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Intensive Replacement Treatment in Haemophilia Patients With Synovitis

NCT04784988 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 64

Last updated 2024-03-27

No results posted yet for this study

Summary

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.

Conditions

Interventions

DRUG

EHL-FVIII

prophylaxis with EHL-FVIII targeting a 12% FVIII through level based on PK assessment with WAPPS-Hemo

DRUG

FVIII concentrate

standard prophylaxis with FVIII concentrate according to current guidelines

Sponsors & Collaborators

  • Federico II University

    lead OTHER

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
12 Years
Max Age
70 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-01
Primary Completion
2023-03-01
Completion
2024-03-01

Countries

  • Italy

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04784988 on ClinicalTrials.gov