A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Purpose
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.
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Over 12 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Recruiting Locations
Birmingham, Alabama 35233
More Details
- Status
- Recruiting
- Sponsor
- Nicole Hamblett
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.