Hemophilia Inhibitor Previously Untreated Patient Study

Hemophilia A Clinical Trial

— HIPS  

Official title:

Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01652027
• Other study ID #: HSC-MS-11-0202
• Secondary ID: HSC-MS-11-0202
• Status: Completed
• Start date: July 2011
• Est. completion date: March 2020

Study information

• Verified date: November 2020
• Source: The University of Texas Health Science Center, Houston
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Hemophilia A is a congenital bleeding disorder caused by deficiency of factor VIII (FVIII) and is treated by replacement therapy with FVIII concentrate. Approximately 30% of people with severe hemophilia A develop neutralizing antibodies, called FVIII inhibitors, which interfere with the function of FVIII concentrates. The reason that some, but not all, people with severe hemophilia A develop inhibitors is incompletely understood. Understanding individual and environmental risk factors is important to be able to prevent and possibly treat inhibitors. This study will look at individual and treatment characteristics in babies with severe hemophilia A who have not yet received treatment with FVIII (called Previously Untreated Patients, or PUPS). Subjects in the study will be asked to provide diaries of treatments, medications, and illnesses. Treatment will be directed by the subjects' physician, but all subjects will receive Advate, a third-generation recombinant FVIII product. Subjects will have blood drawn for laboratory tests, which include studies of the immune system and genetic studies of the FVIII mutation, before and 7-9 days after the first treatment with FVIII, and 5 days (+/-2 days) after the 5th, 10th, 20th, 30th, 40th, and 50th days of treatment with FVIII (exposure days). The duration of the study will be first 50 treatments or 3 years, whichever comes first.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: March 2020
• Est. primary completion date: March 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Severe hemophilia A with FVIII activity < 1% normal

• Weight > 3.5 kg at the time of baseline study evaluation

• Informed consent, approved by appropriate Institutional Review Board/Independent Ethics Committee, has been administered, signed, and dated

Exclusion Criteria:

• Prior exposure to clotting factor concentrates or blood products

• Other chronic disease

• Currently participating in another investigational drug study.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• FVIII concentrate
usual treatment as directed by treating physician

Locations

Country	Name	City	State
Austria	Medical University of Vienna	Vienna
Italy	Angelo Bianchi Bonomi Hemophilia & Thrombosis Center	Milan
Netherlands	Emma Children's Hospital AMC	Amsterdam
Sweden	Malmo Centre for Thrombosis and Haemostasis	Malmo
United States	Emory University	Atlanta	Georgia
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	North Texas Comprehensive Hemophilia Center	Dallas	Texas
United States	Baylor College of Medicine	Houston	Texas
United States	University of Texas Health Science Center-Houston	Houston	Texas
United States	Indiana Hemophilia and Thrombosis Center	Indianapolis	Indiana
United States	University of Kentucky	Lexington	Kentucky
United States	Tulane University	New Orleans	Louisiana
United States	Cornell University	New York	New York
United States	Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania
United States	University of Oregon	Portland	Oregon
United States	Intermountain Hemophilia and Thrombosis Center	Salt Lake City	Utah

Sponsors (3)

Lead Sponsor	Collaborator
The University of Texas Health Science Center, Houston	Baxter Healthcare Corporation, Rho, Inc.

Countries where clinical trial is conducted

United States,  Austria,  Italy,  Netherlands,  Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total number of FOXP3-positive T regulatory cells in the circulation	FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.	50 exposure days to FVIII or 3 years, whichever comes first
Secondary	FVIII-specific T-cells	FVIII-specific T-cells will be compared before and after exposure to FVIII	50 exposure days to FVIII or 3 years, whichever comes first