Clinical trial NCT06504589: A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Official title: A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cystic Fibrosis
Keywords: ineligible and/or not taking CFTR modulators
People with CF
Lead sponsor: Seattle Children's Hospital
Sponsor class: OTHER
Collaborators: name: Washington University School of Medicine
name: Florida State University
name: University of North Carolina, Chapel Hill
name: University of Toronto
name: Cystic Fibrosis Foundation
First posted: 2024-07-17
Enrollment: 400
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in ppFEV1 at 12 months
timeFrame: 12 months
Secondary outcomes:
measure: Change in CFQ-R-RD at 12 months
timeFrame: 12 months
measure: Change in home spirometry ppFEV1 at 12 months
timeFrame: 12 months
measure: Change in LCI 2.5% at 12 months
timeFrame: 12 months
measure: Change in LCI 5% at 12 months
timeFrame: 12 months
measure: Absolute 12-month change in MCC Index
timeFrame: 12 months
measure: Relative 12-month change in MCC Index
timeFrame: 12 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Children's Hospital Alabama, University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Tucson
country: United States
facility: Tucson Cystic Fibrosis Center
state: Arizona
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco - Adult Center
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco - Peds Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta and Emory University
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Boise
country: United States
facility: Saint Luke's Cystic Fibrosis Center of Idaho
state: Idaho
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: Metairie
country: United States
facility: Tulane University
state: Louisiana
city: Baltimore
country: United States
facility: John Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Memorial Health Care
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan, Michigan Medicine
state: Michigan
city: Detroit
country: United States
facility: Wayne State University Harper University Hospital
state: Michigan
city: Grand Rapids
country: United States
facility: Corewell Health Helen DeVos
state: Michigan
city: Minneapolis
country: United States
facility: The Minnesota Cystic Fibrosis Center
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Billings
country: United States
facility: Billings Clinic
state: Montana
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Morristown
country: United States
facility: Morristown Medical Center
state: New Jersey
city: Buffalo
country: United States
facility: The Cystic Fibrosis Center of Western New York
state: New York
city: Lake Success
country: United States
facility: Cohen Children's Medical Center of New York
state: New York
city: New York
country: United States
facility: Columbia University Cystic Fibrosis Program
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center Strong Memorial
state: New York
city: Valhalla
country: United States
facility: New York Medical College at Westchester Medical Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Winston-Salem
country: United States
facility: Atrium Health Wake Forest Baptist
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Cystic Fibrosis Program
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Sciences University
state: Oregon
city: Hershey
country: United States
facility: Hershey Medical Center Pennsylvania State University
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Columbia
country: United States
facility: Prisma Health Children's Hospital - Midlands
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Austin
country: United States
facility: Dell Children's Medical Center of Central Texas
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern / Children's Health
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Salt Lake City
country: United States
facility: Adult Cystic Fibrosis Center at the University of Utah
state: Utah
city: Fairfax
country: United States
facility: Inova L.J. Murphy Pediatric CF Program
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Spokane
country: United States
facility: Providence Medical Group, Cystic Fibrosis Clinic
state: Washington
city: Morgantown
country: United States
facility: West Virginia University - Morgantown
state: West Virginia
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
Study officials:
affiliation: Washington University School of Medicine
name: Deepika Polineni, MD, MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: Seattle Children's Hospital
name: Nicole Mayer-Hamblett, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of North Carolina, Chapel Hill
name: Scott Donaldson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Toronto
name: Felix Ratjen, MD
role: PRINCIPAL_INVESTIGATOR
References: