Clinical trial NCT03914209: 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
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Haemophilia
Lead sponsor: Investigación en Hemofilia y Fisioterapia
Sponsor class: NETWORK
First posted: 2019-04-16
Enrollment: 40
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The sample size is justified in relation to the prevalence of hemophilia A patients in Spain following prophylactic treatment based on EHL products. Based on the established selection criteria (n = 517, according to the census of the Spanish Federation of Hemophilia), the sample size at the national level would be 146, with a confidence or safety level of 95% and an expected dropout rate of 15%.

Currently in Spain, treatment with EHL products (rFVIIIFc) in patients with hemophilia A is being implemented and generalized. Thus, for the 8 Spanish regions where the study will be carried out, a sample of 40 patients is established for the work schedule set.

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.


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

Primary outcomes:
measure: Change from baseline bleeding frequency after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
Secondary outcomes:
measure: Change from baseline dosage of factor VIII with EHL products after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline joint pain after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline joint pain after one year of follow-up: Pressure algometer
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline kinesiophobia after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline joint status after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline lower limb functionality after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline muscle strength after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
measure: Change from baseline adherence to prophylactic treatment after one year of follow-up
timeFrame: Screening visit, at six months and after one year of follow-up
Other outcomes:
Locations:
city: Madrid
country: Spain
facility: Universidad Europea de Madrid
state: Comunity Of Madrid
Study officials:
affiliation: Universidad Europea de Madrid
name: Rubén Cuesta-Barriuso, PhD
role: PRINCIPAL_INVESTIGATOR
References: