Duchenne Muscular DystrophyNCT06445985
Hydrotherapy in Duchenne Muscular Dystrophy (DMD)
A study testing Hydrotherapy in people with Duchenne Muscular Dystrophy, run by Lancashire Teaching Hospitals NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
The goal of this clinical trial aims to establish if there are meaningful benefits to providing a hydrotherapy service for young people with Duchenne muscular dystrophy (DMD). The main aims are to: 1. Read more about this study
- Who is it for?
- Ages 6 to 25 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Hydrotherapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 2 Nov 2022to 12 Dec 2025
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 25 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Established diagnosis of Duchenne Muscular Dystrophy (either by genetics or muscle biopsy)
- Between 6 and 25 years of age
- On stable dose of steroids or not on steroids
Who cannot join
- Younger than 6 years, older than 25 years
- Recent change in steroid dose, less than 3 months prior
- Undertaking formal hydrotherapy supervised by physiotherapist on a regular basis (weekly or more frequent)
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Lancashire Teaching Hospitals NHS Foundation Trust
Preston, Lancashire, PR2 9HT
About this study
Official title: Hydrotherapy for Health in Boys and Adolescents With Duchenne Muscular Dystrophy
The goal of this clinical trial aims to establish if there are meaningful benefits to providing a hydrotherapy service for young people with Duchenne muscular dystrophy (DMD). The main aims are to: 1. to allocate a clinical physiotherapist to a project implementing hydrotherapy in young patients with DMD to establish whether there are meaningful benefits to their daily life. 2. to conduct patient and parent interviews to understand the barriers to completing a hydrotherapy intervention and ensure future research addresses meaningful outcomes for those with DMD.
Read the detailed description
The quality of life in young males with Duchenne muscular dystrophy (DMD) is negatively impacted by daily pain, changes in body composition and a lack of support to undertake physical activity. Hydrotherapy represents a potential means of involving boys and adolescents with DMD in activity that could benefit the negative factors influencing their quality of life. There are presently no guidelines or evidence for the benefits of hydrotherapy with the standards of care for young males with DMD.
This trial will provide evidence that will allow care providers to advocate the use of hydrotherapy within the management of DMD, as an inclusive activity, that can be adopted by those with DMD who are either ambulatory, or non-ambulatory. Through 12-weeks of hydrotherapy, the trial will investigate whether there are benefits to physical function, pain and quality of life. These measures represent meaningful outcomes in the progression of DMD, and have direct patient impact for those affected by DMD. On completion of the hydrotherapy, there will be a series of interviews with some of the participants and their parents. The aim of these interviews is to understand participant and parent barriers to hydrotherapy, and uncover whether hydrotherapy improves the lives of the participants beyond simple clinical measures and questionnaires.
The study aims recruit 44 boys and young men with Duchenne muscular dystrophy. Patients will be recruited from NHS neuromuscular services and through families who attend the local hospice.
Following an informed consent process they will enter the first 12 week stage, as a control to observe and measure their habitual physical activity, along with monitoring their diet. They will then enter the intervention stage where they will have a weekly physiotherapy led pool session with a number of physical activities. During the hydrotherapy period, participants will access the hydrotherapy pool once a week for 12 weeks. Sessions are supervised by an experienced physiotherapist, who will guide the exercise for a session lasting 30 to 60 minutes.
The exercises, intensity and specific hydrotherapy plan will be derived from consultation with the participants and their parents. Due to the variance within the presentation of the condition, and the inclusive age range within the study, an externally valid approach to exercise prescription will be utilised. Diet and physical function will be measured during this stage.
