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Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China (HA-LD-ITI)

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ClinicalTrials.gov Identifier: NCT03598725
Recruitment Status : Recruiting
First Posted : July 25, 2018
Last Update Posted : July 25, 2018
Sponsor:
Information provided by (Responsible Party):
Runhui WU, Beijing Children's Hospital

Brief Summary:
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%~5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.

Condition or disease Intervention/treatment Phase
Hemophilia A With Inhibitor Drug: Coagulation Factor VIII Drug: Prednisone Drug: Rituximab Phase 4

Detailed Description:
Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ(25-50IU/kg)alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 55 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Beijing Children's Hospital, Capital Medical University
Actual Study Start Date : January 1, 2016
Estimated Primary Completion Date : December 1, 2019
Estimated Study Completion Date : December 1, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
Drug: Coagulation Factor VIII
Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day

Drug: Prednisone
2mg/kg every day for 4 weeks then typering in 3 months

Drug: Rituximab
375mg/Square meter for consecutive 4 months




Primary Outcome Measures :
  1. Success rate [ Time Frame: 2 years ]
    Success rate


Secondary Outcome Measures :
  1. Annualized Bleeding Rate [ Time Frame: 2 year ]
    How many times for all types of bleeding

  2. Annualized Joint Bleeding Rate [ Time Frame: 2 year ]
    How many times for joint bleeding

  3. Success time [ Time Frame: 2 years ]
    How long to success



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Ages Eligible for Study:   1 Year to 14 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males
  • from 1 to 14 years old
  • severe or moderate hemophilia A;
  • inhibitors positive before ITI started.

Exclusion Criteria:

  • Females
  • <1 or >14 years old
  • hemophilia B or mild haemophilia A;
  • inhibitor negative before ITI started.

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): NCT03598725


Contacts
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Contact: Xin Ni, master 010-59616643 nixin@bch.com.cn

Locations
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China, Beijing
Beijing Children's Hospital Recruiting
Beijing, Beijing, China, 100045
Contact: Runhui Wu, master    0086-010-59617621    runhuiwu@hotmail.com   
Sponsors and Collaborators
Beijing Children's Hospital

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Responsible Party: Runhui WU, Capital Medical Univercity, Beijing Children's Hospital
ClinicalTrials.gov Identifier: NCT03598725     History of Changes
Other Study ID Numbers: BCH-ITI-20180123
First Posted: July 25, 2018    Key Record Dates
Last Update Posted: July 25, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Prednisone
Rituximab
Factor VIII
Antineoplastic Agents, Immunological
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Coagulants