Brief Title
Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Patients With Hemophilia
Official Title
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
Brief 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.
Study Type
Observational
Primary Outcome
Change from baseline bleeding frequency after one year of follow-up
Secondary Outcome
Change from baseline dosage of factor VIII with EHL products after one year of follow-up
Condition
Haemophilia
Intervention
EHL clotting factor
Study Arms / Comparison Groups
EHL clotting factor
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
40
Start Date
February 2, 2020
Completion Date
December 15, 2020
Primary Completion Date
November 10, 2020
Eligibility Criteria
Inclusion Criteria: - Patients diagnosed with hemophilia A - Patients over 18 years - Patients under prophylactic treatment with rFVIIIFc concentrates. Exclusion Criteria: - Patients with neurological or cognitive disorders preventing the understanding of the various measuring instruments - Patients who are scheduled for major orthopedic surgery (total arthroplasty) - Not signed the Informed Consent Document
Gender
Male
Ages
18 Years - 65 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Contacts
Rubén Cuesta-Barriuso, PhD, +34 607547274, [email protected]
Location Countries
Spain
Location Countries
Spain
Administrative Informations
NCT ID
NCT03914209
Organization ID
LongHEst
Responsible Party
Sponsor
Study Sponsor
Investigación en Hemofilia y Fisioterapia
Study Sponsor
Rubén Cuesta-Barriuso, PhD, Principal Investigator, Universidad Europea de Madrid
Verification Date
January 2020