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Sponsors and Collaborators: |
National Heart, Lung, and Blood Institute (NHLBI) Genentech |
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Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT00331006 |
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.
Condition | Intervention | Phase |
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Hemophilia A |
Drug: Rituximab |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Rituximab for the Treatment of Inhibitors in Congenital Hemophilia A (A TMH CTN Study) |
Estimated Enrollment: | 50 |
Study Start Date: | June 2006 |
Estimated Study Completion Date: | December 2011 |
Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
Rituximab
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Drug: Rituximab
Rituximab by slow intravenous infusion; for participants greater than or equal to 10 kg, 375 mg per m^2 BSA, for participants less than 10 kg, 12.5 mg/kg weekly for 4 weeks
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Hemophilia A is a hereditary blood clotting disorder. It is caused by a deficiency or abnormality of the blood clotting protein factor VIII. Individuals with hemophilia A are unable to form blood clots to stop bleeding and are at risk for experiencing serious and life-threatening bleeding episodes. The most common treatment for this disease is intravenous replacement of factor VIII at the start of a bleeding episode. However, between 30 to 40% of individuals eventually develop inhibitors, or antibodies, to the replacement factor. In these individuals, the immune system recognizes the replacement factor as foreign and attacks it, thereby countering any potential benefits of the treatment. Some individuals with severe hemophilia A may undergo immune tolerance therapy (ITT), in which they receive replacement factor on a regular basis as a way for the body to adjust to the factor and stop inhibitor production. This treatment, however, is not always effective for everyone. Preliminary research has shown that rituximab, a medication used to treat non-Hodgkin's lymphoma, may be successful in suppressing or eliminating the inhibitors that develop. The purpose of this study is to evaluate the effectiveness of rituximab at lowering the levels of factor VIII inhibitors in individuals with severe hemophilia A.
This study will enroll individuals with severe hemophilia A. At study entry, participants will receive one intravenous dose of factor VIII. Inhibitor levels will be measured with a blood test 5 to 7 days following this procedure. If peak inhibitor level is above 5 Bethesda units (BU)/mL, 5 to 9 days later participants will begin receiving rituximab intravenously once a week for 4 weeks. Blood will be collected at each visit for laboratory testing. Two weeks following the last rituximab treatment, participants will have blood drawn for inhibitor testing; this testing will occur every 4 weeks through Week 22. If the participant's inhibitor level falls below 5 BU/mL, participants will receive a repeat dose of factor VIII, and blood will be drawn 5 to 7 days later for inhibitor testing. Follow-up visits will occur at Weeks 36, 52, and 100, and will include a physical examination, blood collection, and monitoring of bleeding events and infections. Telephone interviews will be conduced at Weeks 64, 76, and 88 to monitor bleeding events and infections.
Ages Eligible for Study: | 18 Months and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Evidence of Hepatitis B (HBV) infection, defined as one of the following:
Participants with a high responding inhibitor (at least 5 BU/mL) first detected fewer than 12 months prior to study entry, unless the participant has failed immune tolerance therapy, defined as one of the following:
Contact: Susan F. Assmann, PhD | 617-923-7747 ext 548 | sassmann@neriscience.com |
Contact: Julie Miller, MPH | 617-923-7747 ext 497 | jmiller@neriscience.com |
United States, California | |
Children's Hospital of Los Angeles | Not yet recruiting |
Los Angeles, California, United States, 90027 | |
Contact: Guy Young, MD 323-361-5507 gyoung@chla.usc.edu | |
Principal Investigator: Guy Young, MD | |
United States, Georgia | |
Children's Healthcare of Atlanta | Recruiting |
Atlanta, Georgia, United States, 30322 | |
Contact: Carolyn Bennett, MD 404-785-3240 carolyn.bennett@choa.org | |
Principal Investigator: Carolyn Bennett, MD | |
United States, Illinois | |
Rush University Medical Center | Recruiting |
Chicago, Illinois, United States, 60612 | |
Contact: Leonard Valentino, MD 312-942-8114 lvalentino@rush.edu | |
Principal Investigator: Leonard Valentino, MD | |
United States, Iowa | |
University of Iowa Hospitals and Clinics | Not yet recruiting |
Iowa City, Iowa, United States, 52242 | |
