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

Hemophilia A With Inhibitor Clinical Trial

— HA-LD-ITI  

Official title:

Beijing Children's Hospital, Capital Medical University

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03598725
• Other study ID #: BCH-ITI-20180123
• Status: Recruiting
• Phase: Phase 4
• Start date: January 1, 2016
• Est. completion date: December 1, 2020

Study information

• Verified date: July 2018
• Source: Beijing Children's Hospital
• Contact: Xin Ni, master
• Phone: 010-59616643
• Email: nixin[@]bch.com.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 55
• Est. completion date: December 1, 2020
• Est. primary completion date: December 1, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 1 Year to 14 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A With Inhibitor

Intervention

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

Locations

Country	Name	City	State
China	Beijing Children's Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Beijing Children's Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Success rate	Success rate	2 years
Secondary	Annualized Bleeding Rate	How many times for all types of bleeding	2 year
Secondary	Annualized Joint Bleeding Rate	How many times for joint bleeding	2 year
Secondary	Success time	How long to success	2 years