A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Nicole Hamblett
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.
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 conce…
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
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 c…
Locations (68)
- The Children's Hospital Alabama, University of Alabama at BirminghamBirmingham, Alabama
- Phoenix Children's HospitalPhoenix, Arizona
- Tucson Cystic Fibrosis CenterTucson, Arizona
- University of Arkansas for Medical SciencesLittle Rock, Arkansas
- University of California San DiegoLa Jolla, California
- Childrens Hospital Los AngelesLos Angeles, California