Condition category
Genetic Diseases
Date applied
30/09/2015
Date assigned
30/09/2015
Last edited
29/06/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Plain English Summary under review.

Trial website

Contact information

Type

Scientific

Primary contact

Mr Stephen Dando

ORCID ID

Contact details

Charles Dent Metabolic Unit
Queen Square
London
WC1N 3BG
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

19653

Study information

Scientific title

A pilot study to investigate the effect of aerobic exercise training in adults with Pompe disease

Acronym

Study hypothesis

The aim of this study is to investigate the effects of home based aerobic exercises on muscle and respiratory function, mobility and strength. The effects of cycling while receiving ERT will also be investigated.

Ethics approval

15/LO/0952

Study design

Non-randomised; Interventional; Design type: Treatment

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Other

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Topic: Metabolic and endocrine disorders, Musculoskeletal disorders; Subtopic: Metabolic and Endocrine (all Subtopics), Musculoskeletal (all Subtopics); Disease: Metabolic & Endocrine (not diabetes)

Intervention

Exercise, Cycling during Hospital based infusion. Participants will complete the initial, 3 month and final assessments and exercise testing at designated specialist centers in the UK.

Intervention type

Other

Phase

Drug names

Primary outcome measures

Walking ability measured using a 6 minute walk test (6MWT) which will be recorded at baseline, 3 months and 6 months.

Secondary outcome measures

N/A

Overall trial start date

16/09/2015

Overall trial end date

16/09/2016

Reason abandoned

Eligibility

Participant inclusion criteria

1. Aged between 16 and 70 years
2. Genetically proven diagnosis of late-onset Pompe disease
3. Attending Lysosome Centres in England
4. Can complete >50m in 6 minute walk test
5. Can transfer safely and independently on and off cycle ergometer
6. Can participate in all objective testing
7. Willing and able to attend specialist treatment centres
8. Willing to receive exercise testing and supervision in their own home

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned Sample Size: 16; UK Sample Size: 16

Participant exclusion criteria

1. Have been receiving Enzyme Replacement Therapy for less than 12 months
2. Able to walk more then 550m
3. Dependent on invasive ventilation during the day
4. Have cardiac issues for which exercise would not be recommended
5. Any current or previous orthopaedic medical condition for which exercise is contraindicated

Recruitment start date

16/09/2015

Recruitment end date

16/09/2016

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Charles Dent Metabolic Unit
University College London Hospital Queen Square
London
WC1N 3BG
United Kingdom

Sponsor information

Organisation

UCLH NHS Trust

Sponsor details

University College London
Gower Street
London
WC1E 6BT
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Industry

Funder name

Genzyme Therapeutics

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Aim to publish the results in an appropriate peer-reviewed journal.

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes