Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 21/PR/1748 | Other Identifier | Health Research Authority |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Duchenne UK | UNKNOWN |
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydrotherapy | Other | Physiotherapy led pool session |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 | 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 | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Upper Limb Strength | Upper limb strength using grip and pinch measure using digital, handheld dynamometers North-Star | 24 weeks |
| Range of Motion | Limited ankle range of motion (ROM) Ankle plantarflexion-dorsiflexion (PF-DF ROM) will be assessed through a goniometer |
Not provided
Inclusion Criteria:
Exclusion Criteria:
DMD affects those born male.
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Christian De Goede | Lancashire Teaching Hospitals NHS Foundation Trust | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lancashire Teaching Hospitals NHS Foundation Trust | Preston | Lancashire | PR2 9HT | United Kingdom |
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 22, 2023 | Feb 14, 2024 | Prot_000.pdf |
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D006875 | Hydrotherapy |
| ID | Term |
|---|---|
| D026741 | Physical Therapy Modalities |
| D013812 | Therapeutics |
| D012046 | Rehabilitation |
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
| 24 weeks |
| Pulmonary function | Pulmonary function will be assessed using digital spirometry | 24 weeks |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |