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

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ClinicalTrials.gov Identifier: NCT03384277
Recruitment Status : Recruiting
First Posted : December 27, 2017
Last Update Posted : October 9, 2019
Sponsor:
Collaborators:
Qilu Hospital of Shandong University
Tianjin First Central Hospital
The Second Affiliated Hospital of Kunming Medical University
Henan Cancer Hospital
Information provided by (Responsible Party):
Zhang Lei, Institute of Hematology & Blood Diseases Hospital

Brief Summary:

Purpose:

To evaluate the efficacy when administering steroid combined with small 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


Condition or disease Intervention/treatment Phase
Acquired Hemophilia A Drug: Steroid Drug: Rituximab Drug: Cyclophosphamide Phase 3

Detailed Description:

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Prospective, Randomized, Multicenter Clinical Trial of Acquired Haemophilia A With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab
Actual Study Start Date : October 26, 2017
Estimated Primary Completion Date : October 30, 2020
Estimated Study Completion Date : December 1, 2020


Arm Intervention/treatment
Experimental: Steroid+Rituximab
Methylprednisolone 0.8 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks(then tapering gradually, 8 weeks in total)+Rituximab 375mg/m2 for one dose.
Drug: Steroid
Methylprednisolone 0.8 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks(then tapering gradually, 8 weeks in total)
Other Name: Corticosteroid

Drug: Rituximab
375mg/m2 for one dose

Active Comparator: Steroid +Cyclophosphamide
Methylprednisolone 0.8 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks ( then tapering gradually, 8 weeks in total)+ Cyclophosphamide 2 mg/kg/day until inhibitor negative (no longer than five weeks)
Drug: Steroid
Methylprednisolone 0.8 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks(then tapering gradually, 8 weeks in total)
Other Name: Corticosteroid

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




Primary Outcome Measures :
  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 Outcome Measures :
  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 Outcome Measures:
  1. Major bleeding and infection of two regimens [ Time Frame: During 18 months ]
    The safety outcomes will be the occurrence of major bleeding and infection related immunosuppressive treatment adverse events.



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03384277


Contacts
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Contact: Wei Liu, MD +8613820261971 liuwei1@ihcams.ac.cn
Contact: Lei Zhang, MD +8613502118379 zhanglei1@ihcams.ac.cn

Locations
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China, Tianjin
Ethics Committee of Blood disease hospital, Chinese Academy of Medical Sciences Recruiting
Tianjin, Tianjin, China, 300020
Contact: Lijun Liu, Master    +8613920927593    liulijun@ihcams.ac.cn   
Sponsors and Collaborators
Zhang Lei
Qilu Hospital of Shandong University
Tianjin First Central Hospital
The Second Affiliated Hospital of Kunming Medical University
Henan Cancer Hospital
Investigators
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Principal Investigator: Lei Zhang, MD Blood disease hospital, Chinese academy of medical sciences

Publications:
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Responsible Party: Zhang Lei, vice Director of Thrombosis and Hemostasis Center, Institute of Hematology & Blood Diseases Hospital
ClinicalTrials.gov Identifier: NCT03384277     History of Changes
Other Study ID Numbers: IHBDH-IIT2017006
First Posted: December 27, 2017    Key Record Dates
Last Update Posted: October 9, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual participant data for all primary and secondary outcome will be made available
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Analytic Code
Time Frame: Data will be available within 6 month of study completion
Access Criteria: Data access requests will be reviewed by an external independent Review Panel. Requests will be required to sign a Data Access Agreement
URL: http://www.medresman.org

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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.: No
Keywords provided by Zhang Lei, Institute of Hematology & Blood Diseases Hospital:
Acquired hemophilia A
Cyclophosphamide
Rituximab
Steroid
Additional relevant MeSH terms:
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Neuroprotective Agents
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Methylprednisolone
Cyclophosphamide
Rituximab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antineoplastic Agents, Immunological
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Protective Agents