iSTEP feasibility and sensitivity for boys with haemophilia

• Research type

  Research Study

• Full title

  What is the Feasibility and Sensitivity of the iSTEP in Boys with Haemophilia?

• IRAS ID

  212119

• Contact name

  Melanie Bladen

• Contact email

  melanie.bladen@gosh.nhs.uk

• Sponsor organisation

  Great Ormond Street Hospital for Children NHS Foundation Trust

• Duration of Study in the UK

  1 years, 0 months, 27 days

• Research 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. Recruitment to target number or better
  2. The test procedure is completed within an allocated time (1-2 hours) and by 90% of participants without serious adverse events
  3. 90% of participants achieve a sufficient exercise response (85% maximum heart rate (HRmax))
  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.

• REC name

  London - Central Research Ethics Committee

• REC reference

  17/LO/1192

• Date of REC Opinion

  2 Aug 2017

• REC opinion

  Favourable Opinion