NCT03914209

Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Patients With Hemophilia

Efficacy of a Prophylactic Treatment Using Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Adult Patients With Hemophilia. Observational Multicenter Study

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 40 (estimated)
• Sponsor: Investigación en Hemofilia y Fisioterapia · Network
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Accepted

Summary

Background. Hemophiliac arthropathy is the main long-term physical damage in patients with hemophilia, causing disability and functional limitations. Objective. Assessment of the efficacy of a prophylactic treatment with EHL products for improving the musculoskeletal health of adult patients with hemophilia. Study Design. Multicenter observational clinical study. Method. 40 patients with hemophilia A will be included in this study. Patients will be recruited from 5 centers located in various regions of Spain (Community of Madrid, Galicia, Community of Valenciana, Málaga and Vizcaya). The dependent variables will be: bleeding frequency (self-report of bleeding), changes in the dosage of factor VIII with EHL products (rFVIIIFc), pain (measured with the visual analog scale and a pressure Algometer), degree of kinesiophobia (Tampa Scale of Kinesiophobia), degree of adherence to treatment (Veritas-Pro scale), joint health (using the Hemophilia Joint Health Score), muscle strength (using a dynamometer) and functionality (using the 6-Minute Walking Test). Three evaluations will be carried out: baseline, at 6 months, and at the end of the study period, at 12 months. The evolution of quantitative variables shall be analyzed by parametric tests (t-student test) or non-parametric tests (Wilcoxon test). Pearson's correlation coefficient shall be used to obtain the correlation between the dependent and independent variables. By means of a linear regression analysis we will record the percentage influence of the clinical variables relative to the dependent variables measured in this study Expected results. The aim is to establish the effectiveness of the prophylactic treatment in the improvement of the state health, joint pain, muscle strength and functionality in patients with hemophilia.

Conditions

• Haemophilia

Interventions

Type	Name	Description
OTHER	EHL clotting factor	This study shall not implement any intervention that might alter the normal development of the daily life activities of hemophilia patients included in the study. They will continue with the prophylactic regimen prescribed by their hematologist. Patients will also be asked to continue to develop their physical, work, entertainment and leisure activities in the same way as at baseline.

Timeline

• Start date: 2020-06-02
• Primary completion: 2021-09-21
• Completion: 2021-10-15
• First posted: 2019-04-16
• Last updated: 2021-09-02

Locations

1 site across 1 country: Spain