Condition category
Respiratory
Date applied
12/05/2010
Date assigned
12/05/2010
Last edited
09/09/2014
Prospective/Retrospective
Retrospectively 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

Prof Eric Alton

ORCID ID

Contact details

Emmanuel Kaye Building
1B Manresa Road
London
SW3 6LR
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

5028

Study information

Scientific title

Acronym

Run-in study

Study hypothesis

The UK Cystic Fibrosis Gene Therapy Consortium (Imperial College, University of Edinburgh and Oxford) is working towards a clinical trial of CFTR gene therapy. The aim of this trial is to allow us to assess, for the first time, whether introducing normal copies of the CF gene can lead to clinical improvements. This aim of this trial imposes certain requirements on trial design:
1. The treatment will have to be given repeatedly over a period of time of sufficient length to allow clinical parameters to change
2. The clinical parameters used to assess response must be robust (repeatable, reproducible and sensitive to change)

The study registered here is the first part of this programme and does not involve administering any gene therapy agent to patients. It is to allow us to gain insight into the natural history of the assays we have designed for the future trial, to choose those assays and patients most likely to allow us to assess change sensitively and to provide pre-trial longitudinal data on the trial patients.

On 21/05/2010 the anticipated end date of this trial was extended from 04/08/2008 to 01/07/2011.

Ethics approval

MREC approved (ref: 07/Q0703/78)

Study design

Multicentre non-randomised trial

Primary study design

Observational

Secondary study design

Multi-centre

Trial setting

Other

Trial type

Treatment

Patient information sheet

Condition

Topic: Respiratory, Generic Health Relevance and Cross Cutting Themes; Subtopic: Respiratory (all Subtopics), Generic Health Relevance (all Subtopics); Disease: Respiratory, Paediatrics

Intervention

200 CF patients aged 10 years and above will be recruited. All London patients will be recruited from the Royal Brompton Hospital adult and paediatric clinics. Adult patients in Scotland will be studied at the Western General Hospital, whilst the paediatric patients will be studied at the Royal Hospital for Sick Children. Scottish patients will be recruited from any Health Board in Scotland.

Over a period of approximately 2-3 years the patients will undergo five sets of measurements.

Study entry: registration only

Intervention type

Other

Phase

Not Applicable

Drug names

Primary outcome measures

To allow us to select assays for use in our future trial on the basis of their natural history

Secondary outcome measures

1. To provide informative data for future clinical trials
2. To provide clinical correlation

Overall trial start date

17/09/2007

Overall trial end date

01/07/2011

Reason abandoned

Eligibility

Participant inclusion criteria

Not provided at time of registration

Participant type

Patient

Age group

Not Specified

Gender

Not Specified

Target number of participants

Planned sample size: 200

Participant exclusion criteria

Not provided at time of registration

Recruitment start date

17/09/2007

Recruitment end date

01/07/2011

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Emmanuel Kaye Building
London
SW3 6LR
United Kingdom

Sponsor information

Organisation

Imperial College London (UK)

Sponsor details

ICCH Building
59 North Wharf Road
London
W2 1LA
United Kingdom

Sponsor type

University/education

Website

http://www3.imperial.ac.uk/

Funders

Funder type

Charity

Funder name

Cystic Fibrosis Trust (UK)

Alternative name(s)

Funding Body Type

private sector organisation

Funding Body Subtype

other non-profit

Location

United Kingdom

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