Rituximab to Treat Severe Hemophilia A

Summary

Hemophilia A is a serious blood clotting disorder caused by a lack of factor VIII, a specialized protein needed for normal blood clotting to occur. Individuals with this disease may experience spontaneous bleeding, pain and swelling in their joints due to excess bleeding, and bruising. A common treatment for severe hemophilia A is to intravenously replace the deficient blood clotting factor; however, some individuals may develop antibodies to this replacement factor. This study will evaluate the effectiveness of rituximab at reducing the antibodies that develop in response to the replacement factor in individuals with severe hemophilia A.

Conditions

• Hemophilia A

Interventions

DRUG

Rituximab

Rituximab by slow intravenous infusion; for participants greater than or equal to 10 kg, 375 mg per m\^2 BSA weekly for 4 weeks; for participants less than 10 kg, 12.5 mg/kg weekly for 4 weeks

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Genentech, Inc.

  collaborator INDUSTRY

• Carelon Research

  lead OTHER

Principal Investigators

• Susan F. Assmann, PhD · NERI

• Cindy Leissinger, MD · Tulane University Health Sciences Center

• Joan Gill, MD · Versiti

• Keith McCrae, MD · University Hospital of Cleveland

• Ellis Neufeld, MD · Boston Children's Hospital

• Cassandra Josephson, MD · Children's Healthcare of Atlanta

• Nigel Key, MD · University of North Carolina

• Charles Sexauer, MD · University of Oklahoma

• Janna Journeycake, MD · University of Texas Southwestern Medical Center

• Leslie Raffini, MD · Children's Hospital of Philadelphia

• Margaret Ragni, MD · Hemophilia Center of Western Pennsylvania

• Leonard Valentino, MD · Rush University Medical Center

• Diane Nugent, MD · Children's Hospital of Orange County

• Marcella Torres, MD · Cook Children's Medical Center

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Months

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2006-06-30

Primary Completion

2010-11-30

Completion

2012-01-31

Countries

• United States

Study Locations