Cystic Fibrosis
Conditions
Keywords
Trikafta, Cystic Fibrosis, CFTR Modulator Therapy, Respiratory Disease
Brief summary
This observational study intends to investigate health trends and data in cystic fibrosis patients all across Canada that are receiving modulator treatment so researchers can determine if CFTR treatments are effective over a long period of time and if so, which treatments work best for each individual. The study will collect clinical data from routine standard of care, patient reported outcomes via survey data and samples for a biobank.
Interventions
DRUGTrikafta
Highly Effective CFTR Modulator Therapy
Sponsors
University of British Columbia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Prescribed a CFTR modulator therapy and planning to commence modulator therapy within 30 days OR planning to switch modulator therapies within 30 days * Participates in the Canadian Cystic Fibrosis Registry (CCFR) * Informed consent by participant, or parent/legal guardian or assent
Exclusion criteria
* Known contraindications to CFTR modulator therapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To examine the long-term pulmonary and nutritional effects of CFTR modulators. | 5 years |
| To assess the long-term impact of CFTR modulators on patient-reported outcome measures including quality of life, disease burden, and physical activity. | 5 years |
| To establish a bio-repository to enable further investigations of the effectiveness of CFTR modulator therapies on biological markers and predictors of response. | 5 years |
| To establish the benefits of CFTR modulators compared to propensity-score matched historic CF populations followed in the Canadian CF registry. | 5 years |
Countries
Canada
Contacts
Primary ContactAlam Lakhani
Backup ContactBritney Ha
Outcome results
None listed