Clinical trial NCT06565481: Measurement Properties in People with Hemophilia
Official title: Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Hemophilia a
Hemophilia B
Musculoskeletal Complication
Measurement Error
Lead sponsor: University of Zurich
Sponsor class: OTHER
Collaborators: name: ETH Zurich
First posted: 2024-08-22
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This study, including two time points, is performed to determine validity and reliability of performance based assessments using a one-arm sample.

A test retest design is used to determine reliability and a cross-sectional design to determine validity.

For each participant, the measures will be performed on two time points with a minimal recovery break of 2 days in one single centre in the Canton of Zurich (University Hospital Zurich).
Primary purpose: OTHER

Brief summary: The aim of this project is to determine the measurement properties (namely reliability and validity) of a set of clinical assessments, measuring different aspects of physical function.

Detailed description: The medical treatment of hemophilia has advanced greatly in recent years. There are now medications that reduce the physical symptoms of hemophilia. Nevertheless, those affected by hemophilia suffer from restricted mobility, reduced strength, reduced balance and pain as a result of the repeated bleeding. In order to make well-founded decisions regarding possible supportive therapeutic or medical measures, it is important to be able to estimate the extent of such limitations and whether these will change or remain stable.

The study is needed to validate the measurement properties of measurement instruments used to measure the effects of physical training interventions in participants with hemophilia. Clinical questions can only be solved if measuring instruments with sufficient measuring properties are used. These measurement properties must be evaluated in a relevant population. Otherwise, health professionals cannot assess whether measures or treatment are not effective or whether measured changes are not large enough to detect an actual change. Therefore, research is required to ensure that adequately studied measurement instruments are available for clinical assessment of those affected by adult participants with hemophilia.

Some of the tests (such as the Unipedal Stance Test or Four Square Step Test) have already been studied in children with hemophilia or in participants with other diseases. Since hemophilia is a disease with specific effects, the measuring instruments must also be researched in this participant group. There is still a research gap that the investigators would like to close with this study. For the study, the investigators selected tests that are already being used on adult participants with hemophilia and have had good experiences in practice. The tests are safe and can be carried out with little strain.

Eligibility criteria: Inclusion Criteria:

* Diagnosed Hemophilia Types A or B
* Adult people ≥ 18 years
* Patients on prophylactic factor or non-factor replacement treatment, or on demand factor replacement treatment.
* Able to walk for 20 meters independently, with or without walking aid
* Signed informed consent to participate in the study

Exclusion Criteria:

* Severe health conditions like severe cardiovascular, pulmonary, neurological or musculoskeletal diseases.
* Unable to perform the assessments due to disability or language problems.

Primary outcomes:
measure: Short Physical Performance Battery (SPPB)
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
measure: Four Square Step Test (FSST)
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
measure: Timed up and Go (TUG)
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
measure: Unipedal Stance Test (UST)
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
measure: 30-Second Stair Stand Test
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
measure: 1 kg Arm Lift Test
timeFrame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
Secondary outcomes:
measure: Short Form 36
timeFrame: Baseline (Day 1)
measure: Hemophilia Activities List
timeFrame: Baseline (Day 1)
measure: Hemophilia Joint Health Score Version 2.1
timeFrame: Baseline (Day 1)
Other outcomes:
Locations:
city: Zürich
country: Switzerland
facility: Ruud Knols
Study officials:
affiliation: University of Zurich
name: Ruud Knols, PhD
role: PRINCIPAL_INVESTIGATOR
References: