Severe Hemophilia AHEMOSNCT05127681
Bone Microarchitecture in Men With Hemophilia
A study testing HR-pQCT, Blood sample and Dual energy X-ray absorptiometry in people with Severe Hemophilia A and Osteoporosis, run by Hospices Civils de Lyon at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 to 60 years, men only
- Conditions the study is for
- Severe Hemophilia A, Osteoporosis
- What is being tested?
- HR-pQCT, Blood sample and Dual energy X-ray absorptiometry
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteFrance
- Study dates
- 1 Aug 2023to 6 Feb 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 60 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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
- Being tested
- HR-pQCT, Blood sample, Dual energy X-ray absorptiometry, medical data collection
- Keywords
- Hemophilia, osteoporosis, bone microarchitecture, bone remodeling
- Medical subject headings
- Hemophilia A, Osteoporosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HR-pQCTRadiation
Patients will have a unique HR-pQCT scanner imaging to study their bone microarchitecture
Blood sampleBiological
Patients will have a unique blood sampling of 10 mL for the measurements of sera PINP, CTX-I, periostin levels
Dual energy X-ray absorptiometryRadiation
A dual energy X-ray absorptiometry scan of lumbar spine, hip, distal radius and whole body, body composition, lateral spine will be performed.
medical data collectionOther
Data of healthy men is already available
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: medical data collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 3 months following the inclusion
Secondary outcomes (3)
Sera bone remodeling biomarkers
This biomarkers will be performed:
- PINP (ng/ml)
- CTX-1 (ng/ml)
- Periostin (ng/ml)
Measured: 3 months following the inclusion
reflection of bone strength
Micro-Finite Element Analysis (µFEA) reflection of bone strength will be performed and compared to the values available for the healthy control group
Measured: 3 months following the inclusion
Trabecular Bone Score (TBS)
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
Measured: 3 months following the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2021
- Study start1 Aug 2023
- Primary completion6 Feb 2024
- Study completion6 Feb 2024
Why it stopped: More Hardware
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05127681
- Sponsor's protocol ID
- 69HCL19_0390
- Other IDs
- 2019-A03281-56
- Acronym
- HEMOS
- Status last verified
- Apr 2024
- First submitted
- 6 Oct 2021
- Last updated
- 18 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bone Microarchitecture in Men With Hemophilia
- ClinicalTrials.gov ID
- NCT05127681
- Acronym / sponsor ID
- HEMOS / 69HCL19_0390
- Conditions
- Severe Hemophilia A, Osteoporosis
- Keywords
- Hemophilia, osteoporosis, bone microarchitecture, bone remodeling
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: More Hardware
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Aug 2023 (actual)
- Primary completion
- 6 Feb 2024 (actual)
- Study completion
- 6 Feb 2024 (actual)
- First posted
- 19 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| severe hemophilia A or B patients | Experimental | Radiation: HR-pQCT, Biological: Blood sample, Radiation: Dual energy X-ray absorptiometry30 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. |
| healthy men | Other | Other: medical data collectionData 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. |
Who may qualify
- Ages
- 20 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HR-pQCTPatients will have a unique HR-pQCT scanner imaging to study their bone microarchitecture | Radiation | severe hemophilia A or B patients |
| Blood samplePatients will have a unique blood sampling of 10 mL for the measurements of sera PINP, CTX-I, periostin levels | Biological | severe hemophilia A or B patients |
| Dual energy X-ray absorptiometryA dual energy X-ray absorptiometry scan of lumbar spine, hip, distal radius and whole body, body composition, lateral spine will be performed. | Radiation | severe hemophilia A or B patients |
| medical data collectionData of healthy men is already available | Other | healthy men |
Primary outcome
number of trabecular at distal tibia
Time frame: 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
number of trabecular at distal radius
Time frame: 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
3 secondary outcomes
Sera bone remodeling biomarkers
Time frame: 3 months following the inclusion
reflection of bone strength
Time frame: 3 months following the inclusion
Trabecular Bone Score (TBS)
Time frame: 3 months following the inclusion
Results and safety
The study was stopped early (reason given: More Hardware). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2024.
Related trials for Severe Hemophilia A
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
Active, not recruitingPhase 4
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Severe Hemophilia A, Moderate Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruitingObservational study
Post Approval Phase 4 Study to Learn More About How Safe Octocog Alfa is, and How Well it Works in Patients With Severe Hemophilia A in India
Severe Hemophilia A
RecruitingPhase 1/2
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Hemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing HR-pQCT, Blood sample and Dual energy X-ray absorptiometry in people with Severe Hemophilia A and Osteoporosis, run by Hospices Civils de Lyon at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: More Hardware). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
number of trabecular at distal tibia (time frame: 3 months following the inclusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2024.