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

NCT05200429 | Recruiting

• 1 other identifier: H21-01707
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 2

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.

Conditions

Cystic Fibrosis

Keywords

Trikafta; Cystic Fibrosis; CFTR Modulator Therapy; Respiratory Disease

Outcome Measures

Primary Outcomes (4)

• 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

• 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

Study Arms (1)

Pre-Therapy Participants

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.

Drug: Trikafta

Interventions

Trikafta DRUG

Highly Effective CFTR Modulator Therapy

Also known as: Elexacaftor/Tezacaftor/Ivacaftor

Pre-Therapy Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All 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

You may qualify if:

• 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

You may not qualify if:

• Known contraindications to CFTR modulator therapy

Sponsors & Collaborators

• University of British Columbia: lead

Study Sites (2)

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada

RECRUITING

St Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Urine, Stool and Serum

MeSH Terms

Conditions

Cystic Fibrosis; Respiration Disorders

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jonathan Rayment, MDCM

  University of British Columbia

  PRINCIPAL INVESTIGATOR

• Bradley Quon, MD

  University of British Columbia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alam Lakhani

CONTACT

604-875-2345; alam.lakhani@cw.bc.ca

Britney Ha

CONTACT

604-875-2345; britney.ha@cw.bc.ca

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: January 6, 2022
• First Posted: January 20, 2022
• Study Start: November 1, 2021
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: January 20, 2022

Record last verified: 2022-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2)