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

Sponsor
Beijing Children's Hospital
Study ID
NCT03598725
Phase
PHASE4
Status
Unknown

Conditions

  • Hemophilia A With Inhibitor

Eligibility Criteria

Sex
MALE
Age
1 Year - 14 Years
Healthy Volunteers
Not accepted

Interventions

  • Coagulation Factor VIII — DRUG
    Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day
  • Prednisone — DRUG
    2mg/kg every day for 4 weeks then typering in 3 months
  • Rituximab — DRUG
    375mg/Square meter for consecutive 4 months

Study Details

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.

Key Dates

Start date
Jan 1, 2016
Status verified
Jul 2018
Primary completion
Dec 1, 2019
Completion
Dec 1, 2020

Study Design

Enrollment
55 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • 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.

Primary Outcome Measure

Success rate [ Time Frame: 2 years ]

Central Contacts

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