Actively Recruiting
A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Led by Nicole Hamblett · Updated on 2025-10-01
400
Participants Needed
68
Research Sites
N/A
Total Duration
On this page
Sponsors
N
Nicole Hamblett
Lead Sponsor
W
Washington University School of Medicine
Collaborating Sponsor
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
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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 months
Participants are observed over time to collect research-quality cystic fibrosis outcome data to inform future therapy development.
Regular visits throughout the 12-month period as determined by study protocol
Trial Site Locations
Total: 68 locations
1
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
3
Tucson Cystic Fibrosis Center
Tucson, Arizona, United States, 85724
Actively Recruiting
4
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
Actively Recruiting
5
University of California San Diego
La Jolla, California, United States, 92093
Not Yet Recruiting
6
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
7
Stanford University Medical Center
Palo Alto, California, United States, 94304
Actively Recruiting
8
University of California, San Francisco - Adult Center
San Francisco, California, United States, 94143
Actively Recruiting
9
University of California, San Francisco - Peds Center
San Francisco, California, United States, 94158
Actively Recruiting
10
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
11
National Jewish Health
Denver, Colorado, United States, 80206
Actively Recruiting
12
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
13
University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
14
Central Florida Pulmonary Group
Orlando, Florida, United States, 32803
Actively Recruiting
15
Tampa General Hospital
Tampa, Florida, United States, 33606
Actively Recruiting
16
Children's Healthcare of Atlanta and Emory University
Atlanta, Georgia, United States, 30329
Actively Recruiting
17
Augusta University
Augusta, Georgia, United States, 30912
Actively Recruiting
18
Saint Luke's Cystic Fibrosis Center of Idaho
Boise, Idaho, United States, 83702
Actively Recruiting
19
Northwestern University
Chicago, Illinois, United States, 60611
Actively Recruiting
20
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Actively Recruiting
21
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
22
University of Kentucky
Lexington, Kentucky, United States, 40506
Actively Recruiting
23
Tulane University
Metairie, Louisiana, United States, 70001
Actively Recruiting
24
John Hopkins Hospital
Baltimore, Maryland, United States, 21287
Not Yet Recruiting
25
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
26
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
27
University of Massachusetts Memorial Health Care
Worcester, Massachusetts, United States, 01655
Actively Recruiting
28
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
29
Wayne State University Harper University Hospital
Detroit, Michigan, United States, 48201
Actively Recruiting
30
Corewell Health Helen DeVos
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
31
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
32
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
33
Billings Clinic
Billings, Montana, United States, 59101
Actively Recruiting
34
University of Nebraska Medical Center
Omaha, Nebraska, United States, 69198
Actively Recruiting
35
Morristown Medical Center
Morristown, New Jersey, United States, 07960
Actively Recruiting
36
The Cystic Fibrosis Center of Western New York
Buffalo, New York, United States, 14203
Not Yet Recruiting
37
Cohen Children's Medical Center of New York
New Hyde Park, New York, United States, 11042
Actively Recruiting
38
Columbia University Cystic Fibrosis Program
New York, New York, United States, 10032
Actively Recruiting
39
University of Rochester Medical Center Strong Memorial
Rochester, New York, United States, 14642
Actively Recruiting
40
New York Medical College at Westchester Medical Center
Valhalla, New York, United States, 10595
Actively Recruiting
41
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
42
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States, 27157
Actively Recruiting
43
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
44
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Actively Recruiting
45
Cleveland Clinic Cystic Fibrosis Program
Cleveland, Ohio, United States, 44195
Not Yet Recruiting
46
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
47
Dayton Children's Hospital
Dayton, Ohio, United States, 45404
Actively Recruiting
48
Oregon Health & Sciences University
Portland, Oregon, United States, 97239
Actively Recruiting
49
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
50
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
51
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
52
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
53
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
54
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, United States, 29203
Actively Recruiting
55
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
56
Dell Children's Medical Center of Central Texas
Austin, Texas, United States, 78723
Actively Recruiting
57
University of Texas Southwestern / Children's Health
Dallas, Texas, United States, 75207
Actively Recruiting
58
University of Texas Southwestern
Dallas, Texas, United States, 75390
Actively Recruiting
59
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
60
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
61
Adult Cystic Fibrosis Center at the University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
62
Inova L.J. Murphy Pediatric CF Program
Fairfax, Virginia, United States, 22031
Actively Recruiting
63
Virginia Commonwealth University
Richmond, Virginia, United States, 23219
Actively Recruiting
64
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
65
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
66
Providence Medical Group, Cystic Fibrosis Clinic
Spokane, Washington, United States, 99204
Actively Recruiting
67
West Virginia University - Morgantown
Morgantown, West Virginia, United States, 26507
Actively Recruiting
68
University of Wisconsin
Madison, Wisconsin, United States, 53792
Actively Recruiting
Research Team
O
Olena Boyarska
A
Anna Mead
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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