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

September 25, 2025 updated by: Nicole Hamblett

The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The goal of the REACH study is to collect research data, including health data and specimens, from people with CF who do not take CFTR modulators. This data may be used to inform CF research, help design CF clinical trials and support the development of new treatments for people with CF who do not take CFTR modulators.

Another goal of this study is to learn about research involvement for people with CF who do not take CFTR modulators, engage them in research, and give them an opportunity to learn about what is involved in participating in a CF research study.

Study Overview

Status

Recruiting

Conditions

Detailed Description

People with Cystic Fibrosis (pwCF) who are genetically ineligible and/or not taking cystic fibrosis transmembrane conductance regulator (CFTR) modulators currently face future health that is considerably different from the approximately 90% of pwCF in the United States who benefit from CFTR modulators. New treatments are being advanced for pwCF who are genetically ineligible or not taking CFTR modulators and these therapies will include both nucleic acid-based therapies (NABTs) to address the underlying defect in CFTR and symptomatic therapies aimed at targeting the symptoms of CF. A key concern for this limited and underserved patient population is being able to advance multiple therapeutic programs in parallel. To complete these studies, CF researchers will need to be able to reach this community effectively while also promoting the use of innovative trial designs.

The REACH Study is a prospective, longitudinal, observational research study to obtain research quality (i.e., monitored research) CF outcome data. Primary outcome endpoints of the Core study (collected across all study participants) are aligned with anticipated clinical trial outcome endpoints needed in overall development of therapies for the CF population unable to benefit from CFTR modulators. This study will also include sub-studies to obtain specialized measures which may help inform efficacy and safety evaluations of new therapies by providing CF control data. Finally, this study also seeks to assess research solicitation and research participation for the CF population that is modulator ineligible or not taking modulators. The observational data collected within this study may be used in characterizing this CF population, developing innovative trial designs, for comparison when evaluating new or experimental CF therapies, and/or in CF research.

Study Type

Observational

Enrollment (Estimated)

400

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • The Children's Hospital Alabama, University of Alabama at Birmingham
        • Contact:
    • Arizona
      • Phoenix, Arizona, United States, 85016
      • Tucson, Arizona, United States, 85724
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Contact:
    • California
      • La Jolla, California, United States, 92093
        • Not yet recruiting
        • University of California San Diego
        • Contact:
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Childrens Hospital Los Angeles
        • Contact:
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University Medical Center
        • Contact:
      • San Francisco, California, United States, 94158
        • Recruiting
        • University of California, San Francisco - Peds Center
        • Contact:
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California, San Francisco - Adult Center
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
      • Denver, Colorado, United States, 80206
        • Recruiting
        • National Jewish Health
        • Contact:
    • Florida
      • Gainesville, Florida, United States, 32610
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami
        • Contact:
          • Ylber (Ivan) Whitaker
          • Phone Number: 305-243-5545
          • Email: yiw2@miami.edu
      • Orlando, Florida, United States, 32803
        • Recruiting
        • Central Florida Pulmonary Group
        • Contact:
      • Tampa, Florida, United States, 33606
        • Recruiting
        • Tampa General Hospital
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Children's Healthcare of Atlanta and Emory University
        • Contact:
      • Augusta, Georgia, United States, 30912
        • Recruiting
        • Augusta University
        • Contact:
    • Idaho
      • Boise, Idaho, United States, 83702
        • Recruiting
        • Saint Luke's Cystic Fibrosis Center of Idaho
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Hospital for Children
        • Contact:
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Contact:
    • Kentucky
      • Lexington, Kentucky, United States, 40506
        • Recruiting
        • University of Kentucky
        • Contact:
        • Contact:
          • Phone Number: 859-218-4810
    • Louisiana
      • Metairie, Louisiana, United States, 70001
        • Recruiting
        • Tulane University
        • Contact:
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Not yet recruiting
        • John Hopkins Hospital
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
      • Boston, Massachusetts, United States, 02114
      • Worcester, Massachusetts, United States, 01655
        • Recruiting
        • University of Massachusetts Memorial Health Care
        • Contact:
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan, Michigan Medicine
        • Contact:
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Wayne State University Harper University Hospital
        • Contact:
      • Grand Rapids, Michigan, United States, 49503
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • The Minnesota Cystic Fibrosis Center
        • Contact:
          • CF Trials Contact University of Minnesota, Participant Contact
          • Email: cftrials@umn.edu
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
    • Montana
      • Billings, Montana, United States, 59101
    • Nebraska
      • Omaha, Nebraska, United States, 69198
        • Recruiting
        • University of Nebraska Medical Center
        • Contact:
    • New Jersey
      • Morristown, New Jersey, United States, 07960
    • New York
      • Buffalo, New York, United States, 14203
        • Not yet recruiting
        • The Cystic Fibrosis Center of Western New York
        • Contact:
      • New Hyde Park, New York, United States, 11042
        • Recruiting
        • Cohen Children's Medical Center of New York
        • Contact:
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Cystic Fibrosis Program
        • Contact:
      • Rochester, New York, United States, 14642
      • Valhalla, New York, United States, 10595
        • Recruiting
        • New York Medical College at Westchester Medical Center
        • Contact:
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Recruiting
        • University of North Carolina at Chapel Hill
        • Contact:
      • Winston-Salem, North Carolina, United States, 27157
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
      • Cleveland, Ohio, United States, 44195
        • Not yet recruiting
        • Cleveland Clinic Cystic Fibrosis Program
        • Contact:
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
        • Contact:
      • Columbus, Ohio, United States, 43205
      • Dayton, Ohio, United States, 45404
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Sciences University
        • Contact:
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Hershey Medical Center Pennsylvania State University
        • Contact:
      • Philadelphia, Pennsylvania, United States, 19104
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • University of Pittsburgh Medical Center
        • Contact:
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
        • Contact:
      • Columbia, South Carolina, United States, 29203
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
    • Texas
      • Austin, Texas, United States, 78723
        • Recruiting
        • Dell Children's Medical Center of Central Texas
        • Contact:
      • Dallas, Texas, United States, 75390
      • Dallas, Texas, United States, 75207
      • Fort Worth, Texas, United States, 76104
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • Adult Cystic Fibrosis Center at the University of Utah
        • Contact:
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Inova L.J. Murphy Pediatric CF Program
        • Contact:
      • Richmond, Virginia, United States, 23219
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • University of Washington Medical Center
        • Contact:
      • Seattle, Washington, United States, 98105
      • Spokane, Washington, United States, 99204
        • Recruiting
        • Providence Medical Group, Cystic Fibrosis Clinic
        • Contact:
    • West Virginia
      • Morgantown, West Virginia, United States, 26507
        • Recruiting
        • West Virginia University - Morgantown
        • Contact:
    • Wisconsin
      • Madison, Wisconsin, United States, 53792

