Effects of Trikafta on exercise and breathing in cystic fibrosis patients
Long-term Effects of Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) on Exertional Symptoms, Exercise Performance, Ventilatory Responses, and Body Composition in Adults With Cystic Fibrosis
This study tests how the drug Trikafta affects breathing and exercise ability in people with cystic fibrosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Ages | 19 Years and up |
| Sex | All |
| Sponsor | University of British Columbia Academic / other |
| Locations | 1 site (Vancouver, British Columbia) |
| Trial ID | NCT05279040 on ClinicalTrials.gov |
What this trial studies
This observational study investigates how the drug Trikafta affects shortness of breath and exercise performance in patients with cystic fibrosis (CF). It aims to evaluate the impact of this combination therapy on exercise capacity, which is a critical predictor of survival in CF. Participants will undergo standardized exercise tests to assess dyspnea and performance while on Trikafta. The study seeks to provide insights into the potential health improvements associated with this treatment.
Who should consider this trial
Good fit: Ideal candidates include adults aged 19 and older with a confirmed diagnosis of cystic fibrosis and at least one copy of the F508del mutation who are about to start Trikafta.
Not a fit: Patients with other significant diseases contributing to dyspnea or those with specific chronic infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management of exercise-related symptoms in cystic fibrosis patients, enhancing their quality of life.
How similar studies have performed: Previous studies on other CFTR modulator therapies have shown mixed results, making this approach both relevant and necessary for further exploration.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of CF and at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene * Plan to initiate Trikafta by the treating physician within 30 days of the enrolment visit * Aged 19 years or older * Stable clinical status based on clinical judgment of the treating physician * Forced Expiratory Volume in 1 second (FEV1.0) \< 90% predicted * Body mass index greater than 16 or less than 30 kg/m\^2 * Currently non-smoking or a past smoking history of less than 20 pack-years * Able to read and understand English * Fully vaccinated (at least 2 doses) for Covid-19 Exclusion Criteria: * A disease other than CF that could importantly contribute to dyspnea or exercise limitation * Chronic airway infection with Mycobacterium abscessus, Burkholderia cepacia complex, or other organisms with infection control implications based on the treating physicians * Contraindications to clinical exercise testing * Use of supplemental oxygen or desaturation less than 85% with exercise * Diagnosis of pneumothorax in the past 4 weeks * History of organ transplantation
Where this trial is running
Vancouver, British Columbia
- UBC Centre for Heart Lung Innovation, St. Paul's Hospital — Vancouver, British Columbia, Canada (Recruiting)
Study contacts
- Principal investigator: Jordan A Guenette, PhD — University of British Columbia
- Study coordinator: Satvir S Dhillon, MSc
- Email: satvir.dhillon@hli.ubc.ca
- Phone: 6048068835
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.