Contact: Ronald Strauss, MD 319-356-0387 ronald-strauss@uiowa.edu | |
Principal Investigator: Ronald Strauss, MD | |
United States, Louisiana | |
Tulane University Health Sciences Center | Recruiting |
New Orleans, Louisiana, United States, 70112 | |
Contact: Cindy Leissinger, MD 504-988-5433 cleissi@tulane.edu | |
Principal Investigator: Cindy Leissinger, MD | |
United States, Maryland | |
Johns Hopkins Hospital | Not yet recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: John Strouse, MD 410-955-6132 jstrous1@jhmi.edu | |
Principal Investigator: John Strouse, MD | |
United States, Massachusetts | |
Children's Hospital Boston | Recruiting |
Boston, Massachusetts, United States, 02115 | |
Contact: Ellis Neufeld, MD 617-919-2139 ellis.neufeld@childrens.harvard.edu | |
Principal Investigator: Ellis Neufeld, MD | |
United States, North Carolina | |
UNC at Chapel Hill Hospital | Recruiting |
Chapel Hill, North Carolina, United States, 27514 | |
Contact: Nigel Key, MD 919-966-3311 nigel_key@med.unc.edu | |
Principal Investigator: Nigel Key, MD | |
United States, Ohio | |
University Hospital of Cleveland | Recruiting |
Cleveland, Ohio, United States, 44106 | |
Contact: Keith McCrae, MD 216-368-6606 kxm71@po.cwru.edu | |
Principal Investigator: Keith McCrae, MD | |
United States, Oklahoma | |
University of Oklahoma Health Sciences Center | Recruiting |
Oklahoma City, Oklahoma, United States, 73104 | |
Contact: Kapil Saxena, MD 405-271-3661 kapil-saxena@ouhsc.edu | |
Principal Investigator: Kapil Saxena, MD | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Leslie Raffini, MD 267-426-5029 raffini@email.chop.edu | |
Principal Investigator: Leslie Raffini, MD | |
Hemophilia Center of Western Pennsylvania | Recruiting |
Pittsburgh, Pennsylvania, United States, 15213 | |
Contact: Margaret Ragni, MD 412-209-7288 ragni@dom.pitt.edu | |
Principal Investigator: Margaret Ragni, MD | |
United States, Texas | |
University of Texas Southwestern Medical Center | Recruiting |
Dallas, Texas, United States, 75390 | |
Contact: George Buchanan, MD 214-648-8594 george.buchanan@utsouthwestern.edu | |
Principal Investigator: George Buchanan, MD | |
University of Texas Health Science Center at Houston | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Miguel Escobar, MD 713-500-8360 miguel.escobar@uth.tmc.edu | |
University of Texas Health Science Center San Antonio | Not yet recruiting |
San Antonio, Texas, United States, 78229 | |
Contact: Melissa Frei-Jones, MD 210-704-3405 Frei_Jones_M@kids.wustl.edu | |
Principal Investigator: Melissa Frei-Jones, MD | |
United States, Wisconsin | |
Comprehensive Center for Bleeding Disorders | Recruiting |
Milwaukee, Wisconsin, United States, 53201 | |
Contact: Joan Gill, MD 414-257-2424 joan.gill@bcw.edu | |
Principal Investigator: Joan Gill, MD |
Principal Investigator: | Susan F. Assmann, PhD | NERI |
Principal Investigator: | Cindy Leissinger, MD | Tulane University Health Sciences Center |
Principal Investigator: | Joan Gill, MD | BloodCenter of Wisconsin |
Principal Investigator: | Keith McCrae, MD | University Hospital of Cleveland |
Principal Investigator: | Ellis Neufeld, MD | Children's Hospital Boston |
Principal Investigator: | Carolyn Bennett, MD | Children's Healthcare of Atlanta |
Principal Investigator: | John Strouse, MD | Johns Hopkins University |
Principal Investigator: | Ronald Strauss, MD | University of Iowa |
Principal Investigator: | Nigel Key, MD | University of North Carolina |
Principal Investigator: | Kapil Saxena, MD | University of Oklahoma |
Principal Investigator: | George Buchanan, MD | University of Texas Southwestern Medical Center |
Principal Investigator: | Leslie Raffini, MD | University of Pennsylvania |
Principal Investigator: | Margaret Ragni, MD | Hemophilia Center of Western Pennsylvania |
Principal Investigator: | Miguel Escobar, MD | The University of Texas Health Science Center, Houston |
Principal Investigator: | Melissa Frei-Jones, MD | University of Texas Health Science Center San Antonio |
Principal Investigator: | Guy Young, MD | Children's Hospital Los Angeles |
Principal Investigator: | Leonard Valentino, MD | Rush University Medical Center |
Responsible Party: | New England Research Institutes ( Susan F. Assmann ) |
Study ID Numbers: | 374, U01 HL072268-04 |
Study First Received: | May 26, 2006 |
Last Updated: | October 20, 2008 |
ClinicalTrials.gov Identifier: | NCT00331006 |
Health Authority: | United States: Food and Drug Administration |
Blood Coagulation Factor Inhibitors |
Hemorrhagic Disorders Genetic Diseases, Inborn Rituximab Hematologic Diseases |
Blood Coagulation Disorders Hemophilia A Hemostatic Disorders |
Blood Coagulation Disorders, Inherited Immunologic Factors Antineoplastic Agents Coagulation Protein Disorders |
Therapeutic Uses Physiological Effects of Drugs Antirheumatic Agents Pharmacologic Actions |