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

People with CF who are ineligible and/or not taking an approved CFTR modulator and who are not receiving an investigational therapy.

Description

Consent

A. Written informed consent (and assent when applicable) obtained from participant or participant's legal guardian

B. Is willing and able to adhere to the study visit schedule and other protocol requirements

Demographics

A. ≥ 12 years of age at Visit 1

Medical History

A. For persons of child-bearing potential: must not be pregnant at Visit 1 or plan to get pregnant during the 12-month study period

Disease History

A. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:

  • Sweat chloride ≥ 60 mEq/liter by quantitative pilocarpine iontophoresis test (QPIT)
  • Two well-characterized disease-causing pathogenic variants in the CFTR gene

or

  • One well-characterized disease-causing mutation and a second CFTR variant (with variable or uncharacterized disease-causing potential) and sweat ≥ 30 mmol/liter with permission of the study sponsor-investigators

B. Clinically stable with no significant changes in health status within the 28 days prior to and including Visit 1

C. Does not have a history of lung transplantation

Concomitant Medications

A. Not genetically eligible for a CFTR modulator according to product label indications and/or No use of CFTR modulator for 28 days prior to Visit 1 with no intent to start or restart during the study period

B. No use of an investigational drug within 90 days prior to and including Visit 1

C. Not currently participating in an interventional drug or device trial. Participation in long-term safety follow-up studies (without redosing) and/or behavioral intervention trials is allowed.

D. No initiation of new chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin, Cayston®) within 28 days prior to and including Visit 1

E. No acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 28 days prior to and including Visit 1

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in ppFEV1 at 12 months
Time Frame: 12 months
Mean change (standard deviation) in percent predicted forced expiratory volume in 1 second (ppFEV1) as measured by in-clinic spirometry from baseline to 12 months post-baseline.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in CFQ-R-RD at 12 months
Time Frame: 12 months
Mean change (standard deviation) in the Cystic Fibrosis Question - Revised Respiratory Domain (CFQ-R-RD) from Baseline to 12 months post-baseline. Scores for the CFQ-R-RD range from 0 to 100.
12 months
Change in home spirometry ppFEV1 at 12 months
Time Frame: 12 months
Mean change (standard deviation) in percent predicted forced expiratory volume in 1 second (ppFEV1) as measured by home spirometry from baseline to 12 months post-baseline.
12 months
Change in LCI 2.5% at 12 months
Time Frame: 12 months
Mean change (standard deviation) in lung clearance index (LCI) calculated as 1/40th of starting end-tidal concentration of N2 (LCI 2.5%) as measured by multiple breath washout (MBW) from baseline to 12 months post-baseline.
12 months
Change in LCI 5% at 12 months
Time Frame: 12 months
Mean change (standard deviation) in lung clearance index (LCI) calculated as 1/20th of starting end-tidal concentration of N2 (LCI 5%) as measured by multiple breath washout (MBW) from baseline to 12 months post-baseline.
12 months
Absolute 12-month change in MCC Index
Time Frame: 12 months
Mean change (standard deviation) in mucociliary clearance (MCC) index as by the MCC procedure per gamma scintigraphy from baseline to 12 months post-baseline.
12 months
Relative 12-month change in MCC Index
Time Frame: 12 months
Mean change (standard deviation) in mucociliary clearance (MCC) index as by the MCC procedure per gamma scintigraphy from baseline to 12 months post-baseline divided by the baseline MCC index.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nicole Hamblett

Collaborators

Washington University School of Medicine
University of Toronto
University of North Carolina, Chapel Hill
Florida State University
Cystic Fibrosis Foundation

Investigators

  • Principal Investigator: Nicole Mayer-Hamblett, PhD, Seattle Children's Hospital
  • Principal Investigator: Deepika Polineni, MD, MPH, Washington University School of Medicine
  • Principal Investigator: Scott Donaldson, MD, University of North Carolina, Chapel Hill
  • Principal Investigator: Felix Ratjen, MD, University of Toronto

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 18, 2024

Primary Completion (Estimated)

August 14, 2029

Study Completion (Estimated)

August 14, 2029

Study Registration Dates

First Submitted

June 27, 2024

First Submitted That Met QC Criteria

July 14, 2024

First Posted (Actual)

July 17, 2024

Study Record Updates

Last Update Posted (Estimated)

October 1, 2025

Last Update Submitted That Met QC Criteria

September 25, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • REACH-OB-23

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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