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Trial record 20 of 46 for:    "Hemophilia" | "Antibodies"

Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A (EmiMSK)

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ClinicalTrials.gov Identifier: NCT04131036
Recruitment Status : Recruiting
First Posted : October 18, 2019
Last Update Posted : October 18, 2019
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Rebecca Kruse-Jarres, Bloodworks

Tracking Information
First Submitted Date October 14, 2019
First Posted Date October 18, 2019
Last Update Posted Date October 18, 2019
Actual Study Start Date April 4, 2019
Estimated Primary Completion Date August 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 16, 2019)
joint health comparison [ Time Frame: 3 years ]
Joint health changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by MSKUS at baseline and after 3 years.
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: October 16, 2019)
  • bone density comparison [ Time Frame: 3 years ]
    Bone density changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by DEXA scan at baseline and after 3 years.
  • comparative assessment of joint and overall health [ Time Frame: 3 years ]
    Observational comparative assessment of joint and overall health status evaluated by activity level, functional assessment, pain assessment, joint examination, and adherence.
  • mid-point assessment of joint health and bone density [ Time Frame: 2 years ]
    Observational assessment of joint health and bone density at 1 and 2 years in the different groups.
  • biomarkers for joint and bone health [ Time Frame: through completion of study, average 3 years ]
    Exploration of potential biomarkers for joint and bone health.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Official Title Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Brief Summary The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough >1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.
Detailed Description

Summary: This retrospective/prospective, non-randomized controlled study, whereby patients will be included in 1 of 2 groups depending on their current treatments as outlined below. Patients will be assigned to a specific group by the clinician/PI based on their current regimen with the intent to stay on this regimen for the next 3 years.

Duration of Study and Subject Participation: Study subject enrollment is anticipated to occur over about 12 months and study duration per subject will be 3 years. Thus a total study duration of 4 years is anticipated.

Intervention: By using musculoskeletal ultrasound, the investigators will measure joint health among participants and assess joint health changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by MSKUS at baseline and after 3 years. Other outcomes such as bone density, comparative assessment of joint and overall health status, and exploration of potential biomarkers for joint and bone health will also be assessed.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Plasma samples for biomarkers for storage in the data and sample research repository at Bloodworks Northwest
Sampling Method Non-Probability Sample
Study Population Male severe Hemophilia A patients at one of the several Hemophilia Treatment Center sites participating in this study and who meet the eligibility criteria and are interested in participating in the study.
Condition Hemophilia A
Intervention Other: assessment of joint health and bone density
We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.
Study Groups/Cohorts
  • Arm A
    Male patients with severe Hemophilia A who use prophylaxis with IV factor VIII concentrate with intended trough >1%.
    Intervention: Other: assessment of joint health and bone density
  • Arm B
    Male patients with severe Hemophilia A who use prophylaxis with SQ emicizumab.
    Intervention: Other: assessment of joint health and bone density
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 16, 2019)
40
Original Estimated Enrollment Same as current
Estimated Study Completion Date August 2023
Estimated Primary Completion Date August 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Male gender
  • Severe hemophilia A (factor VIII < 1%)
  • Age ≥ 16 year
  • Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
  • Willing and able to give written informed consent/assent
  • Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
  • Willing to come in for baseline and 3 yearly visits
  • Willing to answer phone survey for bleeding and safety every 3 months

Exclusion Criteria:

  • Current FVIII inhibitor of > 0.6 BU
  • Unable to take FVIII replacement
  • Other known bleeding disorder
  • Other rheumatologic disorder affecting joints
  • Other known neuromotor defect (making physical exam difficult)
Sex/Gender
Sexes Eligible for Study: Male
Ages 16 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Rebecca Kruse-Jarres, MD, MPH (206) 292-6500 RebeccaKr@BloodWorksNW.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04131036
Other Study ID Numbers ML40714
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Rebecca Kruse-Jarres, Bloodworks
Study Sponsor Bloodworks
Collaborators Genentech, Inc.
Investigators
Principal Investigator: Rebecca Kruse-Jarres, MD, MPH Bloodworks NW
PRS Account Bloodworks
Verification Date October 2019