Trikafta Exercise Study in Cystic Fibrosis

NCT05279040 | Recruiting

• 1 other identifier: H21-02125
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis; Lung Diseases

Keywords

Dyspnea; Exercise; Trikafta

Outcome Measures

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.

  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 (51)

• Change From Baseline Exercise Capacity (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.

  Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)

• 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.

  Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)

• 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.

  Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 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.

  Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)

• Change From Baseline Body Composition (Bone Mineral Density) Measurements (Prior to Trikafta Initiation to Full Dose) at 12 and 24 Months After Initiating Full Dose of Trikafta.

  Baseline (i.e., Visit 2), 12 months post Trikafta initiation (i.e., Visit 3), and 24 months post Trikafta initiation (i.e., Visit 4)

Study Arms (1)

Cystic Fibrosis Patients

Participants diagnosed with cystic fibrosis who will be initiating Trikafta treatment

Drug: Trikafta

Interventions

Trikafta DRUG

Elexacaftor/Tezacaftor/Ivacaftor combination therapy

Also known as: Elexacaftor/Tezacaftor/Ivacaftor

Cystic Fibrosis Patients

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The CF patients will be recruited from the St. Paul's Hospital UBC Adult CF Clinic.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of British Columbia: lead

Study Sites (1)

UBC Centre for Heart Lung Innovation, St. Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis; Lung Diseases; Dyspnea; Motor Activity

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Respiration Disorders; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Behavior

Study Officials

• Jordan A Guenette, PhD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Satvir S Dhillon, MSc

CONTACT

6048068835; satvir.dhillon@hli.ubc.ca

Jordan Guenette, PhD

CONTACT

16048068835; jordan.guenette@hli.ubc.ca

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Cardiopulmonary Exercise Physiology Laboratory

Study Record Dates

• First Submitted: November 8, 2021
• First Posted: March 15, 2022
• Study Start: November 10, 2021
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: March 26, 2025

Record last verified: 2025-03

Locations

CA (1)