NCT03358836

Brief Summary

This is a prospective, non-randomized, controlled study to examine whether or not having a higher trough during prophylactic treatment with clotting factor offers better joint protection than the standard trough of 1% Factor IX (FIX or Factor 9). This study will test the hypothesis that an extended half-life (EHL) FIX product with an intended trough of \>10% could offer better protection than previous treatment concentrates. This study also examines whether or not joint damage could be diagnosed earlier using ultrasound images.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
8

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Nov 2017

Typical duration for all trials

Geographic Reach
1 country

5 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

Study Start

First participant enrolled

November 15, 2017

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

November 25, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 2, 2017

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 17, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 17, 2020

Completed
Last Updated

June 22, 2022

Status Verified

June 1, 2022

Enrollment Period

2.8 years

First QC Date

November 25, 2017

Last Update Submit

June 17, 2022

Conditions

Hemophilia B

Keywords

Hemophilia BFIXFactor IXFactor 9

Outcome Measures

Primary Outcomes (1)

  • Joint health status

    Joint health status in all six major joints (elbows, knees, and ankles) in all three groups as assessed by ultrasound

    Change from baseline at up to three years

Secondary Outcomes (5)

  • Joint and overall health status

    Change from baseline at up to three years

  • Joint health at year 1

    Change from baseline at up to one year

  • Joint health at year 2

    Change from baseline at up to two years

  • Acute events/bleeding

    Change from baseline at up to three years

  • Biomarkers

    Change from baseline at up to three years

Study Arms (3)

Group A

Episodic treatment with FIX concentrates for bleeding episodes

Group B

Prophylaxis using any FIX concentrate with an intended trough of 1-5%

Group C

Prophylaxis with an extended half-life (EHL) FIX with an intended trough of \>10%

Eligibility Criteria

Age16 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participants are male, age 16 or older, have severe hemophilia B, and are currently on either an episodic or prophylactic treatment regimen with FIX concentrates

You may qualify if:

  • Severe hemophilia B (FIX \<1%)
  • Either on demand or on prophylaxis with rFIX or EHL-rIX products with the intention to stay on the current regimen for the next 3 years
  • For Group C, start of this treatment regimen up to 6 months prior is permissible

You may not qualify if:

  • Other known bleeding disorder
  • Other rheumatologic disorder affecting joints
  • Other known neuromotor defect (making physical exam difficult)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Orthopaedic Institute for Children at University of California Los Angeles

Los Angeles, California, 90007, United States

Location

University of California San Diego

San Diego, California, 92122, United States

Location

Tulane University

New Orleans, Louisiana, 70112, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239-3098, United States

Location

Washington Center for Bleeding Disorders at Bloodworks Northwest

Seattle, Washington, 98104, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

We will draw up to 40 mL (3 tablespoons) of blood to test for factor level, inhibitor level, and biomarkers

MeSH Terms

Conditions

Hemophilia B

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

Study Officials

  • Rebecca Kruse-Jarres, MD, MPH

    Washington Center for Bleeding Disorders at Bloodworks Northwest

    PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

First Submitted

November 25, 2017

First Posted

December 2, 2017

Study Start

November 15, 2017

Primary Completion

August 17, 2020

Study Completion

August 17, 2020

Last Updated

June 22, 2022

Record last verified: 2022-06

Data Sharing

IPD Sharing
Will not share

Locations

US(5)