Rehabilitation for ataxia study: protocol for a randomised controlled trial of an outpatient and supported home-based physiotherapy programme for people with hereditary cerebellar ataxia

Sarah C Milne; Louise A Corben; Melissa Roberts; David Szmulewicz; J Burns; Anneke C Grobler; Shannon Williams; Jillian Chua; Christina Liang; Phillipa J Lamont; Alison C Grootendorst; Libby Massey; Carolyn Sue; Kim Dalziel; Desiree LaGrappe; Liz Willis; Aleka Freijah; Paul Gerken; Martin B Delatycki

doi:10.1136/bmjopen-2020-040230

Rehabilitation for ataxia study: protocol for a randomised controlled trial of an outpatient and supported home-based physiotherapy programme for people with hereditary cerebellar ataxia

BMJ Open. 2020 Dec 17;10(12):e040230. doi: 10.1136/bmjopen-2020-040230.

Authors

Sarah C Milne^{1

2

3

4}, Louise A Corben^{1

3

4}, Melissa Roberts^{1

2}, David Szmulewicz^{5

6

7

8}, J Burns⁹, Anneke C Grobler^{4

10}, Shannon Williams^{11

12}, Jillian Chua¹³, Christina Liang^{14

15}, Phillipa J Lamont¹⁶, Alison C Grootendorst¹⁷, Libby Massey¹⁷, Carolyn Sue^{14

15}, Kim Dalziel¹⁸, Desiree LaGrappe¹⁷, Liz Willis¹⁷, Aleka Freijah¹⁹, Paul Gerken¹⁹, Martin B Delatycki^{20

4

21}

Affiliations

¹ Bruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, Victoria, Australia.
² Physiotherapy Department, Monash Health, Cheltenham, Victoria, Australia.
³ School of Primary and Allied Health Care, Monash University, Frankston, Victoria, Australia.
⁴ Department of Paediatrics, The University of Melbourne, Parkville, Victoria, Australia.
⁵ Balance Disorders & Ataxia Service, Royal Victorian Eye and Ear Hospital, East Melbourne, Victoria, Australia.
⁶ Cerebellar Ataxia Clinic, Alfred Health, Caulfield, Victoria, Australia.
⁷ Monash Medical Centre, Monash Health, Clayton, Victoria, Australia.
⁸ The Florey Institute of Neuroscience and Mental Health, Parkville, Victoria, Australia.
⁹ University of Sydney School of Health Sciences, Faculty of Medicine and Health & Children's Hospital at Westmead, Sydney, New South Wales, Australia.
¹⁰ Clinical Epidemiology and Biostatistics Unit, Murdoch Children's Research Institute, Parkville, Victoria, Australia.
¹¹ Physiotherapy Department, Royal Perth Hospital, Perth, Western Australia, Australia.
¹² Physiotherapy Department, Sir Charles Gairdner Hospital, Nedlands, Western Australia, Australia.
¹³ Physiotherapy Department, Ryde Hospital, Eastwood, New South Wales, Australia.
¹⁴ Department of Neurology, Royal North Shore Hospital, St Leonards, New South Wales, Australia.
¹⁵ Kolling Institute of Medical Research, University of Sydney, St Leonards, New South Wales, Australia.
¹⁶ Neurogenetic Unit, Royal Perth Hospital, Perth, Western Australia, Australia.
¹⁷ MJD Foundation, Darwin, Northern Territory, Australia.
¹⁸ Melbourne School of Population and Global Health, University of Melbourne, Parkville, Victoria, Australia.
¹⁹ Rehabilitation Services, Royal Darwin and Palmerston Hospitals, Darwin, Northern Territory, Australia.
²⁰ Bruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, Victoria, Australia martin.delatycki@vcgs.org.au.
²¹ Victorian Clinical Genetics Services, Melbourne, Victoria, Australia.

Abstract

Introduction: Emerging evidence indicates that rehabilitation can improve ataxia, mobility and independence in everyday activities in individuals with hereditary cerebellar ataxia. However, with the rarity of the genetic ataxias and known recruitment challenges in rehabilitation trials, most studies have been underpowered, non-randomised or non-controlled. This study will be the first, appropriately powered randomised controlled trial to examine the efficacy of an outpatient and home-based rehabilitation programme on improving motor function for individuals with hereditary cerebellar ataxia.

Methods and analysis: This randomised, single-blind, parallel group trial will compare a 30-week rehabilitation programme to standard care in individuals with hereditary cerebellar ataxia. Eighty individuals with a hereditary cerebellar ataxia, aged 15 years and above, will be recruited. The rehabilitation programme will include 6 weeks of outpatient land and aquatic physiotherapy followed immediately by a 24- week home exercise programme supported with fortnightly physiotherapy sessions. Participants in the standard care group will be asked to continue their usual physical activity. The primary outcome will be the motor domain of the Functional Independence Measure. Secondary outcomes will measure the motor impairment related to ataxia, balance, quality of life and cost-effectiveness. Outcomes will be administered at baseline, 7 weeks, 18 weeks and 30 weeks by a physiotherapist blinded to group allocation. A repeated measures mixed-effects linear regression model will be used to analyse the effect of the treatment group for each of the dependent continuous variables. The primary efficacy analysis will follow the intention-to-treat principle.

Ethics and dissemination: The study has been approved by the Monash Health Human Research Ethics Committee (HREC/18/MonH/418) and the Human Research Ethics Committee of the Northern Territory Department of Health and Menzies School of Health Research (2019/3503). Results will be published in peer-reviewed journals, presented at national and/or international conferences and disseminated to Australian ataxia support groups.

Trial registration number: ACTRN12618000908235.

Keywords: neurology; neuromuscular disease; rehabilitation medicine.

Publication types

Clinical Trial Protocol
Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Ataxia
Australia
Cerebellar Ataxia* / rehabilitation
Exercise Therapy
Humans
Outpatients*
Physical Therapy Modalities*
Quality of Life*
Randomized Controlled Trials as Topic
Single-Blind Method

Associated data

ANZCTR/ACTRN12618000908235