Trial of an Exercise Intervention for Children With Haemophilia

Child, Only Clinical Trial

— DOLPHIN-II  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05895032
• Other study ID #: 2020/HAEMO/01
• Status: Recruiting
• Phase: N/A
• Start date: May 6, 2021
• Est. completion date: June 30, 2025

Study information

• Verified date: May 2023
• Source: East Kent Hospitals University NHS Foundation Trust
• Contact: David Stephensen, PhD
• Phone: 01227783166
• Email: david.stephensen[@]nhs.net
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

"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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 132
• Est. completion date: June 30, 2025
• Est. primary completion date: June 30, 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 12 Years

Eligibility

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.

Related Conditions & MeSH terms

• Child, Only
• Hemophilia
• Hemophilia A
• Musculoskeletal Diseases
• Musculoskeletal Diseases or Conditions

Intervention

Other:
• Exercise
12-week exercise intervention
• Usual Care
usual care

Locations

Country	Name	City	State
United Kingdom	Haemophilia Centre	Canterbury	Kent

Sponsors (3)

Lead Sponsor	Collaborator
East Kent Hospitals University NHS Foundation Trust	Great Ormond Street Hospital for Children NHS Foundation Trust, University of Kent

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Muscle strength of the dominant knee extensors at 24 weeks	Maximum isometric torque (Newton.metres/kilogram of body weight)	24-weeks
Secondary	Muscle strength of the ankle plantarflexors and non-dominant knee extensors at 24 weeks	maximum isometric torque (Newton.metres/kilogram of body weight)	24 weeks
Secondary	Bleeding episodes and coagulation factor usage	Number of bleeding episodes (number) and amount of coagulation factor used for treatment (number)	24 weeks
Secondary	Six-minute timed walk (6MTW)	Distance walked in 6 minutes (metres)	24 weeks
Secondary	Timed up and down stairs (TUDS)	Time taken to ascend and descend a flight of 12 steps (seconds)	24 weeks
Secondary	Participation in physical activity	Time spent in moderate-vigorous physical activity physical activity (MVPA)	24 weeks
Secondary	Child Health Utility 9D (CHU9D)	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