Hemophilia Related Clinical Trials

Baxter Previously Untreated Patients
This is a new study. Subjects may be in the study for 75 exposures or 3 years. Visits are every 3 months. Inclusion criteria are: the subject must be less than 6 years old, the Factor VIII level must be less than 2% and they must have less than 3 exposures to Advate. Exclusion criteria include: exposure to Factor VIII other than Advate; you must not have a detectable Factor VIII inhibitor; you must not be HIV, Hepatitis B or Hepatitis C positive; you must not have received any blood products.

Data Collection on Complications of Hemophilia and Serum Testing and Storage (CDC/UDC)
The HTC has been involved with this study since 1997. To be a subject, a person must have a diagnosis of hemophilia (either factor VIII or factor IX deficiency), von Willebrand Disease, or an acquired inhibitor. The purpose of the study is to monitor the health status of people who are diagnosed with the previously mentioned bleeding disorders. The study is conducted at the time you are seen for your comprehensive hemophilia clinic.

The Second Multicenter Hemophilia Cohort Study (MHCS-II)
To be eligible to enroll in this study you must have a diagnoses of a bleeding disorder factor (V, VIII, IX, XI or von Willebrand Disease) and be Hepatitis C positive. The purpose of the study is to evaluate and follow subjects with an inherited bleeding disorder who have been infected with Hepatitis C. It is hoped that this study will improve the understanding of hemophilia care and its complications.

Genetics of Inhibitor Formation in Hemophilia
To be eligible to participate in this study you have to be diagnosed with a severe factor VIII deficiency and have a brother who has also been diagnosed with a severe factor VIII deficiency. In addition one of you has to have been diagnosed with an inhibitor, or had a history of inhibitors. The purpose of the study is to complete laboratory tests to find out whether there are differences from person to person that might explain why some people with hemophilia develop inhibitors and some people do not. Once we have a better understanding of inhibitor development, we will have a better chance to design treatment to get rid of inhibitors or even be able to prevent them form occurring.

International, Randomized, Controlled Trial of Immune-Tolerance Induction
To be eligible to participate in this study you have to be diagnosed with a severe factor VIII deficiency, have a high titer factor VIII inhibitor, be less than 7 years old, and your inhibitor will need to be less than 10 BU before you can start the study. The purpose of the study is to compare different treatment plans for the treatment of inhibitors.

Hemophilic Genotype, Inhibitors and Response to Immune Tolerance: A Prospective Companion Study
To be eligible for this study you have to be less than 7 years old, have been diagnosed with a severe factor VIII deficiency, and have had an inhibitor for less than a year. You would have to be enrolled in the "International, Randomized, Controlled trial of Immune Tolerance Induction" study to enroll in this study. The purpose of the study is to learn if certain kinds of genetic structures will predict how someone with a severe factor VIII deficiency will respond to factor VIII replacement.

HTRS Registry
The purpose of this study is to learn how to improve treatment for bleeding in persons with inhibitors.

The Hemophilia and Thrombosis Research Society (HTRS) has established a national patient registry through which a common set of data are collected from Hemophilia Treatment Centers (HTCs) to gain a better understanding of the pathophysiology of hemophilia and other coagulation disorders; the current clinical management of patients with these disorders; and the safety and efficacy of alternative treatments.

Role of Genetic Modifiers in Bleeding Disorders (NIH Study)
This study is open to enrollment for people with vWD, Gray Platelet Syndrome and platelet disorders. It requires a one-time visit where you will complete a bleeding questionnaire and have a blood drawn.

Wyeth BeneFIX Study
This is a prophylactic study for severe factor IX deficient patients. The subject will participate for 15 months. They will be randomized to 2 different doses of BeneFIX-R.

Wyeth ReFacto AF surgical Study
Subjects will be given ReFacto AF during a surgical procedure. Presurgical lab work will need to be done. Subjects must be older than 12 years of age and their factor VIII level must be equal to or less than 2 percent.

Thrombophilia Registry
Subjects will be followed for 2 years after having a thrombophilic episode. There are no study interventions - only data collection.

New studies beginning in the fall 2007:
Novo Nordisk will have 2 new prophylactic studies for factor VIII deficient patients with inhibitors.

Wyeth will be starting a new study for children under age 6 using ReFacto AF. The subjects will be randomized to one of two prophylactic doses or continue with on-demand treatment. The study will last for 2 years.

Both Bayer and Baxter are working on long-acting factor studies.

For more information about these trials, or to participate in research, contact Joan Kempf, Study Coordinator, at (319) 356-0529, or joan-kempf@uiowa.edu.