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

NCT03914209 | Unknown

• 1 other identifier: LongHEst
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Haemophilia

Outcome Measures

Primary Outcomes (1)

• Change from baseline bleeding frequency after one year of follow-up

  At baseline, patients will receive a personal self-report of bleeding (hematomas, hemarthrosis and mucous hemorrhagic episodes) where they should indicate the frequency of bleeding during the study period. This record should be delivered to the evaluators in the intermediate assessment and the final evaluation. In this way, changes in the frequency of bleeding throughout the study period will be evaluated

  Screening visit, at six months and after one year of follow-up

Secondary Outcomes (8)

• Change from baseline dosage of factor VIII with EHL products after one year of follow-up

  Screening visit, at six months and after one year of follow-up

• Change from baseline joint pain after one year of follow-up

  Screening visit, at six months and after one year of follow-up

• Change from baseline joint pain after one year of follow-up: Pressure algometer

  Screening visit, at six months and after one year of follow-up

• Change from baseline kinesiophobia after one year of follow-up

  Screening visit, at six months and after one year of follow-up

• Change from baseline joint status after one year of follow-up

  Screening visit, at six months and after one year of follow-up

Study Arms (1)

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.

Other: EHL clotting factor

Interventions

EHL clotting factor OTHER

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.

EHL clotting factor

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Patients diagnosed with hemophilia A
• Patients over 18 years
• Patients under prophylactic treatment with rFVIIIFc concentrates.

You may not qualify if:

• 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

Sponsors & Collaborators

• Investigación en Hemofilia y Fisioterapia: lead

Study Sites (1)

Universidad Europea de Madrid

Madrid, Comunity of Madrid, 28670, Spain

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rubén Cuesta-Barriuso, PhD

  Universidad Europea de Madrid

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rubén Cuesta-Barriuso, PhD

CONTACT

+34 607547274; ruben.cuestab@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2019
• First Posted: April 16, 2019
• Study Start: June 2, 2020
• Primary Completion: September 21, 2021
• Study Completion: October 15, 2021
• Last Updated: September 2, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)