therapy within 1 week before the first dose.
• Clinically significant laboratory abnormalities at screening, including:
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3 × the upper limit of normal (ULN).
• Total bilirubin \>1.5 × ULN (subjects with documented Gilbert syndrome are not excluded based on this criterion);
• Absolute neutrophil count \<1,500/mm³;
• Hemoglobin \<9 g/dL or immunoglobulin G (IgG) \<500 mg/dL;
• Absolute lymphocyte count \<500/mm³;
• Creatinine clearance (CrCl) \<30 mL/min.
• Positive test for HIV antibody or syphilis antibody.
• Positive hepatitis B surface antigen (HBsAg); or positive hepatitis B core antibody (anti-HBc) with detectable HBV DNA by polymerase chain reaction (PCR). Subjects positive for hepatitis C virus (HCV) antibody are excluded.
• Women who are pregnant or breastfeeding, or who plan to become pregnant or breastfeed during the study; men whose partners plan to become pregnant during the study.
• Subjects with psychiatric disorders that impair their ability to provide informed consent or comply with study procedures and follow-up.

Sponsors & Collaborators

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

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, China

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lei Zhang

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wei Liu

CONTACT

+8613820261971; liuwei1@ihcams.ac.cn

Lei Zhang

CONTACT

zhanglei1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2026
• First Posted: August 26, 2026
• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2028
• Last Updated: August 26, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: from 12 months to 36 months after study completion
• Access Criteria: From corresponding author

Locations

CN (1)