400 Participants Needed

Advancing Treatments for Cystic Fibrosis

Recruiting at 70 trial locations
AM
OB
Overseen ByOlena Boyarska
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to gather important health data from people with cystic fibrosis (CF) who do not use CFTR modulators, which are specific medications for CF. This information will help researchers design better treatments and trials for those who can't or don't take these modulators. The trial also seeks to engage participants in CF research and educate them about what it involves. It suits individuals diagnosed with CF who haven't recently used CFTR modulators or new therapies.

As an unphased trial, this study offers participants the chance to contribute to the foundational understanding of CF and influence future treatment options.

Why are researchers excited about this trial?

Researchers are excited about these treatments for cystic fibrosis (CF) because they aim to help people who can't benefit from current modulators. Unlike existing CF treatments that work by improving the function of faulty CFTR proteins, these new therapies focus on bypassing the need for CFTR modulators altogether. They target the underlying genetic causes of CF, offering hope for patients who previously had limited options. This approach could significantly broaden the reach of effective CF therapies, potentially improving quality of life for many.

Who Is on the Research Team?

SD

Scott Donaldson, MD

Principal Investigator

University of North Carolina, Chapel Hill

FR

Felix Ratjen, MD

Principal Investigator

University of Toronto

DP

Deepika Polineni, MD, MPH

Principal Investigator

Washington University School of Medicine

NM

Nicole Mayer-Hamblett, PhD

Principal Investigator

Seattle Children's Hospital

Are You a Good Fit for This Trial?

Inclusion Criteria

I agree to participate in the clinical trial.
I have given my written consent to participate.
I can follow the study's schedule and requirements.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Data Collection

Participants' health data and specimens are collected for research purposes

12 months
Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after data collection

4 weeks

What Are the Treatments Tested in This Trial?

Find a Clinic Near You

Who Is Running the Clinical Trial?

Nicole Hamblett

Lead Sponsor

Trials
7
Recruited
7,900+

University of North Carolina, Chapel Hill

Collaborator

Trials
1,588
Recruited
4,364,000+

Washington University School of Medicine

Collaborator

Trials
2,027
Recruited
2,353,000+

University of Toronto

Collaborator

Trials
739
Recruited
1,125,000+

Cystic Fibrosis Foundation

Collaborator

Trials
199
Recruited
37,800+

Florida State University

Collaborator

Trials
234
Recruited
41,100+