HemophiliaPHILEOSNCT04384341
Haemophilia and Bone Loss - PHILEOS Study
A study testing Bone densitometry (BMD) and Blood sampling for patients only in people with Hemophilia, run by Centre Hospitalier Universitaire de Saint Etienne at 23 sites in 5 countries.
Trial summary
Haemophilia is a rare bleeding disorder, characterized by factor VIII (HA) or factor IX (HB) deficiency. The absence or the reduction of fVIII or fIX result in impaired thrombin generation and clot formation, causing excessive bleeding (mainly haemarthrosis). Read more about this study
- Who is it for?
- Ages 20 to 60 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Hemophilia
- What is being tested?
- Bone densitometry (BMD) and Blood sampling for patients only
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 448 participants
- Where
- 23 sitesin 5 countries
- Study dates
- 2 Jun 2020to 24 Dec 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
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy Volunteers :
- Healthy men aged between 20 to 60 years old
- Haemophilic Patients:
- Haemophilia A and B patients, irrespective of the disease form (mild, moderate, severe with or without prophylaxis)
- Haemophilic patients aged between 20 to 60 years old
- Severe Haemophilia A patients with prophylaxis : last factor VIII injection more than 48 to 120 hours (depending on on the prophylactic treatment) prior blood sampling dedicated to the this research
- Severe Haemophilia B patients : last factor IX injection more than 5 to 21 days (depending on the prophylactic treatment) prior blood sampling dedicated to the this research
Who cannot join
- Healthy Volunteers:
- History of disease known to influence bone metabolism (hyperthyroidism, hyperparathyroidism, hypercorticism, hypogonadism, diseases that require long-term use of corticoids, …)
- Past or present treatment with any osteoporotic medication other than Vit D or Ca++
- Presence of two total hip prostheses
- HIV documented infection
- HCV documented infection (in progress or cured) at cirrhotic stage
Show all 14Show fewer
- Haemophilic Patients:
- Haemophilic patients with current or history of inhibitor anti-fVIII or anti-fIX (>5 Bethesda Units)
- Treatment with HEMLIBRA (Emicizumab). Unless it is possible to use a result of thrombin generation prior to this treatment and achieved with a residual rate not greater than or equal to 5%.
- History of disease known to influence bone metabolism and not related to haemophilia (hyperthyroidism, hyperparathyroidism, hypercorticism, hypogonadism, diseases that require long-term use of corticoids, …)
- Past or present treatment with any anti-osteoporotic medication other than Vit D or Ca++
- Presence of two total hip prostheses
- HIV documented infection
- HCV documented infection (in progress or cured) at cirrhotic stage
Trial locations and contacts
23 sites in 5 countries.
23 sites in 5 countries
France19 sites
Chu de Bordeaux
Bordeaux, 33076
Chu Brest Hopital Morvan
Brest, 29200
HCL - Groupement Hospitalier Est (Hôpital Louis Pradel)
Bron
CHU Caen
Caen
Centre Hospitalier Metropole Savoie
Chambéry, 73000
Chu Cth Estaing Clermont Ferrand
Clermont-Ferrand
Chu de Dijon
Dijon, 21000
Chu Grenoble Alpes
Grenoble, 38700
CHU Lille
Lille
Chu La Timone Marseille
Marseille, 13010
CHU - Saint Eloi
Montpellier, 34295
CHU Nancy
Nancy
CHU de Nantes
Nantes
Chu Necker Paris
Paris, 75015
APHP - Bicêtre
Paris
Chu Rennes Hopital Pontchaillou
Rennes, 35000
CHU de ROUEN
Rouen
CHU de Saint-Etienne
Saint-Etienne, 42055
Chu Strasbourg - Hôpital de Hautepierre
Strasbourg
About this study
Official title: Haemophilia and Bone Loss PHILEOS Study
Haemophilia is a rare bleeding disorder, characterized by factor VIII (HA) or factor IX (HB) deficiency. The absence or the reduction of fVIII or fIX result in impaired thrombin generation and clot formation, causing excessive bleeding (mainly haemarthrosis). Osteoporosis is a systemic bone disease characterized by a low bone mineral density (BMD). A decrease of mean BMD has been described in haemophilic patients compared to healthy controls in several studies. So, osteoporosis could be an underestimated haemophilia-related comorbidity. None of the following risk factors (reduced physical activity, joint damage, vitamin D deficiency and /or hepatitis C virus (HCV) infection) has been retained as a cause of osteoporosis in haemophilic patients. Another hypothesis is that bone loss could be directly linked to fVIII or fIX and/or thrombin deficiency. The aim of this study is to evaluate the prevalence of the bone loss in HA and B patients, according to the type, the severity and the presence (or not) of a prophylactic treatment (depending on the age at which it was began) and to compare it to a control population. The investigators will also evaluate the relation between BMD and FVIII, fIX and thrombin potential.
Read the detailed description
Recruitment of healthy volunteers through registers (Clinical Investigation Centers) and advertisements.
