Hemophilia A Clinical Trial
— PPTIHOfficial title:
Study of Severe Hemophilia A Patients Who Have Only Received a Single Recombinant FVIII Therapeutic for the Purpose of Identifying the Pharmacogenetic Determinants of Tolerance and Immunogenicity
This study is designed to accurately identify the pharmacogenetic determinants of risk of Factor VIII (FVIII) inhibitor development by focusing on only a select group of Hemophilia A (HA) patients who have: (i) received a recombinant FVIII therapeutic product containing the same primary amino acid sequence since their original diagnosis; (ii) verifiable FVIII infusion histories; and (iii) been tested regularly for FVIII inhibitor development.
Status | Not yet recruiting |
Enrollment | 55 |
Est. completion date | June 2014 |
Est. primary completion date | April 2013 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 2 Years and older |
Eligibility |
Inclusion Criteria: - Patients with severe or moderately severe hemophilia A (HA) who have since birth been treated with only a single Factor VIII product (i.e., FVIII protein molecules containing only one primary amino acid sequence). Exclusion Criteria: - HA patients with severities other than severe or moderately severe. - Hemophilia B patients. - HA patients who have been treated with more than one FVIII product. - HA patients who have been treated with more than one FVIII product. - HA patients who do not have verifiable infusion histories. - HA patients who lack documentable inhibitor testing & infusion histories. |
Observational Model: Cohort, Time Perspective: Prospective
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Michigan | Detroit | Michigan |
Lead Sponsor | Collaborator |
---|---|
Victor J Marder, M.D. | Baxter Healthcare Corporation |
United States,
Howard TE, Yanover C, Mahlangu J, Krause A, Viel KR, Kasper CK, Pratt KP. Haemophilia management: time to get personal? Haemophilia. 2011 Sep;17(5):721-8. doi: 10.1111/j.1365-2516.2011.02517.x. Epub 2011 Jun 8. Review. — View Citation
Viel KR, Ameri A, Abshire TC, Iyer RV, Watts RG, Lutcher C, Channell C, Cole SA, Fernstrom KM, Nakaya S, Kasper CK, Thompson AR, Almasy L, Howard TE. Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med. 2009 Apr 16;360(16):1618-27. doi: 10.1056/NEJMoa075760. Erratum in: N Engl J Med. 2009 Jul 30;361(5):544. — View Citation
Yanover C, Jain N, Pierce G, Howard TE, Sauna ZE. Pharmacogenetics and the immunogenicity of protein therapeutics. Nat Biotechnol. 2011 Oct 13;29(10):870-3. doi: 10.1038/nbt.2002. — View Citation
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