Hemophilia Inhibitor Previously Untreated Patient Study

Completed

• Conditions: Hemophilia A
• Interventions: Drug: FVIII concentrate

• Registration Number: NCT01652027
• Lead Sponsor: The University of Texas Health Science Center, Houston

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-06-03
• Study Start: 2011-07
• Study First Submitted QC: 2012-07-26
• Study First Posted: 2012-07-27
• Primary Completion: 2020-03
• Study Completion: 2020-03
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-09
• Last Update Posted: 2020-11-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Previously Untreated Patients with Hemophilia A	FVIII concentrate	-

Primary Outcome Measures

Name	Time	Method
Total number of FOXP3-positive T regulatory cells in the circulation	50 exposure days to FVIII or 3 years, whichever comes first	FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.

Secondary Outcome Measures

Name	Time	Method
FVIII-specific T-cells	50 exposure days to FVIII or 3 years, whichever comes first	FVIII-specific T-cells will be compared before and after exposure to FVIII

Trial Locations

Locations (17)

Emory University; 🇺🇸 Atlanta, Georgia, 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

University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital; 🇺🇸 Cincinnati, Ohio, United States

University of Oregon; 🇺🇸 Portland, Oregon, United States

Hemophilia Center of Western Pennsylvania; 🇺🇸 Pittsburgh, Pennsylvania, United States

North Texas Comprehensive Hemophilia Center; 🇺🇸 Dallas, Texas, United States

Scroll for more (7 remaining)