Clinical trial NCT06445985: Hydrotherapy in Duchenne Muscular Dystrophy (DMD)
Official title: Hydrotherapy for Health in Boys and Adolescents With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Lancashire Teaching Hospitals NHS Foundation Trust
Sponsor class: OTHER
Collaborators: name: Duchenne UK
First posted: 2024-06-06
Enrollment: 31
Sex: MALE
Minimum age (years): 6
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention 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.
Primary purpose: SUPPORTIVE_CARE

Brief 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. 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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)

Primary outcomes:
measure: Body Mass via Bioelectrical impedance (BIA)
timeFrame: 24 weeks
measure: Pain Scale
timeFrame: 24 weeks
measure: PedsQL QoL / DMD QoL - Quality of Life
timeFrame: 24 weeks
Secondary outcomes:
measure: Upper Limb Strength
timeFrame: 24 weeks
measure: Range of Motion
timeFrame: 24 weeks
measure: Pulmonary function
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Preston
country: United Kingdom
facility: Lancashire Teaching Hospitals NHS Foundation Trust
state: Lancashire
Study officials:
affiliation: Lancashire Teaching Hospitals NHS Foundation Trust
name: Christian De Goede
role: PRINCIPAL_INVESTIGATOR
References: