Contracture management in Duchenne muscular dystrophy
| ISRCTN | ISRCTN18436655 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN18436655 |
| IRAS number | 258344 |
| Secondary identifying numbers | IRAS 258344, CPMS 47133 |
- Submission date
- 16/10/2023
- Registration date
- 09/02/2024
- Last edited
- 18/12/2023
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Musculoskeletal Diseases
Plain English summary of protocol
Background and study aims
Duchenne Muscular Dystrophy (DMD) is a condition affecting boys, causing weak muscles and stiff ankles. This makes activities such as walking and climbing stairs difficult. Making sure ankles don’t get too stiff may help with these. This is usually done by stretching the ankles every day and wearing splints overnight. A splint is a hard, rigid boot worn to hold the foot in a fixed position, stretching the ankle and making it less stiff. There are different splints which are worn for different lengths of time but we don’t know which is the best type. The aim of this study is to determine the feasibility of a full-scale study comparing ankle foot orthoses with contracture control devices in the management of ankle contractures in ambulant boys with DMD.
Who can participate?
Boys aged between 4 and 10 years who have been diagnosed with DMD, who can walk but have stiff ankles and who have either not used splints before or have been given one type of splint in the past year can take part. Initially the study was open only to boys who received care from the neuromuscular team in Newcastle but following some changes, boys from other parts of the country were able to take part.
What does the study involve?
Once agreeing to take part, each boy is randomly put into a group and asked to wear either ankle foot orthoses for at least 8 hours overnight or contracture control devices for 2 hours and both groups are required to complete daily stretches and a daily adherence diary. There are five trial visits with assessments over a 12-week period.
What are the potential benefits and risks of participating?
There is no guarantee that participating will offer any direct benefit but it will help find out important information to help improve how we look after boys with DMD in the future. This is a low-risk trial. Both types of splints have been used in patients with DMD in Newcastle and also in patients with other conditions. Both splints have been reported as being safe and well-tolerated. There is a small chance of discomfort or skin irritation if the splint is not fitted well or applied correctly. If this occurs, it will be assessed and managed during the visits. There is also a small chance of discomfort from stretching the tight muscles either from the stretch applied by the splint or during the stretches being performed. This often improves and reduces as the boys become used to wearing the splints and performing the stretches.
Where is the study run from?
Trial visit assessments take place at Peacocks which is an orthotics clinic in Newcastle. The team leading the study are based at the John Walton Muscular Dystrophy Research Centre at the Centre for Life in Newcastle (UK)
When is the study starting and how long is it expected to run for?
January 2020 to December 2022
Who is funding the study?
Duchenne UK
Who is the main contact?
Dionne Moat, Dionne.moat1@nhs.net
Contact information
Public, Scientific, Principal Investigator
JWMDRC
Centre for Life
Newcastle
NE1 3BZ
United Kingdom
| Phone | +44 (0)191 2418756 |
|---|---|
| dionne.moat@newcastle.ac.uk |
Study information
| Study design | Randomized controlled trial pilot study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Workplace, Other |
| Study type | Treatment |
| Participant information sheet | 44410_PIS_adults_V1.4_03Dec21.pdf |
| Scientific title | A pilot study to compare static night time ankle foot orthosis with contracture control device in the management of ankle contractures in ambulant boys with Duchenne muscular dystrophy |
| Study objectives | A pilot study to determine the feasibility of a full scale study comparing ankle foot orthoses (AFO) with contracture control devices (CCDs) in the management of ankle contractures in ambulant boys with Duchenne Muscular Dystrophy (DMD). |
| Ethics approval(s) |
Approved 01/03/2021, London – Surrey Research Ethics Committee (Nottingham Centre, Nottingham, NG1 6FS, United Kingdom; +44 (0)207 104 8088, +44 (0)207 104 8131; surrey.rec@hra.nhs.uk), ref: 20/LO/1107 |
| Health condition(s) or problem(s) studied | Duchenne muscular dystrophy |
