Hemophilia A, Hemophilia B
Conditions
Keywords
Factor VIII Deficiency, Factor FIX Deficiency
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.
Interventions
BIOLOGICALADVATE (Antihemophilic Factor [Recombinant])
BIOLOGICALRIXUBIS (Coagulation Factor IX [Recombinant])
Sponsors
Baxalta now part of Shire
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
13 Years to 65 Years
Healthy volunteers
No
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.)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Activity measurement | Baseline through 6 months | Including: calories burned, steps taken, distance traveled, and active minutes |
| Infusion schedule for participants on prophylaxis | Baseline through 6 months | — |
| 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 | — |
| 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. |
| 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. |
Countries
United States
Outcome results
None listed