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

Primary Purpose

Hemophilia, Child, Only, Musculoskeletal Diseases or Conditions

Status
Recruiting
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
Exercise
Usual Care
Sponsored by
East Kent Hospitals University NHS Foundation Trust
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia focused on measuring Physical Therapy, Exercise, Muscle strength

Eligibility Criteria

6 Years - 12 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Haemophilia CentreRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Exercise

Usual care

Arm Description

12-week exercise intervention

Participants will continue to receive their usual care as prescribed by the haematologist, physiotherapist and other members of the healthcare team.

Outcomes

Primary Outcome Measures

Muscle strength of the dominant knee extensors at 24 weeks
Maximum isometric torque (Newton.metres/kilogram of body weight)

Secondary Outcome Measures

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

Full Information

First Posted
July 21, 2020
Last Updated
May 30, 2023
Sponsor
East Kent Hospitals University NHS Foundation Trust
Collaborators
Great Ormond Street Hospital for Children NHS Foundation Trust, University of Kent
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1. Study Identification

Unique Protocol Identification Number
NCT05895032
Brief Title
Trial of an Exercise Intervention for Children With Haemophilia
Acronym
DOLPHIN-II
Official Title
Development Of a haemophiLia PHysiotherapy INtervention for Optimum Musculoskeletal Health in Children (DOLPHIN-II) - a Randomised Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 6, 2021 (Actual)
Primary Completion Date
June 30, 2025 (Anticipated)
Study Completion Date
June 30, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
East Kent Hospitals University NHS Foundation Trust
Collaborators
Great Ormond Street Hospital for Children NHS Foundation Trust, University of Kent

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia, Child, Only, Musculoskeletal Diseases or Conditions
Keywords
Physical Therapy, Exercise, Muscle strength

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
A single-blinded, two-arm pragmatic randomised controlled trial
Masking
InvestigatorOutcomes Assessor
Masking Description
It is impossible to blind those in receipt and those delivering the intervention as to group allocation. However, it will be possible, although perhaps challenging, to blind the assessor to group allocation. Consequently, we will attempt a single blind approach in which the participant and physiotherapists delivering the intervention will be encouraged to withhold their group allocation from the assessors collecting data.
Allocation
Randomized
Enrollment
132 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Exercise
Arm Type
Experimental
Arm Description
12-week exercise intervention
Arm Title
Usual care
Arm Type
Active Comparator
Arm Description
Participants will continue to receive their usual care as prescribed by the haematologist, physiotherapist and other members of the healthcare team.
Intervention Type
Other
Intervention Name(s)
Exercise
Intervention Description
12-week exercise intervention
Intervention Type
Other
Intervention Name(s)
Usual Care
Intervention Description
usual care
Primary Outcome Measure Information:
Title
Muscle strength of the dominant knee extensors at 24 weeks
Description
Maximum isometric torque (Newton.metres/kilogram of body weight)
Time Frame
24-weeks
Secondary Outcome Measure Information:
Title
Muscle strength of the ankle plantarflexors and non-dominant knee extensors at 24 weeks
Description
maximum isometric torque (Newton.metres/kilogram of body weight)
Time Frame
24 weeks
Title
Bleeding episodes and coagulation factor usage
Description
Number of bleeding episodes (number) and amount of coagulation factor used for treatment (number)
Time Frame
24 weeks
Title
Six-minute timed walk (6MTW)
Description
Distance walked in 6 minutes (metres)
Time Frame
24 weeks
Title
Timed up and down stairs (TUDS)
Description
Time taken to ascend and descend a flight of 12 steps (seconds)
Time Frame
24 weeks
Title
Participation in physical activity
Description
Time spent in moderate-vigorous physical activity physical activity (MVPA)
Time Frame
24 weeks
Title
Child Health Utility 9D (CHU9D)
Description
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.
Time Frame
24 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
David Stephensen, PhD
Phone
01227783166
Email
david.stephensen@nhs.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Stephensen, PhD
Organizational Affiliation
East Kent Hospitals University NHS Foundation Trust
Official's Role
Study Chair
Facility Information:
Facility Name
Haemophilia Centre
City
Canterbury
State/Province
Kent
ZIP/Postal Code
CT1 3NG
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Stephensen, PhD

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
We do not have consent to share individual participant data

Learn more about this trial

Trial of an Exercise Intervention for Children With Haemophilia

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