There will be a number of assessments to be completed at 3 time points. As much as possible these will be arranged as single visits. The first before the control period, the second after the control period, and the third after the hydrotherapy period. The assessments include a number of physical assessments, including assessments of function and ability, body mass and quality of life. After completion, all participants will be invited to complete a qualitative interview, in particular enquiring about the experience and logistics of hydrotherapy.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Hydrotherapy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydrotherapyOther
Physiotherapy led pool session
| Study group | Role | What they receive |
|---|---|---|
| Control | No intervention | Participants will have a 12 week period where they will continue with their habitual physical activity behaviour. Diet and physical activity will be monitored |
| Hydrotherapy | Active comparator | Participants will complete 12-weeks of hydrotherapy, involving up to 60 mins of hydrotherapy once a week. Diet and physical activity will be monitored. Receives: Other: Hydrotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Body Mass via Bioelectrical impedance (BIA)
Fat mass, body fat percentage and fat free mass will be measured using BIA (bioelectrical impedance). BIA has been validated in DMD and is accurate enough to measure longitudinal changes in body composition and muscle mass in this population
Measured: 24 weeks
Pain Scale
A pain map assessment of the topographic distribution of daily pain will also be competed, consistent with our previous work in DMD. Scale of 1-10
Measured: 24 weeks
PedsQL QoL / DMD QoL - Quality of Life
PedsQL QoL - Quality of Life for both participants, and the DMD-QoL Proxy for parents - Scale of 0-4
Measured: 24 weeks
Secondary outcomes (3)
Upper Limb Strength
Upper limb strength using grip and pinch measure using digital, handheld dynamometers North-Star
Measured: 24 weeks
Range of Motion
Limited ankle range of motion (ROM) Ankle plantarflexion-dorsiflexion (PF-DF ROM) will be assessed through a goniometer
Measured: 24 weeks
Pulmonary function
Pulmonary function will be assessed using digital spirometry
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2024
- Study start2 Nov 2022
- Primary completion1 Apr 2025 (estimated)
- Study completion12 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Patients with DMD, show great variability in age and speed of deterioration. As such it is difficult to find matched controls for a short randomised intervention study. Instead, due to the progressive nature of DMD and the inherent variance within a mixed age and ability population, the participants will act as their own controls. This single-arm, non-crossover design where the participant acts as his own control, although not as robust as randomized control exercise trials, is advocated for longitudinal interventions in populations such as the present one.
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lancashire Teaching Hospitals NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Duchenne UK
- Responsible party
- Sponsor
- Investigators
- Christian De Goede, Principal investigator, Lancashire Teaching Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06445985
- Sponsor's protocol ID
- 304633
- Other IDs
- 21/PR/1748
- Status last verified
- Jan 2025
- First submitted
- 14 Feb 2024
- Last updated
- 30 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The results of this study will be used to inform the neuromuscular service in Preston and at other sites in the UK. The results will also be shared more widely by presenting them at regional and or national meetings. The results may be published in a scientific journal and presented at scientific medical conferences and through publications and meetings organised by Duchenne UK.
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydrotherapy for Health in Boys and Adolescents With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06445985
- Acronym / sponsor ID
- 304633
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Lancashire Teaching Hospitals NHS Foundation TrustUniversity, hospital or other
- Collaborators
- Duchenne UK
- Study start
- 2 Nov 2022 (actual)
- Primary completion
- 1 Apr 2025 (estimated)
- Study completion
- 12 Dec 2025 (estimated)
- First posted
- 6 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial aims to establish if there are meaningful benefits to providing a hydrotherapy service for young people with Duchenne muscular dystrophy (DMD). The main aims are to: 1. to allocate a clinical physiotherapist to a project implementing hydrotherapy in young patients with DMD to establish whether there are meaningful benefits to their daily life. 2. to conduct patient and parent interviews to understand the barriers to completing a hydrotherapy intervention and ensure future research addresses meaningful outcomes for those with DMD.
Detailed description
The quality of life in young males with Duchenne muscular dystrophy (DMD) is negatively impacted by daily pain, changes in body composition and a lack of support to undertake physical activity. Hydrotherapy represents a potential means of involving boys and adolescents with DMD in activity that could benefit the negative factors influencing their quality of life. There are presently no guidelines or evidence for the benefits of hydrotherapy with the standards of care for young males with DMD.
This trial will provide evidence that will allow care providers to advocate the use of hydrotherapy within the management of DMD, as an inclusive activity, that can be adopted by those with DMD who are either ambulatory, or non-ambulatory. Through 12-weeks of hydrotherapy, the trial will investigate whether there are benefits to physical function, pain and quality of life. These measures represent meaningful outcomes in the progression of DMD, and have direct patient impact for those affected by DMD. On completion of the hydrotherapy, there will be a series of interviews with some of the participants and their parents. The aim of these interviews is to understand participant and parent barriers to hydrotherapy, and uncover whether hydrotherapy improves the lives of the participants beyond simple clinical measures and questionnaires.
