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

N/ARecruitingNCT05200429

University of British Columbia500 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

500

Conditions

Cystic Fibrosis

Interventions

TrikaftaDRUG

Highly Effective CFTR Modulator Therapy

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (2)

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

RECRUITING

St Paul's Hospital

Vancouver, British Columbia, Canada

RECRUITING

Outcomes

Primary Outcomes

To examine the long-term pulmonary and nutritional effects of CFTR modulators.

Time frame: 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.

Time frame: 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.

Time frame: 5 years

To establish the benefits of CFTR modulators compared to propensity-score matched historic CF populations followed in the Canadian CF registry.

Time frame: 5 years

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

Overview

Conditions

Interventions

Eligibility

Locations (2)

Outcomes

Primary Outcomes

Central Contacts