Clinical trial NCT02190149: Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS
Official title: Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Hemophilia B
Keywords: Factor VIII Deficiency
Factor FIX Deficiency
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2014-07-15
Enrollment: 64
Sex: ALL
Minimum age (years): 13
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Hemophilia treatment center (HTCs) in the US will be identified and invited to enroll eligible patients in this study

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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.)

Primary outcomes:
measure: Advancing the understanding of the relationship between patient physical activity levels, infusion and occurrence of bleeding episodes (BEs)
timeFrame: 6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months
Secondary outcomes:
measure: Types of current physical activities of participants
timeFrame: Baseline through 6 months
measure: Activity measurement
timeFrame: Baseline through 6 months
measure: Infusion schedule for participants on prophylaxis
timeFrame: Baseline through 6 months
measure: Infusion amount (either ADVATE (Hemophilia A), or RIXUBIS (Hemophilia B)
timeFrame: Baseline through 6 months
measure: Occurrence of bleeding episodes (BEs)
timeFrame: 6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months
measure: Patient-reported outcome: Hemophilia Activities List (HAL) questionnaire - for adult patients
timeFrame: Baseline and 6 months
measure: Patient-reported outcome: pediatric Hemophilia Activities List (pedHAL) questionnaire - for pediatric patients
timeFrame: Baseline and 6 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center
state: California
city: San Diego
country: United States
facility: Rady Childrens Hospital
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Peoria
country: United States
facility: Bleeding and Clotting Disorders Institute
state: Illinois
city: Lexington
country: United States
facility: University of Kentucky Medical Center
state: Kentucky
city: Baltimore
country: United States
facility: Johns Hopkins University School Of Medicine
state: Maryland
city: Detroit
country: United States
facility: Childrens Hospital of Michigan
state: Michigan
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: Traverse City
country: United States
facility: Munson Medical Center
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospitals & Clinics
state: Missouri
city: Great Neck
country: United States
facility: North Shore/Long Island Jewish PRIME
state: New York
city: Charlotte
country: United States
facility: The Presbyterian Hospital
state: North Carolina
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: The Vanderbilt Clinic
state: Tennessee
city: Harlingen
country: United States
facility: The University of Texas MD Anderson
state: Texas
city: Seattle
country: United States
facility: Puget Sound Blood Center
state: Washington
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: