Longitudinal assessment of clinical measurements in patients with cystic fibrosis in preparation for a clinical trial of CFTR gene therapy
| ISRCTN | ISRCTN08043947 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN08043947 |
| Secondary identifying numbers | 5028 |
- Submission date
- 12/05/2010
- Registration date
- 12/05/2010
- Last edited
- 23/05/2017
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Respiratory
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English Summary
Not provided at time of registration
Contact information
Prof Eric Alton
Scientific
Scientific
Emmanuel Kaye Building
1B Manresa Road
London
SW3 6LR
United Kingdom
Study information
| Study design | Multicentre non-randomised trial |
|---|---|
| Primary study design | Observational |
| Secondary study design | Multi-centre |
| Study setting(s) | Other |
| Study type | Treatment |
| Scientific title | Longitudinal assessment of clinical measurements in patients with cystic fibrosis in preparation for a clinical trial of CFTR gene therapy |
| Study 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. |
| Ethics approval(s) | ref: 07/Q0703/78 |
| 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 |
| Primary outcome measure | 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 study start date | 17/09/2007 |
| Overall study end date | 01/07/2011 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Not Specified |
| Sex | Not Specified |
| Target number of participants | Planned sample size: 200 |
| Participant inclusion criteria | Not provided at time of registration |
| 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
- England
- United Kingdom
Study participating centre
Emmanuel Kaye Building
London
SW3 6LR
United Kingdom
SW3 6LR
United Kingdom
Sponsor information
Imperial College London (UK)
University/education
University/education
ICCH Building
59 North Wharf Road
London
W2 1LA
England
United Kingdom
| Website | http://www3.imperial.ac.uk/ |
|---|---|
"ROR" |
https://ror.org/041kmwe10 |
Funders
Funder type
Charity
Cystic Fibrosis Trust (UK)
Private sector organisation / Other non-profit organizations
Private sector organisation / Other non-profit organizations
- Alternative name(s)
- Cystic Fibrosis, CF
- Location
- United Kingdom
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Editorial Notes
23/05/2017: No publications found in PubMed, verifying study status with principal investigator
21/05/2010: The anticipated end date of this trial was extended from 04/08/2008 to 01/07/2011.
