Intensive Replacement Treatment in Haemophilia Patients With Synovitis

Brief summary (registry, in English)

Background: Joint haemorrhage represents the most common type of bleeding episode in persons with hemophilia (PwH). In the absence of an adequate prophylaxis with Factor VIII (for hemophilia A) or FIX (for hemophilia B) concentrates up to 85% of patients with severe hemophilia develop a clinically overt joint disease. Screening of early signs of arthropathy is needed. Synovitis is widely considered as one of the parameters to be taken into account for the diagnosis and the surveillance of joint impairment in PwH. Aim: To assess if an intensive factor VIII replacement treatment is able at reverting synovitis in PwH. Methods: The present study is a randomized, open-label, cross-over study. Among patients referred to enrolling Haemophilia Centres, consecutive patients with severe (FVIII \< 1%) or severe-moderate (FVIII \< 2%) haemophilia A without inhibitors will be enrolled. The present study will be organized in 2 phases. * Phase 1 (US screening): All patients will undergo an ultrasound examination of elbows, ankles and knees to define joint status and to identify presence/absence of synovitis according to the HEAD-US system. * Phase 2 (Intervention): Patients with US evidence of synovitis will be randomly assigned at undergoing a PK assessment with my-PK-fit to start a prophylaxis with Adynovi® targeting a 12% FVIII through level (PROPEL-like arm) or to continue ongoing standard treatment (control arm). US examination of the six joints will be repeated monthly for six months and in case of onset of symptoms that might suggest an acute bleeding episode. After six months the two treatment arm will be switched in the frame of a cross-over approach and all PwH will be followed for other 6 months The primary outcome will be represented by changes in synovial status during the intensive factor VIII replacement treatment vs standard treatment.

What you commit to

• Study type: Interventional · Phase 4
• Participants enrolled: 64
• From 1 Mar 2022 to the primary completion date, 1 Mar 2023
• 1 site in 1 country
• Visits and tests: ask the trial team, the registry does not list them

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Who cannot take part

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Who the study is looking for

Age in the registry: 12 years to 70 years · Sex: Male · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

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