What is this study trying to find out about cystic fibrosis?

This study aims to collect health data from people with cystic fibrosis who do not take CFTR modulators to support research and development of new therapies. It focuses on understanding this group's health and participation in research. The lead sponsor is Nicole Hamblett. Participants can expect to contribute valuable information over time to help improve future treatments.

Who may be eligible for this cystic fibrosis clinical trial?

People aged 12 years or older with a confirmed cystic fibrosis diagnosis who are not currently taking CFTR modulators may be eligible. Both males and females can participate, except those who are pregnant or plan to become pregnant during the study. Participants must be stable with no recent lung transplant or investigational drug use. Please review eligibility criteria for more details.

Is there any cost to participate in this cystic fibrosis clinical trial?

Study-related care, including any procedures and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel or related costs.

Actively Recruiting

Age: 12Years +

All Genders

ID06504589

Research Study to Support New Cystic Fibrosis Treatments for People Not Using CFTR Modulators

Led by Nicole Hamblett · Updated on 2025-10-01

400

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

This research aims to advance treatments for people with cystic fibrosis CF who do not take cystic fibrosis transmembrane conductance regulator CFTR modulators. It focuses on those genetically ineligible or not using these modulators, a group with different health challenges compared to most people with CF. The study seeks to collect detailed health data and specimens to support new therapy development and improve clinical trial designs for this underserved population. The study is observational and will follow participants over time, collecting research-quality CF outcome data aligned with clinical trial endpoints important for developing new therapies. Sub-studies will gather specialized measures to help evaluate safety and effectiveness of future treatments. Researchers also aim to understand research participation and engagement in this community. Participants will provide health information and samples during scheduled visits over at least 12 months. Researchers will monitor lung function using measures like ppFEV1, CFQ-R-RD scores, lung clearance index LCI, and mucociliary clearance MCC indices. The study will help characterize this CF group and support the development of innovative trials. It will also assess research involvement and provide data for comparison with new therapies.

CONDITIONS

Brief Title

A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators

Who Can Participate

Age: 12Years +

All Genders

Eligibility Criteria

You may qualify if you...

Provide written informed consent (and assent if applicable)
Be 12 years of age or older at the first visit
Have a confirmed diagnosis of cystic fibrosis by clinical features and either a sweat chloride test or genetic testing
Be clinically stable with no significant health changes within 28 days before the first visit
Not have had a lung transplant previously
Not be genetically eligible for CFTR modulators or not using CFTR modulators for at least 28 days prior to the first visit with no plans to start during the study
Not have used investigational drugs within 90 days before the first visit
Not be currently enrolled in an interventional drug or device trial (behavioral or long-term safety follow-up studies allowed)
Not have started new chronic therapies within 28 days before the first visit
Not have used acute antibiotics or systemic corticosteroids for respiratory symptoms within 28 days before the first visit
For persons who can become pregnant: not be pregnant at the first visit or planning pregnancy during the 12-month study period

You will not qualify if you...

History of lung transplantation
Current use or planned start of CFTR modulator therapy during the study period
Use of investigational drugs within 90 days prior to first visit
Participation in interventional drug or device trials (except behavioral or long-term safety follow-up studies)
Recent start (within 28 days) of new chronic therapies such as ibuprofen, azithromycin, or inhaled antibiotics
Recent use (within 28 days) of acute antibiotics or systemic corticosteroids for respiratory symptoms
Pregnancy or planning pregnancy during the study period if of childbearing potential

Research Team

Olena Boyarska

Anna Mead

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here

Research Study to Support New Cystic Fibrosis Treatments for People Not Using CFTR Modulators

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria