Trial of an Exercise Intervention for Children With Haemophilia (DOLPHIN-II)

August 29, 2024 updated by: East Kent Hospitals University NHS Foundation Trust

Development Of a haemophiLia PHysiotherapy INtervention for Optimum Musculoskeletal Health in Children (DOLPHIN-II) - a Randomised Controlled Trial

"Being able to participate in games and activities with their friends" is one of the things that matters most to boys with haemophilia. At present, there is a lack of robust evidence to determine whether muscle strengthening exercise can improve or negatively affect outcomes for young children with haemophilia. With the help of boys with haemophilia, their parents and physiotherapists the investigators have developed an exercise programme designed to increase muscle strength. Using this intervention the investigators will undertake a single-blinded, two-arm pragmatic randomised controlled trial (RCT) of a 12-week intervention verses usual care of boys with haemophilia aged 6-12 years of age.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Children are born with haemophilia. Females carry the disorder and usually males are affected. It is a disorder affecting 1:10000 people where the blood does not clot normally, leading to bleeding into muscles and joints. As a result, muscles become weak. Joints become painful and difficult to move. "Being able to participate in games and activities with their friends" is one of the things that matters most to boys with haemophilia. "What is the role of exercise for both prevention and treatment of joint damage in haemophilia?" is one of the top unanswered questions that concern patients, carers and healthcare professionals most. At present, there is a lack of robust evidence to determine whether muscle strengthening exercise can improve or negatively affect outcomes for young children with haemophilia. With the help of boys with haemophilia, their parents and physiotherapists the investigators have developed an exercise programme designed to increase muscle strength. The investigators think the exercise programme might have an effect on pain and movement in participants joints, help them participate in games and activities with their friends and improve their health in the long term. The investigators recently showed the exercise programme had no harmful effects, was acceptable to children with haemophilia and that the participants were willing to participate in a study to answer the question, "does muscle strengthening help improve the long term health of children with haemophilia?" To answer this question, the investigators will allocate 66 boys with haemophilia to a group that is asked to complete the 12-week exercise routine to strengthen their leg muscles and another 66 boys to a group that does not do the exercises. The boys will be allocated at random, so that each boy has an equal chance of being in either group. Random allocation helps ensure that two similar groups of boys will be compared. The investigators will monitor the boys throughout the study by measuring their muscle strength, how far they can walk in six-minutes and time taken to ascend and descend 12 steps. The investigators will also record how physically active the boys are using a wrist band as well as how satisfied they are with their health. The study will be managed by a group of health care professionals and researchers with experience and expertise in carrying out this type of research. In addition, the investigators will include parents of boys with haemophilia in the research team to provide invaluable lived experience of living with the condition. So that people hear about what the investigators learn in the study, the investigators will report the findings to other researchers using journals, relevant health care professionals through face to face meetings, and children with haemophilia and their families through newsletters and presentations at Haemophilia Society meetings.

Study Type

Interventional

Enrollment (Estimated)

132

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United Kingdom
    • Kent
      • Canterbury, Kent, United Kingdom, CT1 3NG
        • Recruiting
        • Haemophilia Centre
        • Contact:
          • David Stephensen, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

• Children aged 6-12 years, with severe or moderate haemophilia A or B

Exclusion Criteria:

  • von Willebrand disease
  • past history of fracture or trauma to the lower limb
  • orthopaedic surgery
  • acquired brain injury or 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 or unable to fully comply with verbal instructions.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Exercise
12-week exercise intervention
Other: Exercise
12-week exercise intervention
Active Comparator: Usual care
Participants will continue to receive their usual care as prescribed by the haematologist, physiotherapist and other members of the healthcare team.
Other: Usual Care
usual care

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Muscle strength of the dominant knee extensors at 24 weeks
Time Frame: 24-weeks
Maximum isometric torque (Newton.metres/kilogram of body weight)
24-weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Muscle strength of the ankle plantarflexors and non-dominant knee extensors at 24 weeks
Time Frame: 24 weeks
maximum isometric torque (Newton.metres/kilogram of body weight)
24 weeks
Bleeding episodes and coagulation factor usage
Time Frame: 24 weeks
Number of bleeding episodes (number) and amount of coagulation factor used for treatment (number)
24 weeks
Six-minute timed walk (6MTW)
Time Frame: 24 weeks
Distance walked in 6 minutes (metres)
24 weeks
Timed up and down stairs (TUDS)
Time Frame: 24 weeks
Time taken to ascend and descend a flight of 12 steps (seconds)
24 weeks
Participation in physical activity
Time Frame: 24 weeks
Time spent in moderate-vigorous physical activity physical activity (MVPA)
24 weeks
Child Health Utility 9D (CHU9D)
Time Frame: 24 weeks
a paediatric preference based measure of health related quality of life (utility values on the 0.0 to 1.0 quality adjusted life year scale) Higher score represent higher adjusted life years.
24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

East Kent Hospitals University NHS Foundation Trust

Collaborators

Great Ormond Street Hospital for Children NHS Foundation Trust
University of Kent

Investigators

  • Study Chair: David Stephensen, PhD, East Kent Hospitals University NHS Foundation Trust

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 6, 2021

Primary Completion (Estimated)

June 30, 2025

Study Completion (Estimated)

June 30, 2025

Study Registration Dates

First Submitted

July 21, 2020

First Submitted That Met QC Criteria

May 30, 2023

First Posted (Actual)

June 8, 2023

Study Record Updates

Last Update Posted (Actual)

August 30, 2024

Last Update Submitted That Met QC Criteria

August 29, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2020/HAEMO/01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

We do not have consent to share individual participant data

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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