A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
Don Sanders, MD
Primary Investigator
Brief description of study
What is the purpose of this study?
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 of study
What will happen during the study?
- Participants will be seen at study sites for research visits to include spirometry, patient reported outcomes (PROs), and blood collections on Day 0, Day 90 (3 months), Day 180 (6 months), and Day 360 (12 months).
- Participants will complete home spirometry and electronic PROs (ePROs) monthly throughout the 12-month
study period. - A detailed medical history and additional data from any clinical visits occurring during the 12-month study will be extracted from the medical record and recorded in the study database.
- Participants at selected sites will participate in additional sub-studies. These will include multiple breath washout (MBW) measures of lung clearance index (LCI), mucociliary clearance (MCC) scans, and optional collection of human peripheral blood mononuclear cells (hPBMCs).
- Participants’ duration of participation is up to 12 months. Participants will have an option to re-enroll
into the study a minimum of 12 months after their last study visit or contact.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Cystic Fibrosis, CF, Riley
-
Age: 12 years or above
-
Gender: All
Inclusion criteria:
A. ≥ 12 years of age at Visit 1</p>
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</p>
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</p>
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</p>
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</p>
