Bone Microarchitecture in Men With Hemophilia

NCT05127681 | Terminated

• 2 other identifiers: 69HCL19_03902019-A03281-56
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Hemophilia A and B are hereditary sex-linked deficiencies of coagulation factors VIII and IX characterized by bleeding. Their modern therapy increases life expectancy and risk of age-related diseases, e.g., osteoporosis. Hemophilia-specific risk factors impair formation of peak bone mass and accelerate bone loss. Fractures are more frequent in hemophilic men vs. age-matched men and induce bleeding which is aggravated by manipulations and surgical intervention. The hypothesis of this study is that hemophilic men have poor bone microarchitecture (assessed by High-resolution peripheral quantitative computed tomography (HR-pQCT)) related to an imbalance between bone formation and resorption (assessed by bone turnover markers (BTM) and bone biomarkers). The study aims to assess the difference in low trabecular number (Tb.N) at the distal radius between hemophilic men (cases) and age- height-weight-ethnicity and smoking-matched healthy men (controls). Correlation between BTM and Tb.N will be also studied. Biologic markers of bone remodeling (C-terminal telopeptide of type I collagen (PINP), N-terminal propeptide of type I procollagen (CTX-I), periostin) will be studied.

Conditions

Severe Hemophilia A; Osteoporosis

Keywords

Hemophilia; osteoporosis; bone microarchitecture; bone remodeling

Outcome Measures

Primary Outcomes (2)

• number of trabecular at distal tibia

  The number of trabecular at distal tibia will be performed and compared to the values available for the healthy control group

  3 months following the inclusion

• number of trabecular at distal radius

  The number of trabecular at distal radius will be performed and compared to the values available for the healthy control group

  3 months following the inclusion

Secondary Outcomes (3)

• Sera bone remodeling biomarkers

  3 months following the inclusion

• reflection of bone strength

  3 months following the inclusion

• Trabecular Bone Score (TBS)

  3 months following the inclusion

Study Arms (2)

severe hemophilia A or B patients

EXPERIMENTAL

30 patients with severe hemophilia A or B (Factor (F)VIII or Factor IX (FIX)≤ 1%) aged 20 to 60 years will be included in this study.

Radiation: HR-pQCT; Biological: Blood sample; Radiation: Dual energy X-ray absorptiometry

healthy men

OTHER

Data of healthy men, matching in age- height-weight-ethnicity and smoking-matched with patient will be collected. These data are already available at the "Institut national de la santé et de la recherche médicale" (INSERM) research unit associated.

Other: medical data collection

Interventions

HR-pQCT RADIATION

Patients will have a unique HR-pQCT scanner imaging to study their bone microarchitecture

severe hemophilia A or B patients

Blood sample BIOLOGICAL

Patients will have a unique blood sampling of 10 mL for the measurements of sera PINP, CTX-I, periostin levels

severe hemophilia A or B patients

Dual energy X-ray absorptiometry RADIATION

A dual energy X-ray absorptiometry scan of lumbar spine, hip, distal radius and whole body, body composition, lateral spine will be performed.

severe hemophilia A or B patients

medical data collection OTHER

Data of healthy men is already available

healthy men

Eligibility Criteria

• Age: 20 Years - 60 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Patients:
• Men aged 20 to 60 years
• Severe hemophilia A or B (FVIII or FIX\<1%)
• Regular followed up in Lyon Hemophilia Center
• Ability to give free and informed consent
• Person capable of actively participating in radiological examinations
• Healthy Mens:
• Aged 20 to 60 years

You may not qualify if:

• Women
• Not covered by health system
• Vulnerable (adults unable to consent, protected under guardianship, prisoner)
• Any blood coagulation abnormality other than severe hemophilia A or B
• Having one of the following treatments against osteoporosis of more than 6 months : bisphosphonates, denosumab, teriparatide
• With a chronical disease having a high impact on bone structure and no related to hemophilia disease, such as Cushing or Crohn diseases.

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (1)

Hôpital Cardio-Vasculaire et Pneumologique

Bron, 69677, France

Location

MeSH Terms

Conditions

Hemophilia A; Osteoporosis

Interventions

Blood Specimen Collection; Absorptiometry, Photon

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; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques; Radiography; Diagnostic Imaging; Densitometry; Photometry; Chemistry Techniques, Analytical

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2021
• First Posted: November 19, 2021
• Study Start: August 1, 2023
• Primary Completion: February 6, 2024
• Study Completion: February 6, 2024
• Last Updated: April 18, 2024

Record last verified: 2024-04

Locations

FR (1)