NCT03598725

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

Beijing Children's Hospital, Capital Medical University

• Status: Unknown
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 55 (estimated)
• Sponsor: Beijing Children's Hospital · Academic / Other
• Sex: Male
• Age: 1 Year – 14 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A With Inhibitor

Interventions

Type	Name	Description
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

Timeline

• Start date: 2016-01-01
• Primary completion: 2019-12-01
• Completion: 2020-12-01
• First posted: 2018-07-26
• Last updated: 2018-07-26

Locations

1 site across 1 country: China