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Clinical Trials/NCT03598725
NCT03598725UnknownPhase 4

Beijing Children's Hospital, Capital Medical University

Beijing Children's Hospital1 site in 1 country55 target enrollmentStarted: January 1, 2016Last updated:
Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 4
Sponsor
Enrollment
55
Locations
1
Primary Endpoint
Success rate

Study Overview

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.

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
1 Year to 14 Years (Child)
Sex
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

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

Exclusion Criteria

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

Arms & Interventions

ITI strategy

Experimental

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.

Intervention: Coagulation Factor VIII (Drug)

ITI strategy

Experimental

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.

Intervention: Prednisone (Drug)

ITI strategy

Experimental

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.

Intervention: Rituximab (Drug)

Outcomes

Primary Outcomes

Success rate

Time Frame: 2 years

Success rate

Secondary Outcomes

  • Annualized Bleeding Rate(2 year)
  • Success time(2 years)
  • Annualized Joint Bleeding Rate(2 year)

Investigators

Sponsor
Beijing Children's Hospital
Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Runhui WU

Capital Medical Univercity

Beijing Children's Hospital

Study Sites (1)

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