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

NCT06504589 | Recruiting

• 1 other identifier: REACH-OB-23
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 68

Brief Summary

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.

Conditions

Cystic Fibrosis

Keywords

ineligible and/or not taking CFTR modulators; People with CF

Outcome Measures

Primary Outcomes (1)

• Change in ppFEV1 at 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 Outcomes (6)

• Change in CFQ-R-RD at 12 months

  12 months

• Change in home spirometry ppFEV1 at 12 months

  12 months

• Change in LCI 2.5% at 12 months

  12 months

• Change in LCI 5% at 12 months

  12 months

• Absolute 12-month change in MCC Index

  12 months

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Nicole Hamblett: lead
• Washington University School of Medicine: collaborator
• Florida State University: collaborator
• University of North Carolina, Chapel Hill: collaborator
• University of Toronto: collaborator
• Cystic Fibrosis Foundation: collaborator

Study Sites (68)

The Children's Hospital Alabama, University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Tucson Cystic Fibrosis Center

Tucson, Arizona, 85724, United States

RECRUITING

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

RECRUITING

University of California San Diego

La Jolla, California, 92093, United States

NOT YET RECRUITING

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Stanford University Medical Center

Palo Alto, California, 94304, United States

RECRUITING

University of California, San Francisco - Adult Center

San Francisco, California, 94143, United States

RECRUITING

University of California, San Francisco - Peds Center

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

National Jewish Health

Denver, Colorado, 80206, United States

RECRUITING

University of Florida

Gainesville, Florida, 32610, United States

RECRUITING

University of Miami

Miami, Florida, 33136, United States

RECRUITING

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

RECRUITING

Tampa General Hospital

Tampa, Florida, 33606, United States

RECRUITING

Children's Healthcare of Atlanta and Emory University

Atlanta, Georgia, 30329, United States

RECRUITING

Augusta University

Augusta, Georgia, 30912, United States

RECRUITING

Saint Luke's Cystic Fibrosis Center of Idaho

Boise, Idaho, 83702, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

University of Kentucky

Lexington, Kentucky, 40506, United States

RECRUITING

Tulane University

Metairie, Louisiana, 70001, United States

RECRUITING

John Hopkins Hospital

Baltimore, Maryland, 21287, United States

NOT YET RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, 01655, United States

RECRUITING

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, 48109, United States

RECRUITING

Wayne State University Harper University Hospital

Detroit, Michigan, 48201, United States

RECRUITING

Corewell Health Helen DeVos

Grand Rapids, Michigan, 49503, United States

RECRUITING

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, 55455, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Billings Clinic

Billings, Montana, 59101, United States

RECRUITING

University of Nebraska Medical Center

Omaha, Nebraska, 69198, United States

RECRUITING

Morristown Medical Center

Morristown, New Jersey, 07960, United States

RECRUITING

The Cystic Fibrosis Center of Western New York

Buffalo, New York, 14203, United States

NOT YET RECRUITING

Cohen Children's Medical Center of New York

New Hyde Park, New York, 11042, United States

RECRUITING

Columbia University Cystic Fibrosis Program

New York, New York, 10032, United States

RECRUITING

University of Rochester Medical Center Strong Memorial

Rochester, New York, 14642, United States

RECRUITING

New York Medical College at Westchester Medical Center

Valhalla, New York, 10595, United States

RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, 27157, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

RECRUITING

Cleveland Clinic Cystic Fibrosis Program

Cleveland, Ohio, 44195, United States

NOT YET RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Dayton Children's Hospital

Dayton, Ohio, 45404, United States

RECRUITING

Oregon Health & Sciences University

Portland, Oregon, 97239, United States

RECRUITING

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Prisma Health Children's Hospital - Midlands

Columbia, South Carolina, 29203, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States

RECRUITING

University of Texas Southwestern / Children's Health

Dallas, Texas, 75207, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

RECRUITING

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Adult Cystic Fibrosis Center at the University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

Inova L.J. Murphy Pediatric CF Program

Fairfax, Virginia, 22031, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

University of Washington Medical Center

Seattle, Washington, 98195, United States

RECRUITING

Providence Medical Group, Cystic Fibrosis Clinic

Spokane, Washington, 99204, United States

RECRUITING

West Virginia University - Morgantown

Morgantown, West Virginia, 26507, United States

RECRUITING

University of Wisconsin

Madison, Wisconsin, 53792, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Serum, plasma and white blood cells

MeSH Terms

Conditions

Cystic Fibrosis

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

• Deepika Polineni, MD, MPH

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

• Nicole Mayer-Hamblett, PhD

  Seattle Children's Hospital

  PRINCIPAL INVESTIGATOR

• Scott Donaldson, MD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

• Felix Ratjen, MD

  University of Toronto

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Olena Boyarska

CONTACT

206-884-1681; olena.boyarska@seattlechildrens.org

Anna Mead

CONTACT

206-884-7531; anna.mead@seattlechildrens.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics, Division of Pulmonary and Sleep Medicine, UW School of Medicine Adjunct Professor, Biostatistics, UW School of Medicine Co-Executive Director, CF Therapeutics Development Network

Study Record Dates

• First Submitted: June 27, 2024
• First Posted: July 17, 2024
• Study Start: September 18, 2024
• Primary Completion (Estimated): August 14, 2029
• Study Completion (Estimated): August 14, 2029
• Last Updated: October 1, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (68)