Trikafta Exercise Study in Cystic Fibrosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age19+
SponsorUniversity of British Columbia
About this trial
Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent drug treatment option known as Trikafta, which contains elexacaftor, tezacaftor, and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand the effects of Trikafta on these symptoms and to gain new insight into the potential health improvements in CF from using this treatment option.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial population
The CF patients will be recruited from the St. Paul's Hospital UBC Adult CF Clinic.
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
Trikafta
DrugElexacaftor/Tezacaftor/Ivacaftor combination therapy
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Cystic Fibrosis Patients1 intervention
Trial outcomes
Primary outcomes
1
Change in Iso-time Dyspnea Rating From Baseline (Visit 2) to Visit 3 and 4 During Constant-load Exercise Tests.
Dyspnea intensity and unpleasantness ratings measured using the Borg 0-10 category ratio scale. The intensity of the sensation is how strong or how much breathing sensation the participant feels, while the unpleasantness of the sensation is how bad or how distressed it makes the participant feel. On the Borg 0-10 category ratio scale range, "0" represents no dyspnea intensity and unpleasantness (i.e., better outcome), while "10" represents the most intense or unpleasant dyspnea (i.e., worse outcome). Borg dyspnea intensity and unpleasantness ratings taken at iso-time, defined as the maximum time achieved on constant-load exercise tests performed on visits 2, 3, and 4 will be used in the analysis.
Time frame
Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)Secondary outcomes
1
Change From Baseline Exercise Capacity (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.
Endurance time measures from the start of exercise to exercise cessation on constant-load exercise tests performed on visits 2, 3, and 4 will be used in the analysis.
Time frame
Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)2
Change From Baseline Body Composition (Body Fat Mass) Measurements (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.
Standard measures of body fat mass in units of grams (g) will be performed by dual-energy X-ray absorptiometry (DEXA) scanning using a Hologic Discovery QDR 4500 (Hologic Inc., Bedford, MA.). DEXA scans will be performed before the initiation of Trikafta treatment (Visit 2), 12 months after drug initiation (Visit 3), and 24 months after drug initiation (Visit 4).
Time frame
Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)3
Change From Baseline Body Composition (Lean Body Mass) Measurements (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.
Standard measures of lean body mass in units of grams (g) will be performed by dual-energy X-ray absorptiometry (DEXA) scanning using a Hologic Discovery QDR 4500 (Hologic Inc., Bedford, MA.). DEXA scans will be performed before the initiation of Trikafta treatment (Visit 2), 12 months after drug initiation (Visit 3), and 24 months after drug initiation (Visit 4).
Time frame
Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)4
Change From Baseline Body Composition (Bone Mineral Content) Measurements (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.
Standard measures of bone mineral content in units of grams (g) will be performed by dual-energy X-ray absorptiometry (DEXA) scanning using a Hologic Discovery QDR 4500 (Hologic Inc., Bedford, MA.). DEXA scans will be performed before the initiation of Trikafta treatment (Visit 2), 12 months after drug initiation (Visit 3), and 24 months after drug initiation (Visit 4).
Time frame
Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)Sponsors and contacts
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Source ClinicalTrials.gov