What is the Feasibility of the ISTEP Exercise Test in Boys With Haemophilia

NCT04076306 | Completed

• 1 other identifier: 16HI32
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 1

Brief Summary

This study will be used to assess the feasibility and sensitivity of using the iSTEP, to assess exercise capacity in boys with haemophilia. The feasibility criteria to be explored includes:

1. 1.Recruitment to target number or better
2. 2.The test procedure is completed within an allocated time (1-2 hours) and by 90% of participants without serious adverse events
3. 3.90% of participants achieve a sufficient exercise response (85% maximum heart rate (HRmax))
4. 4.Calculation of estimates of minimum clinically important differences and variability for sample size calculations and responsiveness to severity of haemophilia and orthopaedic status The iSTEP exercise test will be compared to a more commonly used exercise test the modified shuttle walk test (10m- MSWT). Muscle strength (using myometry) will also be tested and compared to exercise performance for any relationships. Physical activity levels will also be assessed to obtain some baseline measurements of physical activity levels in this patient population, which are currently not very well known. The overarching aim of this current study is also to establish a robust and sensitive exercise test and to gain an understanding of the effects of physical activity levels and muscle strength on exercise capacity in this population.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (2)

• Feasibility of the study protocol, as measured by recruitment success.

  Percentage of participants recruited from the number of approached.

  10 months

• Safety and Acceptability of the study protocol, as measured by the occurrence of any serious adverse events or reactions.

  Evaluate whether the test procedure is completed within the allocated time (1-2 hours) without serious adverse events in \>90% of participants.

  10 months

Secondary Outcomes (5)

• Pain before, during, immediately post and 3 mins post exercise tests

  5 minutes

• Time spent in moderate to vigorous activity

  7 days

• Muscle Myometry

  15 minutes.

• Haemophilia Joint Health Score (HJHS) 2.1

  30 minutes.

• Duration exercise test completed in.

  Typically, up to 15 minutes.

Study Arms (1)

iSTEP Feasibility Study Protocol

OTHER

All participants will be asked to undergo the entire protocol: iSTEP exercise test, 10 metre incremental shuttle walk test, myometry, accelerometry and Haemophilia Joint Health Score.

Other: iSTEP exercise testing

Interventions

iSTEP exercise testing OTHER

iSTEP 10 minute step test, up and down on a commercial standard 3-height adjustable step Undertaking the 10m-MSWT 20-30 minutes Procedure takes place on testing track at Great Ormond Street Hospital; a 5 minute walk from the Haemophilia Centre. One of the research team accompanies the family with child to the track and will accompany them back to the Centre once testing is finished. Muscle Myometry 10 minutes, carried out by one of the research team at the end of the physiotherapy clinical assessment in the Haemophilia Centre. Wearing of accelerometer (activity/ step counter)7 days Device will be set up and provided at the end of the testing on the day of attendance at clinic. They will be instructed to wear this throughout the week at home when they are awake (at least 8 hours). Haemophilia Joint Health Score (HJHS) - a standardised outcome measure of joint health in people with Haemophilia, undertaken by the Clinical Specialist Physiotherapists in Haemophilia.

Also known as: 10 metre Incremental Shuttle Walk Test, Muscle Myometry, Accelerometry, Haemophilia Joint Health Score (HJHS)

iSTEP Feasibility Study Protocol

Eligibility Criteria

• Age: 6 Years - 15 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Boys with mild, moderate and severe haemophilia aged 6 - 15 years (haemophilia A or B)
• Inhibitor or non-inhibitor
• Child able to follow simple verbal instructions, provide informed consent or assent

You may not qualify if:

• Child has a past history of acquired brain injury
• Child has a past history of any other disturbance of the central nervous system
• Joint or muscle bleed in the lower limb in the past 6 weeks
• Presence of lower limb pain on day of testing
• Diagnosis of severe asthma or exercise induced bronchoconstriction

Sponsors & Collaborators

• Great Ormond Street Hospital for Children NHS Foundation Trust: lead
• University College, London: collaborator

Study Sites (1)

Research and Development Office, Division of Research and Innovation,

London, WC1N 1EH, United Kingdom

Location

Related Publications (1)

• Thorpe N, Harniess P, Main E, Hubert N, Rand S, Stephensen D, Liesner R, Bladen M. Feasibility, safety and acceptability of select outcome measures in a physiotherapy study protocol for boys with haemophilia. Pilot Feasibility Stud. 2021 May 6;7(1):105. doi: 10.1186/s40814-021-00831-1.

  PMID: 33957997 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Accelerometry

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

Intervention Hierarchy (Ancestors)

Investigative Techniques

Study Officials

• Nicola Thorpe

  Great Ormond Street Hospital

  PRINCIPAL INVESTIGATOR

Study Design

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

Model Details: Each child with haemophilia will undertake both the iSTEP exercise test and a modified shuttle walk test (10m-MSWT) in a randomised order on the same day with a washout period of at least 1 hour. This data will then be compared to previously collected normative data for the iSTEP test from healthy school age children from the original iSTEP development and validation study (Rand et al, 2015). The second component will include the feasibility of collecting standardised lower limb clinical muscle myometry measurements. The third component will be to explore the viability of collecting physical activity level data, in this population over a 7 day period using an accelerometer. This will be evaluated by monitoring the adherence to accelerometry wear. The value of the data will also be used to determine if it is useful at informing other aspects of fitness for boys with haemophilia.

Study Record Dates

• First Submitted: August 11, 2019
• First Posted: September 3, 2019
• Study Start: January 25, 2018
• Primary Completion: November 2, 2018
• Study Completion: November 2, 2018
• Last Updated: September 6, 2019

Record last verified: 2019-09

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)