Evaluation of the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Patients With Severe Hemophilia A

A Prospective, Non-interventional, Multi-center, Open-label Study to Evaluate the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Subjects With Severe Hemophilia A Receiving Prophylaxis and On-demand Treatment Regimen

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00782470

Enrollment

Registered

2008-10-31

Start date

2007-12-31

Completion date

2010-11-30

Last updated

2014-11-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Blood Coagulation Disorders, Hematologic Disease

Brief summary

Understanding how often the bleeding events occur in the subjects who voluntarily decide to switch from prophylaxis to on-demand and in those subjects who remain on prophylaxis. Also look into the consequences of switching treatment in QoL (quality of life), development of target joints, activity level and reasons that might influence the desire to switch.

Interventions

BEHAVIORALRecombinant Factor VIII (Kogenate, BAY 14-2222)

Subjects electing to stay on the prophylactic treatment (prospective)

Study design

Observational model

COHORT

Eligibility

Sex/Gender

MALE

Age

14 Years to 29 Years

Healthy volunteers

Inclusion criteria

* Severe hemophilia A (\<2%) * For subjects who elect staying on Prophylaxis only: Have been on continuous prophylactic treatment for the past 5 years prior to study entry * For subjects who elect switching to on-demand only: Have been on continuous prophylactic treatment for the past 5 years, but may have been on intermediate or reduced prophylaxis for the 1-12 months prior to study entry * For subjects currently on-demand: a retrospective arm of subjects who have been on continuous prophylactic treatment for at least 5 years and switched to on-demand treatment between 13 and 24 months prior to study entry * Current treatment with rFVIII

Exclusion criteria

* Other known hematological / bleeding disorders other than hemophilia A * Participating on another study that may have an impact on bleeding or the objectives of this study * Known alcohol and drug abuse

Design outcomes

Primary

Measure	Time frame
To evaluate the frequency of all bleeds (spontaneous and trauma) during the study	End of Study

Secondary

Measure	Time frame
To evaluate the change from baseline in HRQoL (health-related quality of life)	End of Study
To evaluate number of patients that want to return to prophylaxis treatment after having switched to on-demand therapy	End of Study
To evaluate the change from baseline in the Gilbert score	End of Study
To evaluate the number of target joint development	End of Study

Countries

Canada, Germany, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026