HEMOS: Bone Microarchitecture in Men With Hemophilia

Sponsor
Hospices Civils de Lyon (Other)
Overall Status
Recruiting
CT.gov ID
NCT05127681
Collaborator
(none)
30
1
2
27
1.1

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Radiation: HR-pQCT
  • Biological: Blood sample
  • Radiation: Dual energy X-ray absorptiometry
  • Other: medical data collection
N/A

Study Design

Study Type:
Interventional
Anticipated Enrollment :
30 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Diagnostic
Official Title:
Bone Microarchitecture in Men With Hemophilia
Anticipated Study Start Date :
Mar 1, 2022
Anticipated Primary Completion Date :
Jun 1, 2024
Anticipated Study Completion Date :
Jun 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: severe hemophilia A or B patients

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

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

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

Other: healthy men

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
Data of healthy men is already available

Outcome Measures

Primary Outcome Measures

  1. number of trabecular at distal tibia [3 months following the inclusion]

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

  2. number of trabecular at distal radius [3 months following the inclusion]

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

Secondary Outcome Measures

  1. Sera bone remodeling biomarkers [3 months following the inclusion]

    This biomarkers will be performed: PINP (ng/ml) CTX-1 (ng/ml) Periostin (ng/ml)

  2. reflection of bone strength [3 months following the inclusion]

    Micro-Finite Element Analysis (µFEA) reflection of bone strength will be performed and compared to the values available for the healthy control group

  3. Trabecular Bone Score (TBS) [3 months following the inclusion]

    Trabecular Bone Score (TBS) will be performed and compared to the values available for the healthy control group The trabecular bone score is a measure of bone texture correlated with bone microarchitecture and a marker for the risk of osteoporosis

Eligibility Criteria

Criteria

Ages Eligible for Study:
20 Years to 60 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
Inclusion Criteria:
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
Exclusion Criteria:
  • 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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Hôpital Cardio-Vasculaire et Pneumologique Bron France 69677

Sponsors and Collaborators

  • Hospices Civils de Lyon

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Hospices Civils de Lyon
ClinicalTrials.gov Identifier:
NCT05127681
Other Study ID Numbers:
  • 69HCL19_0390
  • 2019-A03281-56
First Posted:
Nov 19, 2021
Last Update Posted:
Mar 9, 2022
Last Verified:
Feb 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Hospices Civils de Lyon
Additional relevant MeSH terms:

Study Results

No Results Posted as of Mar 9, 2022