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CompletedNCT02093065Updated Jul 14, 2020

Platelet Function in Patients With Hemophilia A

An observational study in Hemophilia A, sponsored by Boston Children's Hospital. Completed at 1 site in United States. Open to male participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2020-07-14.

Sponsored by Boston Children's Hospital · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
36
Ages
2 Years to 18 Years
Sex
Male
01

Study 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.

02

Conditions studied

  • Hemophilia A

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Keywords

  • severe hemophilia A
  • coated platelets
  • bleeding
03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 36 is below the median of 80 across 314 observational studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Boston Children's Hospital is the lead sponsor of 598 studies on the registry; 151 are open to participants now.

Of its 31 completed or terminated interventional studies of FDA-regulated products, 18 (58%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 18 Years
Sexes eligible
Male
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients with severe hemophilia A who do not have inhibitors against FVIII and who have a bleeding history of at least 6 months.

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

Exclusion Criteria:

  • Presence of FVIII inhibitors.
  • Greater than 7 days since active bleeding.
05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
36 participants (actual)
Patient registry
No
Biospecimen retention
Samples with dna
06

What researchers measure

Primary outcomes

  1. The percentage of coated platelets.

    Time frame: 2 years

Secondary outcomes

  1. Platelet reactivity.

    Time frame: 2 years

  2. The number of procoagulant platelet-derived microparticles.

    Time frame: 2 years

07

Study locations

1 site
  • Boston Children's Hospital, Boston Hemophilia Center
    Boston, Massachusetts 02115, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 14, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT02093065
Lead sponsor
Boston Children's Hospital
Collaborators
Baxter Healthcare Corporation
Responsible party
Alan D. Michelson (Professor of Pediatrics and Professor of Medicine, Harvard Medical School Director, Center for Platelet Research Studies Director, Thrombosis and Anticoagulation Program Boston Children's Hospital / Dana-Farber Cancer Institute, Boston Children's Hospital) — Principal investigator
First posted
Mar 20, 2014
Start date
Mar 2015
Primary completion
Jun 2018
Completion
Feb 1, 2020
Last update
Jul 14, 2020

Study contacts

Alan D Michelson, MD
principal investigator · Boston Children's Hospital

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jul 2020. You cannot join it, but the record below documents what was studied.

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