Recruiting

Emicizumab vs. Efanesoctocog Alfa

Sponsor:

Arkansas Children's Hospital Research Institute

Code:

NCT07582276

Conditions

Hemophilia a

Eligibility Criteria

Sex: Male

Age: 30 - 50

Healthy Volunteers: Not accepted

Study Details

Brief summary:

This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa. Participants will undergo assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period. The study aims to evaluate whether differences in prophylactic therapy are associated with differences in bone health outcomes.

Conditions

Hemophilia a

Study ID

NCT07582276

Start date

Sep 21, 2026

Status verified date

Oct, 2026

Completion date

Sep, 2032

Anticipated

Primary completion date

Sep, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 30 - 50

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. The participant or legally authorized representative is willing and able to provide written informed consent.
2. Diagnosis of severe hemophilia A (factor VIII activity < 1%).
3. Male sex.
4. Age between 30 and 50 years (inclusive).
5. BMI between 18.5 and 40 kg/m2
6. The participant must have been on prophylaxis with Efanesoctocog alfa or Emicizumab for at least 3 months prior to enrollment and intend to remain on the current regimen for the next 5 years.
7. Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples.
8. Willingness to complete all standard-of-care bleeding and treatment logs.

Exclusion Criteria:

1. Unwillingness of the participant, parent, or legally authorized representative to provide informed consent.
2. Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A.
3. Active Factor VIII inhibitors at the time of enrollment
4. History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism)
5. Past or present treatment with any anti-osteoporotic medication, excluding oral vitamin D or oral calcium supplements.
6. Documented HIV infection or HCV infection (whether in progress or cured) at the cirrhotic stage.
7. Presence of a non-removable metal device that would interfere with research procedures.
8. Inability to tolerate a DEXA scan due to limited range of motion or body habitus.
9. History of bone fractures or surgical repair within 8 weeks prior to enrollment.
10. Participants with weight >300 pounds, due to limitations of DEXA scanner

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

Patients with Severe Hemophilia A on Efanesoctocog alfa prophylaxis

Participants with severe hemophilia A receiving prophylaxis with efanesoctocog alfa as part of routine clinical care. Participants will undergo longitudinal assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period.

Patients with Severe Hemophilia A on Emicizumab prophylaxis

Participants with severe hemophilia A receiving prophylaxis with emicizumab as part of routine clinical care. Participants will undergo longitudinal assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period.

Primary outcome measure

  • Longitudinal change in femoral neck bone mineral density (g/cm²) [ Time Frame: Baseline and annually through 5 years. ]

Central Contacts and Locations

Central contacts

Locations

Arkansas Children's

Recruiting

Little Rock, Arkansas, United States, 72202

More Information

Sponsor

Arkansas Children's Hospital Research Institute

Last update posted

Oct 5, 2026

Last verified

Oct, 2026

Keywords

  • Bone Health
  • Emicizumab
  • Efanesoctocog alfa
  • hemophilia

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-06. This information was provided to ClinicalTrials.gov by Arkansas Children's Hospital Research Institute on 2026-10-05. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.