Bone Microarchitecture in Men With Hemophilia

NCT ID: NCT05127681

Last Updated: 2024-04-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-01

Study Completion Date

2024-02-06

Brief Summary

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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.

Detailed Description

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Conditions

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Severe Hemophilia A Osteoporosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

HR-pQCT

Intervention Type RADIATION

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

Blood sample

Intervention Type BIOLOGICAL

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

Dual energy X-ray absorptiometry

Intervention Type RADIATION

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

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.

Group Type OTHER

medical data collection

Intervention Type OTHER

Data of healthy men is already available

Interventions

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HR-pQCT

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

Intervention Type RADIATION

Blood sample

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

Intervention Type BIOLOGICAL

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.

Intervention Type RADIATION

medical data collection

Data of healthy men is already available

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

20 Years

Maximum Eligible Age

60 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hôpital Cardio-Vasculaire et Pneumologique

Bron, , France

Site Status

Countries

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France

Other Identifiers

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2019-A03281-56

Identifier Type: OTHER

Identifier Source: secondary_id

69HCL19_0390

Identifier Type: -

Identifier Source: org_study_id

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