Haemophilia and Bone Loss - PHILEOS Study

Launched by CENTRE HOSPITALIER UNIVERSITAIRE DE SAINT ETIENNE · May 11, 2020

Keywords

ClinConnect Summary

The PHILEOS Study is a clinical trial focused on understanding bone loss in patients with hemophilia, a rare bleeding disorder. Researchers want to find out how common osteoporosis (a condition where bones become weak and fragile) is among people with hemophilia A and B, especially considering factors like disease severity and treatment. They will also compare these findings to healthy individuals to see how bone health differs. The study aims to explore whether the bleeding factors that hemophilia patients are missing might be directly linked to bone loss.

To participate, men aged 20 to 60 who either have hemophilia (regardless of its severity or treatment status) or are healthy volunteers in the same age range can apply. Participants will need to meet certain health criteria, and those with specific medical conditions or treatments that could affect bone health may not be eligible. If you join the study, you can expect to undergo tests to assess your bone health and help researchers learn more about the relationship between hemophilia and osteoporosis, ultimately aiming to improve care for patients like you.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• * 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
• Exclusion Criteria:
• * 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
• * 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