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

Overview

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.

Primary outcome measures

• Change in ppFEV1 at 12 months [Time frame: 12 months]

Secondary outcome measures (6)

• Change in CFQ-R-RD at 12 months [Time frame: 12 months]
• Change in home spirometry ppFEV1 at 12 months [Time frame: 12 months]
• Change in LCI 2.5% at 12 months [Time frame: 12 months]
• Change in LCI 5% at 12 months [Time frame: 12 months]
• Absolute 12-month change in MCC Index [Time frame: 12 months]
• Relative 12-month change in MCC Index [Time frame: 12 months]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

United States · 68 centers

• The Children's Hospital Alabama, University of Alabama at Birmingham — Birmingham
• Phoenix Children's Hospital — Phoenix
• Tucson Cystic Fibrosis Center — Tucson
• University of Arkansas for Medical Sciences — Little Rock
• University of California San Diego — La Jolla
• Childrens Hospital Los Angeles — Los Angeles
• Stanford University Medical Center — Palo Alto
• University of California, San Francisco - Adult Center — San Francisco
• … and 60 more centers

Identifiers

NCT: NCT06504589 · REACH-OB-23

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