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

Recruiting

• Conditions: Cystic Fibrosis

• Registration Number: NCT06504589
• Lead Sponsor: Nicole Hamblett

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.

Study Timeline

• Study First Submitted: 2024-06-27
• Status Verified: 2024-07
• Study First Submitted QC: 2024-07-14
• Study First Posted: 2024-07-17
• Study Start: 2024-09-18
• Last Update Submitted: 2025-03-24
• Last Update Posted: 2025-03-27
• Primary Completion: 2029-08-14
• Study Completion: 2029-08-14

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 400

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in ppFEV1 at 12 months	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.

Secondary Outcome Measures

Name	Time	Method
Change in LCI 5% at 12 months	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.
Change in CFQ-R-RD at 12 months	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.
Change in LCI 2.5% at 12 months	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.
Absolute 12-month change in MCC Index	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.
Change in home spirometry ppFEV1 at 12 months	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.
Relative 12-month change in MCC Index	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.

Trial Locations

Locations (68)

The Children's Hospital Alabama, University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Tucson Cystic Fibrosis Center; 🇺🇸 Tucson, Arizona, United States

University of Arkansas for Medical Sciences; 🇺🇸 Little Rock, Arkansas, United States

University of California San Diego; 🇺🇸 La Jolla, California, United States

Childrens Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

Stanford University Medical Center; 🇺🇸 Palo Alto, California, United States

University of California, San Francisco - Adult Center; 🇺🇸 San Francisco, California, United States

University of California, San Francisco - Peds Center; 🇺🇸 San Francisco, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

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