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

Beijing Children's Hospital, Capital Medical University

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.

Study Overview

• Status: Unknown
• Conditions: Hemophilia A With Inhibitor
• Intervention / Treatment: Drug: Coagulation Factor VIII; Drug: Prednisone; Drug: Rituximab

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 Type: Interventional
• Enrollment (Anticipated): 55
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100045
      • Recruiting
      • Beijing Children's Hospital
      • Contact: Runhui Wu, master; Phone Number: 0086-010-59617621; Email: runhuiwu@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 14 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

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.

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Success rate	Success rate	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Bleeding Rate	How many times for all types of bleeding	2 year
Annualized Joint Bleeding Rate	How many times for joint bleeding	2 year
Success time	How long to success	2 years

Collaborators and Investigators

• Sponsor: Beijing Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2016
• Primary Completion (Anticipated): December 1, 2019
• Study Completion (Anticipated): December 1, 2020

Study Registration Dates

• First Submitted: January 23, 2018
• First Submitted That Met QC Criteria: July 23, 2018
• First Posted (Actual): July 26, 2018

Study Record Updates

• Last Update Posted (Actual): July 26, 2018
• Last Update Submitted That Met QC Criteria: July 23, 2018
• Last Verified: July 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antirheumatic Agents; Antineoplastic Agents; Immunologic Factors; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Antineoplastic Agents, Immunological; Coagulants; Rituximab; Prednisone; Factor VIII
• Other Study ID Numbers: BCH-ITI-20180123

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No