Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS
- Conditions
- Hemophilia AHemophilia B
- Interventions
- Biological: ADVATE (Antihemophilic Factor [Recombinant])Biological: RIXUBIS (Coagulation Factor IX [Recombinant])
- Registration Number
- NCT02190149
- Lead Sponsor
- Baxalta now part of Shire
- Brief Summary
This 6-month prospective outcomes study addresses the association between timing of infusion, patient activity levels, and bleeding episodes through patient-reported measurements.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 64
-
13 to 65 years old at the time of screening
-
has moderately severe or severe hemophilia A or B (FVIII/FIX level
≤2%), with or without transient inhibitors
-
Hemophilia A patient currently prescribed ADVATE (FVIII) or Hemophilia B patient currently prescribed RIXUBIS (FIX)
-
previously treated with plasma-derived FVIII/FIX concentrates or recombinant FVIII/FIX for ≥150 documented exposure days (EDs)
-
is willing and able to comply with the requirements of the protocol
-
is proficient in the English language to allow for use of the SPACE eDiary
- inhibitor titer ≥0.6 Bethesda units or currently being treated for an inhibitor
- has participated in another clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving a medicinal product or device during the course of this study
- is a family member or employee of the investigator
- elective major surgery is planned within 6 months after enrollment which may interfere with activities of daily living (at investigator's discretion)
- continuously require walking assistance devices (eg, wheelchair, crutches, etc.)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description ADVATE (Factor VIII) ADVATE (Antihemophilic Factor [Recombinant]) Participants will remain on their current (pre-study) treatment regimen of ADVATE throughout the study period RIXUBIS (Factor IX) RIXUBIS (Coagulation Factor IX [Recombinant]) Participants will remain on their current (pre-study) treatment regimen of RIXUBIS throughout the study period
- Primary Outcome Measures
Name Time Method Advancing the understanding of the relationship between patient physical activity levels, infusion and occurrence of bleeding episodes (BEs) 6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months After the informed consent form is signed, retrospective data on physical activity levels, infusion and occurrence of BEs will be collected. The retrospective period will be the 6 months prior to signing the ICF.
- Secondary Outcome Measures
Name Time Method Infusion amount (either ADVATE (Hemophilia A), or RIXUBIS (Hemophilia B) Baseline through 6 months Types of current physical activities of participants Baseline through 6 months Activity measurement Baseline through 6 months Including: calories burned, steps taken, distance traveled, and active minutes
Patient-reported outcome: pediatric Hemophilia Activities List (pedHAL) questionnaire - for pediatric patients Baseline and 6 months The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for participants 13-17 years of age:
Occurrence of bleeding episodes (BEs) 6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months After the informed consent form is signed, retrospective data on physical activity levels, infusion and occurrence of BEs will be collected. The retrospective period will be the 6 months prior to signing the ICF.
Infusion schedule for participants on prophylaxis Baseline through 6 months Patient-reported outcome: Hemophilia Activities List (HAL) questionnaire - for adult patients Baseline and 6 months The HAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults.
Trial Locations
- Locations (17)
Johns Hopkins University School Of Medicine
🇺🇸Baltimore, Maryland, United States
North Shore/Long Island Jewish PRIME
🇺🇸Great Neck, New York, United States
Rady Childrens Hospital
🇺🇸San Diego, California, United States
Children's Mercy Hospitals & Clinics
🇺🇸Kansas City, Missouri, United States
Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center
🇺🇸Los Angeles, California, United States
Childrens Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Michigan State University
🇺🇸East Lansing, Michigan, United States
Munson Medical Center
🇺🇸Traverse City, Michigan, United States
University of Colorado
🇺🇸Aurora, Colorado, United States
Bleeding and Clotting Disorders Institute
🇺🇸Peoria, Illinois, United States
The Presbyterian Hospital
🇺🇸Charlotte, North Carolina, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
The Vanderbilt Clinic
🇺🇸Nashville, Tennessee, United States
The University of Texas MD Anderson
🇺🇸Harlingen, Texas, United States
Puget Sound Blood Center
🇺🇸Seattle, Washington, United States
University of Kentucky Medical Center
🇺🇸Lexington, Kentucky, United States