HaemophiliaNCT03914209
Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Patients With Hemophilia
An observational study of Haemophilia, run by Investigación en Hemofilia y Fisioterapia at 1 site in Spain.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Haemophilia
- What is being tested?
- EHL clotting factor
- How many people
- 40 participants
- Where
- 1 siteSpain
- Study dates
- 2 Jun 2020to 15 Oct 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- Men only
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- Patients diagnosed with hemophilia A
- Patients over 18 years
- Patients under prophylactic treatment with rFVIIIFc concentrates.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Spain1 site, 1 recruiting
- Recruiting
Universidad Europea de Madrid
Madrid, Comunity of Madrid, 28670
Rubén Cuesta-Barriuso, PhD, +34607547274, ruben.cuestab@gmail.com
About this study
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
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
- Being tested
- EHL clotting factor
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EHL clotting factorOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: EHL clotting factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Changes in the dosage of factor VIII with EHL products (rFVIIIFc) during the study period will be included in all assessments. Dosing data (UI) and weekly administration frequency (days / week) will be collected.
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline joint pain after one year of follow-up
Visual analog scale. This scale will be used to evaluate the perception of joint pain, where joint pain ranges from 0 to 10 points (no pain to maximum perceived pain).
Measured: 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
Pressure algometer. Measuring instrument used to evaluate pressure joint pain. The instrument shall consist of a measuring device attached to a hard rubber tip 1.1 cm in diameter. Its sphere shall be calibrated in kg/cm2 with divisions of 0.1 kg/cm2. Before taking the measurements, patients will be instructed to say "yes" as soon as pressure exerted by the algometer becomes slightly unpleasant. The pressure algometer shall be kept perpendicular to the surface of the skin with the right hand, stabilizing the axis of the instrument using the thumb and index finger of the left hand. The pressure shall be increased at a uniform rate of approximately 1 kg/cm2 per second. As soon as the subject, according to the instructions, says "yes", the pressure will be stopped and the score will be noted.
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline kinesiophobia after one year of follow-up
Tampa Scale of Kinesiophobia (TSK-11SV). This questionnaire will be used to assess the fear of movement in study patients. It consists of 11 items and the values range from 1 to 4 points (the higher the score, the greater the fear of movement).
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline joint status after one year of follow-up
Hemophilia Joint Health Score (HJHS). This measuring instrument will be used to evaluate the joint condition of knees, ankles, and elbows. It includes 8 items (inflammation and its duration, pain, atrophy and muscle strength, crepitus, and reduced flexion and extension) ranging from 0 to 20 points per joint (the higher the score, the greater the joint deterioration).
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline lower limb functionality after one year of follow-up
6-Minute Walking test (6-MWT). The 6-MWT test is a performance-based test that measures walking speed and the submaximal capacity to perform an exercise. Patients are instructed to walk along a 30 to 50-meter track for 6 minutes attempting to cover as much distance as possible without running. The distance traveled in 6 minutes is recorded, allowing the use of a walking aid or orthopedic devices.
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline muscle strength after one year of follow-up
Dynamometry. The maximum isometric strength of the flexor and extensor muscles of the main joints affected in hemophilia (knees, ankles, and elbows) will be evaluated on both limbs with a manual dynamometer using a mark test. For this test, the rater needs to keep the dynamometer stationary while the patient exerts maximum force against it.
Measured: Screening visit, at six months and after one year of follow-up
Change from baseline adherence to prophylactic treatment after one year of follow-up
Veritas-Pro. Specific adhesion questionnaire for hemophilia patients receiving prophylactic treatment. The Validated Hemophilia Regimen Treatment Adherence Scale - Prophylaxis (Veritas-Pro) questionnaire was developed in the United States and has already been validated in Spanish. The Veritas-Pro scale consists of 24 Likert-type items from 1 to 5, grouped in five dimensions: Time, Dose, Plan, Remembering, Skip and Communicate. The minimum adherence score is 24 points and the maximum adherence score is 120, and in each sub-scale the score ranges from 4 and 20 points.
Measured: Screening visit, at six months and after one year of follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2019
- Study start2 Jun 2020
- Primary completion21 Sep 2021 (estimated)
- Study completion15 Oct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HaemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Investigación en Hemofilia y Fisioterapia
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Rubén Cuesta-Barriuso, PhD, Principal investigator, Universidad Europea de Madrid
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03914209
- Sponsor's protocol ID
- LongHEst
- Status last verified
- Sep 2021
- First submitted
- 11 Apr 2019
- Last updated
- 2 Sep 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03914209
- Acronym / sponsor ID
- LongHEst
- Conditions
- Haemophilia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Investigación en Hemofilia y FisioterapiaResearch network
- Study start
- 2 Jun 2020 (actual)
- Primary completion
- 21 Sep 2021 (estimated)
- Study completion
- 15 Oct 2021 (estimated)
- First posted
- 16 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EHL clotting factor | – | Other: EHL clotting factorThis 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- Patients diagnosed with hemophilia A
- Patients over 18 years
- Patients under prophylactic treatment with rFVIIIFc concentrates.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EHL clotting factorThis 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 |
Primary outcome
Change from baseline bleeding frequency after one year of follow-up
Time frame: Screening visit, at six months and 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
8 secondary outcomes
Change from baseline dosage of factor VIII with EHL products after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline joint pain after one year of follow-up
Time frame: 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
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline kinesiophobia after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline joint status after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline lower limb functionality after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline muscle strength after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Change from baseline adherence to prophylactic treatment after one year of follow-up
Time frame: Screening visit, at six months and after one year of follow-up
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rubén Cuesta-Barriuso, PhD principal investigator
Universidad Europea de Madrid
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia, run by Investigación en Hemofilia y Fisioterapia at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rubén Cuesta-Barriuso, PhD (+34 607547274, ruben.cuestab@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline bleeding frequency after one year of follow-up (time frame: Screening visit, at six months and after one year of follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2021.