Measurement Properties in People with Hemophilia

NCT06565481 | Recruiting

• 1 other identifier: 2024-01354
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia a; Hemophilia B; Musculoskeletal Complication; Measurement Error

Outcome Measures

Primary Outcomes (6)

• Short Physical Performance Battery (SPPB)

  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.

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

• Four Square Step Test (FSST)

  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.

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

• Timed up and Go (TUG)

  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.

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

• Unipedal Stance Test (UST)

  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.

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

• 30-Second Stair Stand Test

  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

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

• 1 kg Arm Lift Test

  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

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

Secondary Outcomes (3)

• Short Form 36

  Baseline (Day 1)

• Hemophilia Activities List

  Baseline (Day 1)

• Hemophilia Joint Health Score Version 2.1

  Baseline (Day 1)

Study Arms (1)

Single Test-Re Test Arm

OTHER

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).

Other: Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia

Interventions

Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia OTHER

This study evaluates the reliability (Test-Retest) of six health related instruments that assess physical function in people with hemophilia: Short Physical Performance Battery, Unipedal Stance Test, Four Square Step Test, Timed up and Go, 1 kg Arm Lift Test, 30 Second Chair Stand Test. This study is needed to determine the psychometric properties of these six assessments. Validity of these test will be determined with the Short Form 36, the Hemophilia Activities List and the Hemophilia Joint Health Score Methodology: Reliability (Test re-test study and validity (cross sectional study) of the six tests reported in patients with hemophilia. This intervention contains physiotherapie assessments, however, no drugs nor medical devices.

Single Test-Re Test Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• University of Zurich: lead
• ETH Zurich: collaborator

Study Sites (1)

Ruud Knols

Zurich, 8091, Switzerland

RECRUITING

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

hydroxyethyl methacrylate

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; Genetic Diseases, X-Linked

Study Officials

• Ruud Knols, PhD

  University of Zurich

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ruud H Knols, PhD

CONTACT

0442558809; ruud.knols@usz.ch

Andrea Baerlocher, MSc

CONTACT

0442558679; andrea.baerlocher@usz.ch

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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).

Study Record Dates

• First Submitted: July 23, 2024
• First Posted: August 22, 2024
• Study Start: October 31, 2024
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): May 31, 2026
• Last Updated: January 14, 2025

Record last verified: 2024-08

Locations

CH (1)