NCT04131036 · Washington Institute for Coagulation
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
(EmiMSK)
What this study is about
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current the usual treatment, most patients are on prophylactic FVIII replacement therapy given through a vein (IV) 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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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)
Where
- Los Angeles, California
- San Diego, California
- San Antonio, Texas
- Seattle, Washington
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 5, 2023 · Source of record for eligibility and locations