A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
ConditionCystic Fibrosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12+
SponsorNicole Hamblett
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Sweat chloride ≥ 60 mEq/liter by quantitative pilocarpine iontophoresis test (QPIT)
Two well-characterized disease-causing pathogenic variants in the CFTR gene
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
Disqualifiers
None
Trial population
People with CF who are ineligible and/or not taking an approved CFTR modulator and who are not receiving an investigational therapy.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
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.
Time frame
12 monthsSecondary outcomes
1
Change in CFQ-R-RD at 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.
Time frame
12 months2
Change in home spirometry ppFEV1 at 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.
Time frame
12 months3
Change in LCI 2.5% at 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.
Time frame
12 months4
Change in LCI 5% at 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.
Time frame
12 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Nicole Hamblett
Lead sponsor
Seattle Children's Hospital
Sponsor institution
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
Source ClinicalTrials.gov