Clinical trial NCT05127681: Bone Microarchitecture in Men With Hemophilia
Official title: Bone Microarchitecture in Men With Hemophilia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Severe Hemophilia A
Osteoporosis
Keywords: Hemophilia
osteoporosis
bone microarchitecture
bone remodeling
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2021-11-19
Enrollment: 10
Sex: MALE
Minimum age (years): 20
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: DIAGNOSTIC

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.


Eligibility criteria: 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.

Primary outcomes:
measure: number of trabecular at distal tibia
timeFrame: 3 months following the inclusion
measure: number of trabecular at distal radius
timeFrame: 3 months following the inclusion
Secondary outcomes:
measure: Sera bone remodeling biomarkers
timeFrame: 3 months following the inclusion
measure: reflection of bone strength
timeFrame: 3 months following the inclusion
measure: Trabecular Bone Score (TBS)
timeFrame: 3 months following the inclusion
Other outcomes:
Locations:
city: Bron
country: France
facility: Hôpital Cardio-Vasculaire et Pneumologique
Study officials:
References: