MAGNIFY: A study on quality of life for Cystic Fibrosis Patients treated with Orkambi® or Symkevi® and their caregivers
Details
- Therapeutic approach
- Observational
- Trial status
- Recruitment complete Participating Centres
- Trials Tracker ID
- TT007206
- Last updated
- 14/07/2022
Quality of Life for Cystic Fibrosis Patients Treated With Orkambi® or Symkevi® and for Primary Caregivers in the United Kingdom
Study detailsTo improve medical care, it is important to collect information about the quality of life of people who receive medicines and also that of their parents.
Vertex Pharmaceuticals Inc., the company who developed Orkambi and Symkevi for the treatment of Cystic Fibrosis, is conducting this observational study at hospitals in the UK to collect additional information on the quality of life of 6-11 year olds receiving these medicines and that of their parents.
For those who are eligible to participate, after signing the data consent form, participants’ medical records relating to the history of their CF disease, including current and previous medications, will be reviewed along with demographic and CF genotype data from parents’ records. In addition, parents will be asked to complete electronic questionnaires on how their child is doing through a mobile study application (app) that has been developed for this study.
Participation in the study will be for one year with 5 sets of questionnaires to be completed every 3 months during routine hospital visits / check ins.
- Phase
- Not applicable
- Length of participation
- 1 year
- Recruitment target
- 100
- CF sponsor
- Vertex
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 6 to 11 years
- Including people
Patients with CF who are 6 to11 years of age, inclusive, at the time of enrolment who are eligible for Orkambi or Symkevi (includes those who already started treatment, those who have received a prescription for treatment with Orkambi or Symkevi, and those who will start treatment during the study).
Patients who already started treatment must still be on their initial treatment regimen
For Caregivers Inclusion Criteria:
Self-identified as a parent for a patient with CF who is eligible for Orkambi or Symkevi. This includes parents of patients who have already started treatment prior to enrolment and those who will start treatment during the study enrolment period as part of standard care.- Excluding people
Patients with CF who are or were previously enrolled in any interventional study will be excluded from this study.
Patients who have previously received CFTR modulator therapy or received Orkambi or Symkevi before November 2019