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Rare CFTR Mutation Cell Collection Protocol (RARE)
NCT03161808 · View on ClinicalTrials.gov ↗
Study Summary
Over 1,900 mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein are implicated in causing Cystic Fibrosis (CF). Potential therapies that directly target defective CFTR are being evaluated in important clinical trials, but most target the most common CFTR mutation F508del. Many patients with rare CF mutations are not able to participate in those studies. The RARE study is specifically designed for people with CF caused by rare mutations. Eligible rare mutations are listed below: • CF patients who are heterozygous for pre-mature stop codons as noted below: i. one allele must be a F508del ii. the other allele must be a pre-mature stop codon mutation • CF Patients with other genotypes that require Study PI permission: i. CF patients with two mutations that are not eligible for Trikafta ii. CF patients homozygous or heterozygous (other allele must be F508del) for rare mutations of special interest (e.g., 711+3A-\>G, 2789+5G-\>A, 3272-26A-\>G, 3849+10kbC-\>T). Other rare mutations will be considered on a case by case basis This is a multi-site, specimen collection study. Investigators will collect blood, intestinal cells and nasal cells from each participant. Cells from these specimens will be used to test future CFTR modulators to see if they might work for people with study eligible rare mutations. Having cells to test in the lab is an important first step in identifying potential new therapies for people with these mutations.
Conditions Studied
Study Locations (1)
Alabama
- University of Alabama at Birmingham — Birmingham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 500 participants |
| Start Date | 2017-10-05 |
| Est. Completion | 2028-12-01 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03161808
The ClinicalTrials.gov registry entry for NCT03161808 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 500 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is George Solomon, which has 2 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Cystic Fibrosis appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03161808 reports 1 study location spanning 1 distinct geographic area — top geographies include Alabama. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03161808 about?
NCT03161808 is a clinical study titled "Rare CFTR Mutation Cell Collection Protocol (RARE)". Over 1,900 mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein are implicated in causing Cystic Fibrosis (CF). Potential therapies that directly target defective CFTR are being evaluated in important clinical trials, but most target the most common CFTR m...
What is the current status of trial NCT03161808?
This trial is currently recruiting. The enrollment target is 500 participants. The study started on 2017-10-05. Estimated completion is 2028-12-01.
What conditions does trial NCT03161808 study?
This clinical trial studies the following conditions: Cystic Fibrosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT03161808?
This trial is sponsored by George Solomon, which has 2 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03161808 being conducted?
This trial has 1 study location across Alabama. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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