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

3. juni 2026 opdateret af: Divyaswathi Citla Sridhar, Arkansas Children's Hospital Research Institute

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)

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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Detaljeret beskrivelse

Reduced bone mineral density, osteoporosis, and fractures are increasingly recognized in persons with severe hemophilia A. The mechanisms underlying impaired bone health in hemophilia are multifactorial and may include reduced physical activity, chronic joint disease, inflammation, and abnormalities in coagulation-related pathways involved in bone remodeling.

Thrombin has been shown to play an important role in bone metabolism through activation of protease-activated receptor-1 (PAR-1) signaling pathways that influence osteoblast and osteoclast activity. Reduced thrombin generation in severe hemophilia A may contribute to decreased bone formation and increased bone resorption.

Efanesoctocog alfa is an extended half-life factor VIII replacement therapy that maintains higher circulating factor VIII levels and supports thrombin generation. Emicizumab is a non-factor prophylactic therapy that effectively prevents bleeding but does not replace factor VIII. The comparative effects of these therapies on long term bone health have not been well established.

This prospective observational study will compare longitudinal changes in bone mineral density among patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa over 5 years. Participants will undergo serial dual-energy X-ray absorptiometry (DXA) assessments and evaluation of bone remodeling biomarkers, inflammatory cytokines, thrombin generation, plasmin generation, and joint health over a five-year period.

Undersøgelsestype

Observationel

Tilmelding (Anslået)

50

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen

Tager imod sunde frivillige

Ingen

Prøveudtagningsmetode

Ikke-sandsynlighedsprøve

Studiebefolkning

Patients that are seen at dedicated Hemophilia treatment Centers

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Kohorter og interventioner

Gruppe / kohorte
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.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Longitudinal change in femoral neck bone mineral density (g/cm²)
Tidsramme: Baseline and annually through 5 years.
Bone mineral densitometry
Baseline and annually through 5 years.

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Longitudinal change in Lumbar spine (L1-L4) bone mineral density (g/cm²)
Tidsramme: Baseline and annually through 5 years.
Bone mineral densitometry
Baseline and annually through 5 years.
Longitudinal change in total hip bone mineral density (g/cm²)
Tidsramme: Baseline and annually through 5 years.
Bone mineral densitometry
Baseline and annually through 5 years.
Longitudinal Change in Bone Remodeling Biomarkers and Cytokines
Tidsramme: Baseline and annually through 5 years.
PINP, CTX-I, OPG, RANKL, IL-1β, IL-6, and TNF-α
Baseline and annually through 5 years.
Change in Thrombin Generation and Plasmin Generation Parameters
Tidsramme: Baseline and annually through 5 years
Simultaneous Thrombin and Plasmin Generation Assay
Baseline and annually through 5 years

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Arkansas Children's Hospital Research Institute

Samarbejdspartnere

Genzyme, a Sanofi Company

Efterforskere

  • Ledende efterforsker: Divyaswathi Citla Sridhar, MD, Arkansas Children's Reserach Institute

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. juli 2026

Primær færdiggørelse (Anslået)

1. august 2031

Studieafslutning (Anslået)

1. august 2032

Datoer for studieregistrering

Først indsendt

6. maj 2026

Først indsendt, der opfyldte QC-kriterier

6. maj 2026

Først opslået (Faktiske)

12. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

4. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

3. juni 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • 300130

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

IPD-planbeskrivelse

Individual participant data will not be shared with with other researchers , and each participating site has access to only their centers data; however , aggregated data will be included in future publications and will be made available toparticipating centers following study completion

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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