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 Sex
- 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 4049979, Alabama 4829764 35233
Phoenix 5308655, Arizona 5551752 85016
Tucson 5318313, Arizona 5551752 85724
Los Angeles 5368361, California 5332921 90027
San Francisco 5391959, California 5332921 94158
Aurora 5412347, Colorado 5417618 80045
Boise 5586437, Idaho 5596512 83702
Indianapolis 4259418, Indiana 4921868 46202
Kansas City 4273837, Kansas 4273857 66160
Lexington 4297983, Kentucky 6254925 40506
Boston 4930956, Massachusetts 6254926 02114
Ann Arbor 4984247, Michigan 5001836 48109
Grand Rapids 4994358, Michigan 5001836 49503
Minneapolis 5037649, Minnesota 5037779 55455
Billings 5640350, Montana 5667009 59101
Morristown 5101427, New Jersey 5101760 07960
Rochester 5134086, New York 5128638 14642
Cleveland 5150529, Ohio 5165418 44106
Dayton 4509884, Ohio 5165418 45404
Portland 5746545, Oregon 5744337 97239
Hershey 5193342, Pennsylvania 6254927 17033
Philadelphia 4560349, Pennsylvania 6254927 19104
Philadelphia 4560349, Pennsylvania 6254927 19104
Charleston 4574324, South Carolina 4597040 29425
Austin 4671654, Texas 4736286 78723
Fort Worth 4691930, Texas 4736286 76104
Houston 4699066, Texas 4736286 77030
Salt Lake City 5780993, Utah 5549030 84132
Seattle 5809844, Washington 5815135 98105
Seattle 5809844, Washington 5815135 98195
Spokane 5811696, Washington 5815135 99204
Madison 5261457, Wisconsin 5279468 53792
More Details
- NCT ID
- NCT06504589
- 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.