A Multi-institution Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)

NCT07582276 | Not Yet Recruiting

• 1 other identifier: 300130
• Study Type: observational
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is comparing bone mineral density in patients with Severe Hemophilia A on Emicizumab prophylaxis vs patients on Efanesoctocog alfa prophylaxis

Conditions

Hemophilia a

Keywords

Bone Health; Emicizumab; Efanesoctocog alfa

Outcome Measures

Primary Outcomes (2)

• the longitudinal change in femoral neck bone mineral density (BMD, g/cm²)

  The primary endpoint of this study is the longitudinal change in femoral neck bone mineral density(BMD, g/cm²)

  Over a 5 year period

• Change in femoral neck bone mineral density

  The primary endpoint of this study is the longitudinal change in femoral neck bone mineral density (BMD, g/cm²)

  over a five-year period

Study Arms (2)

patients with Severe Hemophilia A on factor VIII prophylaxis

patients with Severe Hemophilia A on Efa Emi prophylaxis

Eligibility Criteria

• Age: 30 Years - 50 Years
• Gender Eligibility Details: Males
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients that are seen at dedicated Hemophilia treatment Centers

You may qualify if:

• The participant or legally authorized representative is willing and able to provide written informed consent.
• Diagnosis of severe hemophilia A (factor VIII activity \< 1%).
• Male sex.
• Age between 30 and 50 years (inclusive).
• BMI between 18.5 and 40 kg/m2
• 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.
• Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples.
• Willingness to complete all standard-of-care bleeding and treatment logs.

You may not qualify if:

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

Sponsors & Collaborators

• Arkansas Children's Hospital Research Institute: lead
• Genzyme, a Sanofi Company: collaborator

Related Publications (13)

• Sivagurunathan S, Pagel CN, Loh LH, Wijeyewickrema LC, Pike RN, Mackie EJ. Thrombin inhibits osteoclast differentiation through a non-proteolytic mechanism. J Mol Endocrinol. 2013 Apr 23;50(3):347-59. doi: 10.1530/JME-12-0177. Print 2013 Jun.

  PMID: 23419317 BACKGROUND

• Song SJ, Pagel CN, Campbell TM, Pike RN, Mackie EJ. The role of protease-activated receptor-1 in bone healing. Am J Pathol. 2005 Mar;166(3):857-68. doi: 10.1016/S0002-9440(10)62306-1.

  PMID: 15743797 BACKGROUND

• Pagel CN, Song SJ, Loh LH, Tudor EM, Murray-Rust TA, Pike RN, Mackie EJ. Thrombin-stimulated growth factor and cytokine expression in osteoblasts is mediated by protease-activated receptor-1 and prostanoids. Bone. 2009 May;44(5):813-21. doi: 10.1016/j.bone.2008.12.031. Epub 2009 Jan 15.

  PMID: 19442625 BACKGROUND

• Pagel CN, de Niese MR, Abraham LA, Chinni C, Song SJ, Pike RN, Mackie EJ. Inhibition of osteoblast apoptosis by thrombin. Bone. 2003 Oct;33(4):733-43. doi: 10.1016/s8756-3282(03)00209-6.

  PMID: 14555279 BACKGROUND

• Al Dieri R, de Laat B, Hemker HC. Thrombin generation: what have we learned? Blood Rev. 2012 Sep;26(5):197-203. doi: 10.1016/j.blre.2012.06.001. Epub 2012 Jul 2.

  PMID: 22762893 BACKGROUND

• Goldscheitter G, Recht M, Sochacki P, Manco-Johnson M, Taylor JA. Biomarkers of bone disease in persons with haemophilia. Haemophilia. 2021 Jan;27(1):149-155. doi: 10.1111/hae.13986. Epub 2020 Aug 27.

  PMID: 32856388 BACKGROUND

• Gerstner G, Damiano ML, Tom A, Worman C, Schultz W, Recht M, Stopeck AT. Prevalence and risk factors associated with decreased bone mineral density in patients with haemophilia. Haemophilia. 2009 Mar;15(2):559-65. doi: 10.1111/j.1365-2516.2008.01963.x. Epub 2009 Feb 1.

  PMID: 19187193 BACKGROUND

• Wallny TA, Scholz DT, Oldenburg J, Nicolay C, Ezziddin S, Pennekamp PH, Stoffel-Wagner B, Kraft CN. Osteoporosis in haemophilia - an underestimated comorbidity? Haemophilia. 2007 Jan;13(1):79-84. doi: 10.1111/j.1365-2516.2006.01405.x.

  PMID: 17212729 BACKGROUND

• Paschou SA, Anagnostis P, Karras S, Annweiler C, Vakalopoulou S, Garipidou V, Goulis DG. Bone mineral density in men and children with haemophilia A and B: a systematic review and meta-analysis. Osteoporos Int. 2014 Oct;25(10):2399-407. doi: 10.1007/s00198-014-2773-7. Epub 2014 Jul 8.

  PMID: 25001982 BACKGROUND

• Ghosh K, Shetty S. Bone health in persons with haemophilia: a review. Eur J Haematol. 2012 Aug;89(2):95-102. doi: 10.1111/j.1600-0609.2012.01803.x. Epub 2012 Jun 22.

  PMID: 22587752 BACKGROUND

• Walker IR, Julian JA. Causes of death in Canadians with haemophilia 1980-1995. Association of Hemophilia Clinic Directors of Canada. Haemophilia. 1998 Sep;4(5):714-20. doi: 10.1046/j.1365-2516.1998.00179.x.

  PMID: 9873876 BACKGROUND

• Smit C, Rosendaal FR, Varekamp I, Brocker-Vriends A, Van Dijck H, Suurmeijer TP, Briet E. Physical condition, longevity, and social performance of Dutch haemophiliacs, 1972-85. BMJ. 1989 Jan 28;298(6668):235-8. doi: 10.1136/bmj.298.6668.235.

  PMID: 2493872 BACKGROUND

• Bunta AD. It is time for everyone to own the bone. Osteoporos Int. 2011 Aug;22 Suppl 3:477-82. doi: 10.1007/s00198-011-1704-0. Epub 2011 Aug 17.

  PMID: 21847769 BACKGROUND

Biospecimen

Retention: SAMPLES WITHOUT DNA

The following analytes will be processed: markers of bone formation, including Procollagen Type 1 N-terminal Propeptide (PINP); markers of bone resorption, including C-terminal Telopeptide of Type I Collagen (CTX1); markers of osteoclast activity, including Osteoprotegerin (OPG) and Receptor Activator of Nuclear Factor Kappa- B Ligand (RANKL); cytokine profiles, including Interleukin-1 Beta (IL-1β), Interleukin-6 (IL-6), and Tumor Necrosis Factor-alpha Samples will be retained for five years following study completion, after which they will be destroyed according to biohazard waste disposal procedures. Only study personnel knowledgeable about the study and adhering to U.S. Department of Transportation regulations for biohazard transport will handle these specimens.

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Divyaswathi Citla Sridhar, MD

  Arkansas Children's Reserach Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carol D Pierce, RN

CONTACT

501-364-4440; piercecarold@uams.edu

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2026
• First Posted: May 12, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): August 1, 2031
• Study Completion (Estimated): August 1, 2032
• Last Updated: May 12, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share