Hemophilia Inhibitor Previously Untreated Patient Study

Completed

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT01652027

Run by

The University of Texas Health Science Center, Houston

People needed

25

Starts

2011-07-01

Expected to finish

2020-03-01

Last updated by the study team

2020-11-10

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Prior exposure to clotting factor concentrates or blood products
• Other chronic disease
• Currently participating in another investigational drug study.

Where it is running

• 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
• Baylor College of Medicine — Houston, Texas, United States
• University of Texas Health Science Center-Houston — Houston, Texas, United States
• Intermountain Hemophilia and Thrombosis Center — Salt Lake City, Utah, United States
• Medical University of Vienna — Vienna, Austria
• Angelo Bianchi Bonomi Hemophilia & Thrombosis Center — Milan, Italy
• Emma Children's Hospital AMC — Amsterdam, Netherlands
• Malmo Centre for Thrombosis and Haemostasis — Malmö, Sweden

Full record on ClinicalTrials.gov

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