The study aims recruit 44 boys and young men with Duchenne muscular dystrophy. Patients will be recruited from NHS neuromuscular services and through families who attend the local hospice.
Following an informed consent process they will enter the first 12 week stage, as a control to observe and measure their habitual physical activity, along with monitoring their diet. They will then enter the intervention stage where they will have a weekly physiotherapy led pool session with a number of physical activities. During the hydrotherapy period, participants will access the hydrotherapy pool once a week for 12 weeks. Sessions are supervised by an experienced physiotherapist, who will guide the exercise for a session lasting 30 to 60 minutes.
The exercises, intensity and specific hydrotherapy plan will be derived from consultation with the participants and their parents. Due to the variance within the presentation of the condition, and the inclusive age range within the study, an externally valid approach to exercise prescription will be utilised. Diet and physical function will be measured during this stage.
There will be a number of assessments to be completed at 3 time points. As much as possible these will be arranged as single visits. The first before the control period, the second after the control period, and the third after the hydrotherapy period. The assessments include a number of physical assessments, including assessments of function and ability, body mass and quality of life. After completion, all participants will be invited to complete a qualitative interview, in particular enquiring about the experience and logistics of hydrotherapy.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients with DMD, show great variability in age and speed of deterioration. As such it is difficult to find matched controls for a short randomised intervention study. Instead, due to the progressive nature of DMD and the inherent variance within a mixed age and ability population, the participants will act as their own controls. This single-arm, non-crossover design where the participant acts as his own control, although not as robust as randomized control exercise trials, is advocated for longitudinal interventions in populations such as the present one.
| Arm | Type | Receives |
|---|---|---|
| Control | No intervention | –Participants will have a 12 week period where they will continue with their habitual physical activity behaviour. Diet and physical activity will be monitored |
| Hydrotherapy | Active comparator | Other: HydrotherapyParticipants will complete 12-weeks of hydrotherapy, involving up to 60 mins of hydrotherapy once a week. Diet and physical activity will be monitored. |
Who may qualify
- Ages
- 6 to 25 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Established diagnosis of Duchenne Muscular Dystrophy (either by genetics or muscle biopsy)
- Between 6 and 25 years of age
- On stable dose of steroids or not on steroids
Key exclusion criteria (3 listed)
- Younger than 6 years, older than 25 years
- Recent change in steroid dose, less than 3 months prior
- Undertaking formal hydrotherapy supervised by physiotherapist on a regular basis (weekly or more frequent)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydrotherapyPhysiotherapy led pool session | Other | Hydrotherapy |
Primary outcome
Body Mass via Bioelectrical impedance (BIA)
Time frame: 24 weeks
Fat mass, body fat percentage and fat free mass will be measured using BIA (bioelectrical impedance). BIA has been validated in DMD and is accurate enough to measure longitudinal changes in body composition and muscle mass in this population
Pain Scale
Time frame: 24 weeks
A pain map assessment of the topographic distribution of daily pain will also be competed, consistent with our previous work in DMD. Scale of 1-10
PedsQL QoL / DMD QoL - Quality of Life
Time frame: 24 weeks
PedsQL QoL - Quality of Life for both participants, and the DMD-QoL Proxy for parents - Scale of 0-4
3 secondary outcomes
Upper Limb Strength
Time frame: 24 weeks
Range of Motion
Time frame: 24 weeks
Pulmonary function
Time frame: 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2025.
- Individual participant dataSharing planned: No. The results of this study will be used to inform the neuromuscular service in Preston and at other sites in the UK. The results will also be shared more widely by presenting them at regional and or national meetings. The results may be published in a scientific journal and presented at scientific medical conferences and through publications and meetings organised by Duchenne UK.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christian De Goede principal investigator
Lancashire Teaching Hospitals NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hydrotherapy in people with Duchenne Muscular Dystrophy, run by Lancashire Teaching Hospitals NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Body Mass via Bioelectrical impedance (BIA) (time frame: 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2025.