| Intervention | A single-centre, single-blinded, randomised control trial pilot study comparing CCDs and AFOs over 12 weeks of intervention. Once agreeing to take part, each boy is randomly put into a group and asked to wear either AFO for at least 8 hours overnight or CCDs for 2 hours and both groups are required to complete daily stretches and a daily adherence diary. Participants were randomized in a 1:1 ratio. A randomisation application (Sealed Envelope Ltd, 2019) was used. Group 1 – current standard of care treatment AFO + daily stretches Splints were worn overnight for at least 8 hours as per recommendations and active or passive ankle stretches as demonstrated by the clinical physiotherapist performed daily as per recommendations Group 2 – CCDs + daily stretches Splints were worn for 2 hours daily as per recommendations for this device and active or passive ankle stretches as demonstrated by the clinical physiotherapist performed daily as per recommendations. There are five trial visits with assessments over a 12-week period. Benefit was evaluated using range of motion (ROM) of ankles and North Star Ambulatory Assessment (NSAA). Inter and intra-rater reliability of these was also calculated using ICC. Patient satisfaction and adherence to both stretches and devices were evaluated using a self-completed diary and a validated satisfaction questionnaire, the Orthotic and Prosthetic User Satisfaction Survey (OPUS). |
| Intervention type | Device |
| Pharmaceutical study type(s) | Not Applicable |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Ankle Foot Orthosis (AFO), Contracture Control Device (CCD) |
| Primary outcome measure | Feasibility of trial design, measured using attrition rate assessed using the number of participants who consented to participate who remained in the study until the end of follow-up at 12 weeks. |
| Secondary outcome measures | 1. Ankle ROM measured using goniometry at [baseline T1, baseline T2, week 4 T4, week 8 T5 (no week 8 visits following amendment), week 12 end of study T6] 2. Motor function measured using the North Star ambulatory assessment (NSAA) at baseline T1, baseline T2, week 4 T4, week 8 T5 (no week 8 visits following amendment), week 12 end of study T6 3. Satisfaction with service and device measured using the Orthotics and Prosthetics User's Survey (OPUS) at week 2 T3, week 4 T4, week 8 T5 (no week 8 visits following amendment), week 12 end of study T6 4. Adherence to the intervention measured using a daily diary for 12 weeks |
| Overall study start date | 29/01/2020 |
| Completion date | 02/12/2022 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 4 Years |
| Upper age limit | 10 Years |
| Sex | Male |
| Target number of participants | 20 |
| Total final enrolment | 5 |
| Key inclusion criteria | Inclusion criteria: primary submission: 1. Genetic diagnosis of DMD 2. Ankle ROM of between +10 degrees of dorsiflexion and -10 degrees of dorsiflexion. 3. On assessment ROM has either deteriorated or remained the same since prior appointment despite good adherence to current stretching regime 4. Aged between 4 and 10 years of age 5. Orthotic naïve AMENDMENT to inclusion criteria: Re-submission: 1. Ankle orthotic naïve or supplied with an AFO in the last 18 months that currently fits well 2. From JWMDRC clinics or referred to us by a centre which borders us 3. Email or written evidence that the local specialist team are amenable to our oversight of orthotics for the duration of the study |
| Key exclusion criteria | 1. Significant behavioural issues that would make adherence problematic 2. Previous or current lower limb fracture within the last year 3. Previous tenotomy or other interventions for contracture management 4. Non-English speaking |
| Date of first enrolment | 21/04/2021 |
| Date of final enrolment | 20/10/2022 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
Centre For Life
Central Parkway
Newcastle
NE1 3BZ
United Kingdom
Sponsor information
Hospital/treatment centre
Freeman Road
High Heaton
Newcastle upon Tyne
NE7 7DN
England
United Kingdom
| Phone | +44 (0)191 2336161 |
|---|---|
| elaine.chapman4@nhs.net | |
| Website | http://www.newcastle-hospitals.org.uk/ |
"ROR" |
https://ror.org/05p40t847 |
Funders
Funder type
Charity
Government organisation / Other non-profit organizations
- Alternative name(s)
- Duchenne UK, THE DUCHENNE RESEARCH FUND, DRF
- Location
- United Kingdom
Results and Publications
| Intention to publish date | 30/06/2024 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | 1. Planned publication in a high-impact peer-reviewed journal 2. Poster presentation at the World Muscle Society International conference completed 2022 |
| IPD sharing plan | The datasets generated during and/or analysed during the current study will be available upon request from Dionne Moat (Dionne.moat1@nhs.net). |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Participant information sheet | Adults version 1.4 |
03/12/2021 | 06/11/2023 | No | Yes |
| Participant information sheet | Children aged 4 to 6 years version 1.3 |
03/12/2021 | 06/11/2023 | No | Yes |
| Participant information sheet | Children aged 6 to 10 years version 1.3 |
03/12/2021 | 06/11/2023 | No | Yes |
Additional files
- 44410_PIS_children4-6_V1.3_03Dec21.pdf
- Children aged 4 to 6 years
- 44410_PIS_children 6-10_V1.3_03Dec21.pdf
- Children aged 6 to 10 years
- 44410_PIS_adults_V1.4_03Dec21.pdf
- Adults
Editorial Notes
20/10/2023: Trial's existence confirmed by the National Institute for Health and Care Research (NIHR) (UK).