Recruitment of haemophilic patients during a routine visit at the haemophilia centre.
Information of the subjects that the study requires a BMD measure for all and a blood sampling for patients only.
After inclusion and exclusion criteria have been checked, the subject can sign the consent.
For all subjects, an appointment will be made for BMD measure. For patients and controls: BMD will be measured by Dual Energy X-ray Absorptiometry (DXA) technology, on femoral and lumbar spine (L2-L4) sites.
For patients, fVIII/fIX activity and antigen, thrombin generation potential and plasmatic markers of bone remodelling will be measured centrally.
- Conditions
- Hemophilia
- Being tested
- Bone densitometry (BMD), Blood sampling for patients only
- Keywords
- Osteoporosis, fVIII, fIX, thrombin, Haemophilia, Hemophilia
- 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.
Bone densitometry (BMD)Radiation
Recruitment of haemophilic patients during a routine visit at the haemophilia centre.
Information of the subjects that the study requires a BMD measure for all and a blood sampling for patients only.
After inclusion and exclusion criteria have been checked, the subject can sign the consent.
For all subjects, an appointment will be made for BMD measure.
For patients and controls: BMD will be measured by Dual Energy X-ray Absorptiometry (DXA) technology, on femoral and lumbar spine (L2-L4) sites.
Recruitment of healthy volunteers through registers (Clinical Investigation Centers) and advertisements.
Blood sampling for patients onlyBiological
For patients, fVIII/fIX activity and antigen, thrombin generation potential and plasmatic markers of bone remodelling will be measured centrally.
| Study group | Role | What they receive |
|---|---|---|
| Haemophilic patients | Experimental | Blood sampling Bone Densitometry (BMD) Receives: Radiation: Bone densitometry (BMD); Biological: Blood sampling for patients only |
| Healthy volunteers | Other | Bone Densitometry (BMD) Receives: Radiation: Bone densitometry (BMD) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Osteoporosis defined by a T-score < -2.5 in severe haemophilic patients without prophylaxis and in healthy subjects.
Bone mineral densitometry
Measured: During the procedure
Secondary outcomes (5)
Osteoporosis defined by a T-score < -2.5 in the different groups of haemophilic patients and in in healthy subjects.
Bone mineral densitometry
Measured: During the procedure
Osteopenia defined by a T-score < -1 in the different groups of haemophilic patients and in in healthy subjects.
Bone mineral densitometry
Measured: During the procedure
Bone mineral density (expressed as a T-score) in the different groups of haemophilic patients and in healthy subjects.
Bone mineral densitometry
Measured: During the procedure
Basal level of fVIII/fIX (expressed as an Ag level or as a %) or thrombin generation potential (expressed as an endogenous thrombin potential (ETP), nmol/min) and Bone mineral density (expressed as a T-score and Z-score)
Blood test Relation between the basal level of fVIII or fIX (expressed as an Ag level or as a %) or the thrombin generation potential (expressed as an endogenous thrombin potential (ETP), nmol/min) and Bone mineral density (expressed as a T-score and Z-score)
Measured: At the inclusion
Markers influencing bone metabolism in all haemophilic patients included
Blood test
Measured: At the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2020
- Study start2 Jun 2020
- Primary completion12 Dec 2024
- Study completion24 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- 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
- Screening
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 448 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Saint Etienne
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, France
- Responsible party
- Sponsor
- Investigators
- Brigitte TARDY, MD, Principal investigator, CHU de Saint Etienne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04384341
- Sponsor's protocol ID
- 19PH224
- Other IDs
- 2019-A03358-49
- Acronym
- PHILEOS
- Status last verified
- Jun 2026
- First submitted
- 5 May 2020
- Last updated
- 3 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Tardy-Poncet B, Play B, Montmartin A, Damien P, Ollier E, Presles E, Garcin A, Tardy B. PHILEOS (haemoPHILia and ostEoporOSis) Study: protocol of a multicentre prospective case-control study. BMJ Open. 2021 Jan 13;11(1):e042283. doi: 10.1136/bmjopen-2020-042283.
PubMed 33441362
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haemophilia and Bone Loss PHILEOS Study
- ClinicalTrials.gov ID
- NCT04384341
- Acronym / sponsor ID
- PHILEOS / 19PH224
- Conditions
- Hemophilia
- Keywords
- Osteoporosis, fVIII, fIX, thrombin, Haemophilia, Hemophilia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 448 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de Saint EtienneUniversity, hospital or other
- Collaborators
- Ministry of Health, France
- Study start
- 2 Jun 2020 (actual)
- Primary completion
- 12 Dec 2024 (actual)
- Study completion
- 24 Dec 2024 (actual)
- First posted
- 12 May 2020 (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.
