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

NCT ID: NCT03598725

Last Updated: 2018-07-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 55 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-01
• Study Completion Date: 2020-12-01

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.

Conditions

Hemophilia A With Inhibitor

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Coagulation Factor VIII

Intervention Type: DRUG

Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day

Prednisone

Intervention Type: DRUG

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

Rituximab

Intervention Type: DRUG

375mg/Square meter for consecutive 4 months

Interventions

Coagulation Factor VIII

Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day

Intervention Type: DRUG

Prednisone

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

Intervention Type: DRUG

Rituximab

375mg/Square meter for consecutive 4 months

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 14 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Beijing Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Runhui WU

Capital Medical Univercity

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Beijing Children's Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Xin Ni, master

Role: CONTACT

nixin@bch.com.cn

010-59616643

Facility Contacts

Runhui Wu, master

Role: primary

runhuiwu@hotmail.com

0086-010-59617621

References

Li Z, Tang Y, Chen Z, Liu G, Yao W, Li G, Cheng X, Peng Y, Cai S, Cui C, Ai D, Zhang J, Poon MC, Zhang W, Wu R. Outcomes of immune tolerance induction with rituximab to eradicate high-titer inhibitor of hemophilia A: depicted by exponential decay model and the gene expression profile of different outcomes by RNA-sequencing. J Thromb Haemost. 2025 Aug;23(8):2436-2448. doi: 10.1016/j.jtha.2025.04.015. Epub 2025 Apr 24.

Reference Type: DERIVED

PMID: 40286913 (View on PubMed)

Other Identifiers

BCH-ITI-20180123

Identifier Type: -

Identifier Source: org_study_id