Hemophilia aHEMOPRONCT06565481
Measurement Properties in People with Hemophilia
A study testing Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia in people with Hemophilia a and Hemophilia B, run by University of Zurich at 1 site in Switzerland.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia a, Hemophilia B, Musculoskeletal Complication
- What is being tested?
- Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteSwitzerland
- Study dates
- 31 Oct 2024to 31 May 2026
- Phase
- Interventional
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Severe health conditions like severe cardiovascular, pulmonary, neurological or musculoskeletal diseases.
- Unable to perform the assessments due to disability or language problems.
Trial locations and contacts
1 site in 1 country.
Switzerland1 site, 1 recruiting
- Recruiting
Ruud Knols
Zurich, 8091
Ruud Knols, PhD, 0041442558809, ruud.knols@usz.ch
About this study
Official title: Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia
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.
Read the 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.
- Being tested
- Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with HemophiliaOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Other: Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).
Secondary outcomes (3)
Short Form 36
The Short Form 36 is a generic health related quality of life instrument consisting of 36 items pertaining to eight dimensions ('physical functioning', 'role physical functioning', 'bodily pain', 'general health perception', 'vitality', 'social functioning', 'role emotional functioning' and 'mental health'. The possible score ranges from 0 to 100 points, with 0 points representing the greatest possible health restriction, while 100 points indicate no health restriction.
In addition, each item is scored on a 0 to 100 range so that the lowest and highest possible scores are 0 and 100, respectively.
Measured: Baseline (Day 1)
Hemophilia Activities List
The Hemophilia Activities List measures the impact of hemophilia on self-perceived functional abilities in adults. It contains 42 multiple choice questions in seven domains: Lying/sitting/kneeling/standing (8 items), Functions of the legs (9 items), Functions of the arms (4 items), Use of transportation (3 items), Self-care (5 items), Household tasks (6 items), Leisure activities and sports (7 items). The final version of the HAL has good internal consistency and convergent validity and gives the clinician insight into a patient's self-perceived ability to perform activities of daily life. The possible scores range from 0 to 100, where 0 represents the worst possible functional status and 100 the best possible functional status.
Measured: Baseline (Day 1)
Hemophilia Joint Health Score Version 2.1
The Hemophilia Joint Health Score2.1 includes swelling, duration (of swelling), muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain, strength, and global gait.
For each of the Elbow, Knee and Ankle joints (left / rigth), the following items are scored: swelling (scored 0-3), duration of swelling (0-1), muscle atrophy (0-2), crepitus on motion (0-2), flexion loss (0-3), extension loss (0-3), joint pain (0-2), and strength (0-4). The maximum score for an individual index joint is 20. Gait is scored 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
Measured: Baseline (Day 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2024
- Study start31 Oct 2024
- Primary completion31 May 2026 (estimated)
- Study completion31 May 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia aStudies of the same condition, open and under way.
- More trials run by University of ZurichThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- 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).
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Zurich
- Sponsor type
- University, hospital or other
- Collaborators
- ETH Zurich
- Responsible party
- Sponsor
- Investigators
- Ruud Knols, PhD, Principal investigator, University of Zurich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06565481
- Sponsor's protocol ID
- 2024-01354
- Acronym
- HEMOPRO
- Status last verified
- Aug 2024
- First submitted
- 23 Jul 2024
- Last updated
- 14 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Measurement Properties (Reliability and Validity) of a Set of Assessments Evaluating Physical Function in People with Hemophilia
- ClinicalTrials.gov ID
- NCT06565481
- Acronym / sponsor ID
- HEMOPRO / 2024-01354
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- University of ZurichUniversity, hospital or other
- Collaborators
- ETH Zurich
- Study start
- 31 Oct 2024 (actual)
- Primary completion
- 31 May 2026 (estimated)
- Study completion
- 31 May 2026 (estimated)
- First posted
- 22 Aug 2024 (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.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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).
| Arm | Type | Receives |
|---|---|---|
| Single Test-Re Test Arm | Other | Other: Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with HemophiliaThis 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (2 listed)
- Severe health conditions like severe cardiovascular, pulmonary, neurological or musculoskeletal diseases.
- Unable to perform the assessments due to disability or language problems.
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 |
|---|---|---|
| Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with HemophiliaThis 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. | Other | Single Test-Re Test Arm |
Primary outcome
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.
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.
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.
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
3 secondary outcomes
Short Form 36
Time frame: Baseline (Day 1)
Hemophilia Activities List
Time frame: Baseline (Day 1)
Hemophilia Joint Health Score Version 2.1
Time frame: Baseline (Day 1)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2025.
Related trials for Hemophilia a
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
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.
Ruud Knols, PhD principal investigator
University of Zurich
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia in people with Hemophilia a and Hemophilia B, run by University of Zurich at 1 site in Switzerland.
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 5 inclusion and 2 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 14 Jan 2025. 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 Switzerland. 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 Ruud H Knols, PhD (0442558809, ruud.knols@usz.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Short Physical Performance Battery (SPPB) (time frame: Baseline (Day 1) followed after a minimum break of 48 hours by Follow up (Day 2).). The study lists 6 primary outcomes in all. 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 14 Jan 2025.