Haemophilia is a rare bleeding disorder, characterized by factor VIII (HA) or factor IX (HB) deficiency. The absence or the reduction of fVIII or fIX result in impaired thrombin generation and clot formation, causing excessive bleeding (mainly haemarthrosis). Osteoporosis is a systemic bone disease characterized by a low bone mineral density (BMD). A decrease of mean BMD has been described in haemophilic patients compared to healthy controls in several studies. So, osteoporosis could be an underestimated haemophilia-related comorbidity. None of the following risk factors (reduced physical activity, joint damage, vitamin D deficiency and /or hepatitis C virus (HCV) infection) has been retained as a cause of osteoporosis in haemophilic patients. Another hypothesis is that bone loss could be directly linked to fVIII or fIX and/or thrombin deficiency. The aim of this study is to evaluate the prevalence of the bone loss in HA and B patients, according to the type, the severity and the presence (or not) of a prophylactic treatment (depending on the age at which it was began) and to compare it to a control population. The investigators will also evaluate the relation between BMD and FVIII, fIX and thrombin potential.
Detailed description
Recruitment of healthy volunteers through registers (Clinical Investigation Centers) and advertisements.
Recruitment of haemophilic patients during a routine visit at the haemophilia centre.
Information of the subjects that the study requires a BMD measure for all and a blood sampling for patients only.
After inclusion and exclusion criteria have been checked, the subject can sign the consent.
For all subjects, an appointment will be made for BMD measure. For patients and controls: BMD will be measured by Dual Energy X-ray Absorptiometry (DXA) technology, on femoral and lumbar spine (L2-L4) sites.
For patients, fVIII/fIX activity and antigen, thrombin generation potential and plasmatic markers of bone remodelling will be measured centrally.
Study design
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Haemophilic patients | Experimental | Radiation: Bone densitometry (BMD), Biological: Blood sampling for patients onlyBlood sampling Bone Densitometry (BMD) |
| Healthy volunteers | Other | Radiation: Bone densitometry (BMD)Bone Densitometry (BMD) |
Who may qualify
- Ages
- 20 to 60 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Healthy Volunteers :
- Haemophilic Patients:
Key exclusion criteria (14 listed)
- Healthy Volunteers:
- Haemophilic Patients:
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 |
|---|---|---|
| Bone densitometry (BMD)Recruitment of haemophilic patients during a routine visit at the haemophilia centre. Information of the subjects that the study requires a BMD measure for all and a blood sampling for patients only. After inclusion and exclusion criteria have been checked, the subject can sign the consent. For all subjects, an appointment will be made for BMD measure. For patients and controls: BMD will be measured by Dual Energy X-ray Absorptiometry (DXA) technology, on femoral and lumbar spine (L2-L4) sites. Recruitment of healthy volunteers through registers (Clinical Investigation Centers) and advertisements. | Radiation | Haemophilic patients, Healthy volunteers |
| Blood sampling for patients onlyFor patients, fVIII/fIX activity and antigen, thrombin generation potential and plasmatic markers of bone remodelling will be measured centrally. | Biological | Haemophilic patients |
Primary outcome
Osteoporosis defined by a T-score < -2.5 in severe haemophilic patients without prophylaxis and in healthy subjects.
Time frame: During the procedure
Bone mineral densitometry
5 secondary outcomes
Osteoporosis defined by a T-score < -2.5 in the different groups of haemophilic patients and in in healthy subjects.
Time frame: During the procedure
Osteopenia defined by a T-score < -1 in the different groups of haemophilic patients and in in healthy subjects.
Time frame: During the procedure
Bone mineral density (expressed as a T-score) in the different groups of haemophilic patients and in healthy subjects.
Time frame: During the procedure
Basal level of fVIII/fIX (expressed as an Ag level or as a %) or thrombin generation potential (expressed as an endogenous thrombin potential (ETP), nmol/min) and Bone mineral density (expressed as a T-score and Z-score)
Time frame: At the inclusion
Markers influencing bone metabolism in all haemophilic patients included
Time frame: At the inclusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Derived from this study: PubMed 33441362Tardy-Poncet B, Play B, Montmartin A, Damien P, Ollier E, Presles E, Garcin A, Tardy B. PHILEOS (haemoPHILia and ostEoporOSis) Study: protocol of a multicentre prospective case-control study. BMJ Open. 2021 Jan 13;11(1):e042283. doi: 10.1136/bmjopen-2020-042283.
- Individual participant dataSharing planned: No
Related trials for Hemophilia
Not yet recruitingPhase 1
Lentiviral FVIII Gene Therapy
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A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
Hemophilia A, Healthy Adult Participants
RecruitingPhase 3
A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
Haemophilia A
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A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
RecruitingObservational study
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Hemophilia A
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.
Brigitte TARDY, MD principal investigator
CHU de Saint Etienne
23 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Bone densitometry (BMD) and Blood sampling for patients only in people with Hemophilia, run by Centre Hospitalier Universitaire de Saint Etienne at 23 sites in 5 countries.
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 7 inclusion and 14 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: finished, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in France, Belgium, Croatia, Hungary, Romania. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Osteoporosis defined by a T-score < -2.5 in severe haemophilic patients without prophylaxis and in healthy subjects. (time frame: During the procedure). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04384341, which also lists 1 linked publication.
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 3 Jun 2026.