Measurement Properties in People with Hemophilia

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Short Physical Performance Battery (SPPB) [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
• Four Square Step Test (FSST) [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
• Timed up and Go (TUG) [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
• Unipedal Stance Test (UST) [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
• 30-Second Stair Stand Test [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]
• 1 kg Arm Lift Test [Срок оценки: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).]

Вторичные конечные точки (3)

• Short Form 36 [Срок оценки: Baseline (Day 1)]
• Hemophilia Activities List [Срок оценки: Baseline (Day 1)]
• Hemophilia Joint Health Score Version 2.1 [Срок оценки: Baseline (Day 1)]

Критерии участия

Критерии включения

• 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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Другое

Центры проведения

Швейцария · 1 центр

• Ruud Knols — Zurich

Идентификаторы

NCT: NCT06565481 · 2024-01354

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