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Completed

NCT Number: NCT03384277

Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab

Purpose:

To evaluate the efficacy when administering steroid combined with single dose rituximab to eliminate the antibody in acquired hemophilia A patients compared to treatment using steroid with cyclophosphamide.

The study will test the hypothesis that steroid combined with small dose rituximab is as effective as steroid combined with cyclophosphamide for FVIII inhibitor eradication in Chinese patients with acquired hemophilia A.

Study design Allocation: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment

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Key information

Conditions

Acquired Hemophilia A Factor 8 deficiency, acquired

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Ethics Committee of Blood disease hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

About this study

This is a prospective randomized multi-center controlled pilot trial comparing the regimen of steroid with rituximab and steroid with cyclophosphamide to eradicate anti-factor VIII antibodies in Chinese patients with acquired hemophilia A.

Patients will be randomized to two regimens: methylprednisolone 0.8mg/kg/day (or equivalent corticosteroid doses) for 3 weeks (then tapering gradually,8 weeks in total) with rituximab (375mg/m2 for one dose) or methylprednisolone 0.8mg/kg/day (or equivalent corticosteroid doses) for 3 weeks (then tapering gradually,8 weeks in total) with cyclophosphamide 2mg/kg/day until inhibitor negative(no longer than five weeks).

Patients will be randomized to the treatment cohorts according to the biostatistical methods.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 18-80 years old
  • Men or women
  • Women post-menopausal or with ongoing contraception
  • Diagnosis of acquired hemophilia A
  • Patient must be insured
  • Patient has provided written informed consent prior to enrollment
  • Patient compliant

Exclusion criteria

  • Congenital hemophilia
  • Ongoing treatment with prednisone > 20mg/d （or equivalent corticosteroid doses） more than 1 month
  • Ongoing treatment with prednisone >0.7mg/kg（or equivalent corticosteroid doses） more than 10 days
  • Pregnant and breastfeeding women
  • Allergy to steroid
  • Immunosuppressive agents treatment within 30 days
  • Serum transaminase and bilirubin greater than 1.5 times the upper limit of normal value
  • Hepatitis B surface antigen or hepatitis C antibody or HIV antibody (I + II) or syphilis antibody positive
  • Patients with diabetes, hypertension, glaucoma, peptic ulcer, herpes zoster, pulmonary infection and so on, who should not be treated with glucocorticoids
  • Patients with poor compliance
  • Those who can not take contraceptive measures during the test period
  • Patient who is considered by the investigator not suitable for clinical study
  • Thrombocytopenia
  • Leucocytopenia

Treatment and study plan

Steroid

Drug

Methylprednisolone 0.8 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks（then tapering gradually, 8 weeks in total）

Other names: Corticosteroid

Rituximab

Drug

375mg/m2 for one dose

Cyclophosphamide

Drug

cyclophosphamide 2 mg/kg/day until inhibitor negative (no longer than five weeks)

Primary outcomes

  1. Proportion of inhibitor eradication and time to attain first remission

    Time frame: During 18 months

    The proportion of patients achieving complete remission (CR) defined as titer FVIII inhibitor lower than 0.6 Bethesda unit, factor VIII level> 50% and no bleeding events without bypass treatments for 24 hours and the time to attain first remission will be evaluated.

Secondary outcomes

  1. Relapse rate and time to relapse

    Time frame: During 18 month

    The proportion of patients who relapse and the time to relapse of each regimen will be measured.

Other outcomes

  1. Infection of two regimens

    Time frame: During 18 months

    The safety outcomes will be the occurrence of infection related to immunosuppressive treatment adverse events.

  2. Hemocytopenia of two regimens

    Time frame: During 18 month

    The safety outcomes will be the occurrence of Hemocytopenia related to immunosuppressive treatment adverse events.

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Collaborators

  • Henan Cancer Hospital
  • Qilu Hospital of Shandong University
  • The Second Affiliated Hospital of Kunming Medical University
  • Tianjin First Central Hospital

Registry information

Official study title

A Prospective, Randomized, Multicenter Clinical Trial of Acquired Haemophilia A With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab

Important dates

Study start
2017
Primary completion
2022
Study completion
2022
First posted
Dec 27, 2017
Registry last updated
Feb 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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