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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT00782470
First received: October 29, 2008
Last updated: October 20, 2013
Last verified: October 2013
  Purpose

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.


Condition Intervention
Hematologic Disease
Blood Coagulation Disorders
Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222)

Study Type: Observational
Study Design: Observational Model: Cohort
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

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • To evaluate the frequency of all bleeds (spontaneous and trauma) during the study [ Time Frame: End of Study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To evaluate the change from baseline in HRQoL (health-related quality of life) [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • To evaluate number of patients that want to return to prophylaxis treatment after having switched to on-demand therapy [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • To evaluate the change from baseline in the Gilbert score [ Time Frame: End of Study ] [ Designated as safety issue: No ]
  • To evaluate the number of target joint development [ Time Frame: End of Study ] [ Designated as safety issue: No ]

Enrollment: 38
Study Start Date: December 2007
Study Completion Date: November 2010
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Group 1 Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222)
Subjects electing to stay on the prophylactic treatment (prospective)
Group 2 Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222)
Subjects electing to switch to on-demand treatment (prospective)
Group 3 Behavioral: Recombinant Factor VIII (Kogenate, BAY 14-2222)
Subjects remaining on-demand treatment (retrospective)

  Eligibility

Ages Eligible for Study:   14 Years to 29 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Cohort 1 - Prospective Prophylaxis Group Cohort 2 - Prospective On-demand Group Cohort 3 - Retrospective On-demand Group

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00782470

Locations
United States, California
Duarte, California, United States, 91010
United States, Colorado
Denver, Colorado, United States, 80262
United States, Illinois
Peoria, Illinois, United States, 61614
United States, Missouri
Jefferson City, Missouri, United States, 65109
United States, Nevada
Las Vegas, Nevada, United States, 89109
United States, Texas
Fort Worth, Texas, United States, 76104
Houston, Texas, United States, 77030
Canada
Many Locations, Canada
Germany
Many Locations, Germany
United Kingdom
Many Locations, United Kingdom
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
No publications provided

Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT00782470     History of Changes
Other Study ID Numbers: 12749
Study First Received: October 29, 2008
Last Updated: October 20, 2013
Health Authority: United States: Aspire Independent Review Board

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Hemophilia A
Hemorrhage
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Coagulation Protein Disorders
Genetic Diseases, Inborn
Pathologic Processes
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 22, 2014