VX14-770-116: Ph IV - Observational Study for CF Patients
Research type
Research Study
Full title
Observational Study of Outcomes in Cystic Fibrosis Patients with Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
IRAS ID
162187
Contact name
Nicholas J. Simmonds
Contact email
Sponsor organisation
Vertex Pharmaceuticals
Research summary
This is a multi-site, multi-national observational study for Cystic Fibrosis (CF) patients who have certain cell mutations (any of the following CFTR G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).
The study will be conducted in Europe, will last 48 months and comprise of about 90 patients from the age of 6 years.
There will be no study drug or any other medical procedure involved and study doctors will manage patients in accordance with their medical judgment and continuation of the patient's existing treatment.
However it will involve the taking of a known drug treatment for CF, Kalydeco, which the patient may or may not have been prescribed before entering the study.
The study does include retrospective data collection (to ensure that for each patient, data is collected for a minimum of 12 months before the start of Kalydeco treatment) and intended future or prospective data collection.
Prospective data collection will begin at enrollment*.
After enrollment, data will be collected at 3-month intervals for 48 months and Quality of Life (HRQoL) data, via questionnaire, will be collected prospectively at 6-month intervals over the same timeframe.
HRQoL assessments will also be performed at a date when Kalydeco treatment is first started and when there is a coinciding clinic visit (even when that visit falls outside the normal 6-month interval) and will be completed before any other assessments when clinic visits occur.
All other data will be taken from the patients’ medical records.
All data collected will be recorded.
*A patient will be considered as enrolled in the study when they sign the informed consent form or, if applicable, assent form.
REC name
London - Brent Research Ethics Committee
REC reference
14/LO/1580
Date of REC Opinion
9 Oct 2014
REC opinion
Favourable Opinion