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Clinical Trials/NCT06565481
NCT06565481
Recruiting
Not Applicable

Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia

University of Zurich1 site in 1 country50 target enrollmentOctober 31, 2024

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Hemophilia a
Sponsor
University of Zurich
Enrollment
50
Locations
1
Primary Endpoint
Timed up and Go (TUG)
Status
Recruiting
Last Updated
last year

Overview

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.

Registry
clinicaltrials.gov
Start Date
October 31, 2024
End Date
May 31, 2026
Last Updated
last year
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Responsible Party
Sponsor

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.

Outcomes

Primary Outcomes

Timed up and Go (TUG)

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

The TUG is a performance-based outcome measure that assesses lower extremity function, mobility, and fall risk. In this test, the participant starts in a seated position, with his back against a standard height chair, without armrest. The participant gets up and walks in a comfortable pace for 10 feet (three meters), turns around and returns to a seated position in the chair. The time to perform the task is measured in seconds. Shorter time indicates better mobility.

30-Second Stair Stand Test

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

The 30-Second Chair Stand Test measures the muscle function of the lower extremity by counting how many times a patient rises from a seated position to a full stand at his/her own pace

1 kg Arm Lift Test

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

The 1 kg arm lift test measures the muscle function of the upper extremity. It counts how many time a patient can lift a 1 kilogram weight above their head within 30 seconds at his/her own pace

Short Physical Performance Battery (SPPB)

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

SPPB comprises three components: Standing balance (open stand, half tandem stand and tandem stand), timed four meter usual paced walk, and timed test of five sit-to-stands from a standard chair. Each component is scored from 0 to 4, with maximum of 12and timed test of five sit-to-stands from a standard chair.

Four Square Step Test (FSST)

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

The FSST is a performance-based outcome measure assessing dynamic balance. The test set up consists of four walking sticks of the same width in a cross formation. Participants step into each square in a clockwise sequence and back anticlockwise as fast as possible while not being allowed to hit the sticks.

Unipedal Stance Test (UST)

Time Frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).

The UST is a balance assessment that is widely used in clinical settings to monitor neurological and musculoskeletal conditions. Abnormal values may indicate conditions that may impair balance and will quantify balance status for those who are at increased risks for fall. The test is performed barefoot, hands crossed over the chest and once with eyes open and once with eyes closed. Patient stands on one leg unassisted; time begins when opposite foot leaves the ground. Time stops immediately when arms are uncrossed, the raised foot is used to maintain balance or touches the ground, weight-baring foot is moved, 45 second have elapsed or when eyes were opened on eyes closed trial. Each condition (eyes open or eyes closed) is performed three times, alternatively.

Secondary Outcomes

  • Short Form 36(Baseline (Day 1))
  • Hemophilia Joint Health Score Version 2.1(Baseline (Day 1))
  • Hemophilia Activities List(Baseline (Day 1))

Study Sites (1)

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