NCT02190149

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
64

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jul 2014

Geographic Reach
1 country

17 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 14, 2014

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 15, 2014

Completed
7 days until next milestone

Study Start

First participant enrolled

July 22, 2014

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 29, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 29, 2016

Completed
Last Updated

March 17, 2021

Status Verified

March 1, 2021

Enrollment Period

1.7 years

First QC Date

July 14, 2014

Last Update Submit

March 16, 2021

Conditions

Keywords

Factor VIII DeficiencyFactor FIX Deficiency

Outcome Measures

Primary Outcomes (1)

  • Advancing the understanding of the relationship between patient physical activity levels, infusion and occurrence of bleeding episodes (BEs)

    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.

    6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months

Secondary Outcomes (7)

  • Types of current physical activities of participants

    Baseline through 6 months

  • Activity measurement

    Baseline through 6 months

  • Infusion schedule for participants on prophylaxis

    Baseline through 6 months

  • Infusion amount (either ADVATE (Hemophilia A), or RIXUBIS (Hemophilia B)

    Baseline through 6 months

  • Occurrence of bleeding episodes (BEs)

    6 months retrospectively (after the informed consent form (ICF) is signed) through 6 months

  • +2 more secondary outcomes

Study Arms (2)

ADVATE (Factor VIII)

Participants will remain on their current (pre-study) treatment regimen of ADVATE throughout the study period

Biological: ADVATE (Antihemophilic Factor [Recombinant])

RIXUBIS (Factor IX)

Participants will remain on their current (pre-study) treatment regimen of RIXUBIS throughout the study period

Biological: RIXUBIS (Coagulation Factor IX [Recombinant])

Interventions

Also known as: Octocog alfa
ADVATE (Factor VIII)

Eligibility Criteria

Age13 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Hemophilia treatment center (HTCs) in the US will be identified and invited to enroll eligible patients in this study

You may qualify if:

  • 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

You may not qualify if:

  • 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.)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center

Los Angeles, California, 90007, United States

Location

Rady Childrens Hospital

San Diego, California, 92123, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61615, United States

Location

University of Kentucky Medical Center

Lexington, Kentucky, 40504, United States

Location

Johns Hopkins University School Of Medicine

Baltimore, Maryland, 21205, United States

Location

Childrens Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Michigan State University

East Lansing, Michigan, 48823, United States

Location

Munson Medical Center

Traverse City, Michigan, 49684, United States

Location

Children's Mercy Hospitals & Clinics

Kansas City, Missouri, 64108, United States

Location

North Shore/Long Island Jewish PRIME

Great Neck, New York, 11021, United States

Location

The Presbyterian Hospital

Charlotte, North Carolina, 28210, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

The Vanderbilt Clinic

Nashville, Tennessee, 37232, United States

Location

The University of Texas MD Anderson

Harlingen, Texas, 78550, United States

Location

Puget Sound Blood Center

Seattle, Washington, 98104, United States

Location

Related Links

MeSH Terms

Conditions

Hemophilia AHemophilia B

Interventions

Factor VIIIF8 protein, humanFactor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological FactorsEnzyme PrecursorsEnzymes and Coenzymes

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 14, 2014

First Posted

July 15, 2014

Study Start

July 22, 2014

Primary Completion

March 29, 2016

Study Completion

March 29, 2016

Last Updated

March 17, 2021

Record last verified: 2021-03

Locations