Cystic Fibrosis
Conditions
Keywords
ineligible and/or not taking CFTR modulators, People with CF
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.
Interventions
None listed
Sponsors
Washington University School of Medicine
Florida State University
University of North Carolina, Chapel Hill
University of Toronto
Cystic Fibrosis Foundation
Nicole Hamblett
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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. |
| 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. |
| 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. |
Countries
United States
Contacts
Primary ContactOlena Boyarska
Backup ContactAnna Mead
Outcome results
None listed