Canadian Observational Study Evaluating the Long-term IMPACT of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators on People With CF

Recruiting

• Conditions: Cystic Fibrosis
• Interventions: Drug: Trikafta

• Registration Number: NCT05200429
• Lead Sponsor: University of British Columbia

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-11-01
• Status Verified: 2022-01
• Study First Submitted: 2022-01-06
• Study First Submitted QC: 2022-01-06
• Last Update Submitted: 2022-01-06
• Study First Posted: 2022-01-20
• Last Update Posted: 2022-01-20
• Primary Completion: 2027-12-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Pre-Therapy Participants	Trikafta	All CF people with CF who do not have any known contraindications to CFTR modulator therapy and will be initiated on CFTR modulator therapy by their treating physician as part of clinical care are eligible and will be asked to participate in this study.

Primary Outcome Measures

Name	Time	Method
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
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

Trial Locations

Locations (2)

British Columbia Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada

St Paul's Hospital; 🇨🇦 Vancouver, British Columbia, Canada