None listed
Conditions
Brief summary
This study aims to compare the effectiveness of an intensive rehabilitation program (consisting of a six-week outpatient rehabilitation program followed by a supported home exercise program (HEP)) compared with standard care on motor function in individuals with hereditary cerebellar ataxia. The study will be a multi-centre randomised controlled trial. The intervention group will receive outpatient rehabilitation three days per week for six-weeks followed by 24 weeks of a supported HEP (fortnightly teleconference/home visits), while the control group will be asked to continue their current care for 30 weeks. Rehabilitation will be based on six domains of rehabilitation: strengthening, balance, functional mobility practice, postural control, sensory stimulation and coordination and control, and will include land and aquatic physiotherapy. Assessment will occur at baseline, and six, 18 and 30 weeks after baseline. The primary outcome will be the motor domain of the Functional Independence Measure. Secondary outcomes will measure ataxia symptoms, quality of life, balance and patient perceived benefit.
Interventions
The intervention involves a six-week outpatient rehabilitation program followed by a 24-week supported home exercise program. The individualised rehabilitation is targeted at improving motor function, mobility and balance and the home exercise program has been designed to enhance completion of the program. The outpatient program will consist of two hours of rehabilitation provided by a physiotherapist, three times per week. Therapy will be one-on-one. This will be held in a public hospital physi
The intervention involves a six-week outpatient rehabilitation program followed by a 24-week supported home exercise program. The individualised rehabilitation is targeted at improving motor function, mobility and balance and the home exercise program has been designed to enhance completion of the program. The outpatient program will consist of two hours of rehabilitation provided by a physiotherapist, three times per week. Therapy will be one-on-one. This will be held in a public hospital physiotherapy gym and hydrotherapy pool. The home exercise program will require participants to exercise for one hour, five days per week. The home exercise program will be supported by monthly remote supervision, via telephone or video applications, alternating with fortnightly home visits by the physiotherapist. Participants will be required to record exercise completion with a participant diary and will be asked about their exercise completion at each fortnightly physiotherapy session. The rehabilitation (both outpatient and home-based) will be based on multi-faceted individualised rehabilitation designed on six domains of rehabilitation: strengthening, sensory stimulation, balance training, postural control, coordination and control and functional mobility. A specific time is allocated to each domain. A list of therapy options within each domain is provided for the treating physiotherapists to select from. The physiotherapist will assess each individual at the beginning of the intervention to ensure the chosen therapy options are appropriate for (i) the participant’s capacity and impairments; (ii) the environment of the home exercise program; (iii) the specific needs of each participant; and (iv) the feasibility of successfully delivering the rehabilitation at the site. Exercises will be progressed according to the individual’s performance of each exercise, their fatigue and motivation levels, and their goals.
Sponsors
Murdoch Children's Reseach Institute
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
15 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Have a molecular diagnosis or at least three generations affected, of a recessive or dominant inherited cerebellar ataxia. 2. Aged over 15 years 3. A minimum score of 2 for question 1: Gait of the Scale for the Assessment and Rating of Ataxia (SARA) [2 = Gait clearly abnormal, tandem walking > 10 steps not possible]. 4. A minimum score of 4 for item I: Transfers Bed, Chair, Wheel-chair of the Functional Independence Measure [4 = Minimal Assistance, the participant completes 75% or more of the task]. 5. Given clearance by cardiologist or other appropriate medical professional for participation in rehabilitation and the hydrotherapy component. 6. Able to give informed consent.
Exclusion criteria
1. Individuals with non-genetic ataxia or non-degenerative ataxia. 2. Musculoskeletal injury limiting ability to weight-bear. 3. Another medical condition not related to their hereditary cerebellar ataxia that impacts on mobility. 4. Undergone major orthopaedic surgery in the last six months. 5. Need for immediate intensive intervention for safety reasons. 6. Pregnancy. 7. Dementia or significant cognitive impairment limiting ability to give informed consent and participate in the rehabilitation program. 8. Received botulinum toxin injections for spasticity management (except for regular longstanding paraspinal botulinum injections - this is defined as at least two doses of botulinum injections in the same muscle/s within eight months of the screening period). 9. Already completing greater than three hours per week physical exercise/therapy focused on the lower limb (i.e. pilates, personal trainer, home exercise program, independent gym program, exercise physiology) or is participating in a structured goal-based physiotherapy rehabilitation program. This does not include physical activity that occurs as part of the person's daily life, i.e. walking to the shops. 10. Currently enrolled in a clinical trial or planned enrolment in a clinical trial during the period of the study. 11. Has a medical condition that precludes entry into a hydrotherapy pool, including bowel incontinence.
Outcome results
None listed