Clinical trial NCT00782470: Evaluation of the Reasons and Consequences of Bleeding in Late Teens and Early Adulthood Patients With Severe Hemophilia A
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hematologic Disease
Blood Coagulation Disorders
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2008-10-31
Enrollment: 38
Sex: MALE
Minimum age (years): 14
Maximum age (years): 29
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Cohort 1 - Prospective Prophylaxis Group Cohort 2 - Prospective On-demand Group Cohort 3 - Retrospective On-demand Group

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.


Eligibility criteria: 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

Primary outcomes:
measure: To evaluate the frequency of all bleeds (spontaneous and trauma) during the study
timeFrame: End of Study
Secondary outcomes:
measure: To evaluate the change from baseline in HRQoL (health-related quality of life)
timeFrame: End of Study
measure: To evaluate number of patients that want to return to prophylaxis treatment after having switched to on-demand therapy
timeFrame: End of Study
measure: To evaluate the change from baseline in the Gilbert score
timeFrame: End of Study
measure: To evaluate the number of target joint development
timeFrame: End of Study
Other outcomes:
Locations:
city: Duarte
country: United States
state: California
city: Denver
country: United States
state: Colorado
city: Peoria
country: United States
state: Illinois
city: Jefferson City
country: United States
state: Missouri
city: Las Vegas
country: United States
state: Nevada
city: Fort Worth
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: Many Locations
country: Canada
city: Many Locations
country: Germany
city: Many Locations
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Manco-Johnson MJ, Sanders J, Ewing N, Rodriguez N, Tarantino M, Humphries T; TEEN/TWEN Study Group. Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study. Haemophilia. 2013 Sep;19(5):727-35. doi: 10.1111/hae.12177. Epub 2013 Jun 11.