Condition category
Nervous System Diseases
Date applied
12/06/2014
Date assigned
12/06/2014
Last edited
27/06/2014
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Mrs Chin Maguire

ORCID ID

Contact details

Health Services Research
School of Health & related Research (ScHARR) Regents Court
30 Regent Street
Sheffield
S1 4DA
United Kingdom
c.maguire@sheffield.ac.uk

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

16831

Study information

Scientific title

Acronym

Hydrotherapy for Duchenne Muscular Dystrophy (DMD)

Study hypothesis

Duchenne muscular dystrophy (DMD) is a rare disease mainly affecting boys. DMD causes muscle cells to gradually break down so that with time, a patient’s muscles become weak to the point where they are unable to conduct many of the activities they used to. There is no cure for this disease, but doctors and physiotherapists try to slow down its progression and the development of complications by prescribing steroids and a physical management programme. Clinical experience shows that physical activity helps to maintain functional abilities. Mostly physical management programmes are done on dry land. However they can also be performed in warm water, under supervision by a physiotherapist; this is known as 'hydrotherapy' or 'aquatic therapy'. Hydrotherapy enables affected people to perform exercises which may not be possible on land due to the support provided by the water. Additionally the activity is seen as fun for them and their carers. Despite this, hydrotherapy is difficult to access in many places in the UK. Many NHS trusts do not fund it or have not got the facilities to offer it.
While we know people with DMD value hydrotherapy, we are not sure whether it really adds anything to land-based exercises alone in terms of helping with walking and other daily activities. We are therefore undertaking a small scale pilot study to help decide if a larger scale trial would be feasible and if so, how we should best conduct it. We plan to recruit 40 boys with DMD in the UK within 4 to 6 sites. Equal numbers will be allocated at random to either receive hydrotherapy plus land based physiotherapy (active intervention group) or land based physiotherapy alone (control group). Study participants will be assessed for key outcome measures at 3 time points: consent and screen 1 visit; baseline visit; 26 week visit. We will collect information on a number of outcomes relating to the feasibility of conducting the trial which will include interviews with participants.

Ethics approval

14/EE/0204

Study design

Randomised; Interventional; Design type: Treatment

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Other

Trial type

Treatment

Patient information sheet

Condition

Topic: Children, Genetics; Subtopic: All Diagnoses, Genetics Research and Congenital Disorders (all subtopics); Disease: Genetics Research and Congenital Disorders, All Diseases

Intervention

Primary Intervention: Participants will be allocated on a ratio of 1:1 using simple randomisation with permuted blinded block size to:
1. Control group to receive optimised land-based exercises (as defined by local community physiotherapy services and recorded by trial team) (n=20);
2. The intervention group will receive the same plus hydrotherapy (30 min, twice weekly, for 6 months: active assisted and/or passive stretching regime; simulated or real functional activities; sub-maximal exercise) (n=20).

Intervention type

Other

Phase

Not Applicable

Drug names

Primary outcome measures

Primary Outcome; Timepoint(s): Determining the feasibility of recruitment to the main trial is the primary objective of the study (

Secondary outcome measures

Not provided at time of registration

Overall trial start date

01/12/2014

Overall trial end date

30/05/2015

Reason abandoned

Eligibility

Participant inclusion criteria

1. Genetically or biopsy confirmed DMD
2. Age 7-16 years
3. Established on glucocorticosteroids
4. North Star Ambulatory Assessment score 8-34 (stable over 4 weeks)
5. Able to complete 10 metre walk (6 minute walk test)

Participant type

Patient

Age group

Child

Gender

Both

Target number of participants

Planned Sample Size: 40; UK Sample Size: 40

Participant exclusion criteria

1.More than a 20% variation between baseline North Star Ambulatory Assessment scores
2.Unable to commit to the programme of twice weekly hydrotherapy for 6 months
3.Any absolute contraindications or precautions to hydrotherapy

Recruitment start date

01/12/2014

Recruitment end date

30/05/2015

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Health Services Research, School of Health & related Research (ScHARR) Regents Court
Sheffield
S1 4DA
United Kingdom

Sponsor information

Organisation

Sheffield Children's Hospital (UK)

Sponsor details

Western Bank
Sheffield
S10 2TH
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC) (UK)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes