Platelet Function in Patients With Hemophilia A

Hemophilia A Clinical Trial

Official title:

Decreased Platelet Function as a Cause of Increased Bleeding in Patients With Hemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02093065
• Other study ID #: BCH-CPRS-hemophilia A
• Status: Completed
• Start date: March 2015
• Est. completion date: February 1, 2020

Study information

• Verified date: July 2020
• Source: Boston Children’s Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are classified into 3 different categories based on residual FVIII activity compared to normal: mild (6-40%), moderate (1-5%) and severe (<1%). This categorization correlates to some degree with bleeding phenotype, but does not completely define it. Some patients with hemophilia A bleed less often than others despite identical plasma FVIII levels. The cause(s) of this phenotype heterogeneity in hemophilia A remains largely unknown, despite a number of studies of possible factors.

Activated platelets, in addition to their role in primary hemostasis, play a major role in secondary hemostasis (coagulation) by providing a phospholipid surface to which coagulation factors bind. A role for platelets in the hemorrhagic propensity of hemophilia A has been suggested in the past, but only a small number of studies have been performed with limitations in assays performed and numbers of patients. The purpose of the present study is to determine whether platelet reactivity in severe hemophilia A patients is associated with past bleeding frequency and/or predicts future bleeding frequency.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 36
• Est. completion date: February 1, 2020
• Est. primary completion date: June 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 18 Years

Eligibility

Inclusion Criteria:

• Patients with severe hemophilia A who are being prophylactically treated with FVIII.

• Age of at least 2 years.

• Bleeding history of at least 6 months.

• IRB-approved informed consent.

Exclusion Criteria:

• Presence of FVIII inhibitors.

• Greater than 7 days since active bleeding.

Related Conditions & MeSH terms

• Hemophilia A

Locations

Country	Name	City	State
United States	Boston Children's Hospital, Boston Hemophilia Center	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Boston Children’s Hospital	Baxter Healthcare Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The percentage of coated platelets.		2 years
Secondary	Platelet reactivity.		2 years
Secondary	The number of procoagulant platelet-derived microparticles.		2 years