Rare Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are 17 years of age or older at the time of signing the consent form
- You have been diagnosed with cystic fibrosis (CF), confirmed by at least one of the following: a sweat chloride test result, a genetic test showing two known CF gene mutations, or a specific nasal electrical measurement test
- You have a specific type of CF gene mutation that the trial is currently focused on — at the start of the trial, this means having two copies of a particular type of gene error called a "premature stop codon" (confirm with trial site for current targets)
- You are able and willing to follow the study requirements and have signed a consent form (or have a legal representative who has done so)
- You are willing to travel to a regional study site if needed, for a cell collection procedure
Who may not be able to join:
- You have a health condition, physical abnormality, or abnormal blood test result that the study doctors believe could put you at risk during a research tissue sample (biopsy) from the rectum, or could affect the quality of the data
- You have significant problems with the tissue in the lower bowel or rectum area — such as severe hemorrhoids, abnormal blood vessels, bowel infection, or a history of radiation treatment to that area — that could make the biopsy risky
- Your blood test results show certain concerning values, such as low platelet count, low red blood cell levels, high white blood cell count, or problems with how your blood clots (confirm with trial site for specific values)
- You are pregnant (if you are a woman who could become pregnant, a pregnancy test will be done at the study visit)
- You are currently breastfeeding and would need sedation for the procedure
- You have been taking high-dose steroid tablets (over 20 mg per day) or other medications that significantly weaken the immune system for more than 14 days before the biopsy
- You have previously had an organ transplant
- You have taken blood-thinning medications (such as warfarin) or certain pain relievers that affect blood clotting (such as aspirin) within 7 days before the biopsy
- You are unwilling or unable to stop taking blood-thinning or clot-affecting medications for 7 days after the biopsy
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: 205-638-9568
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
The Number of samples collected from cystic fibrosis participants with rare CFTR mutations; The number of nasal cells collected; Number of Blood samples; Number of rectal samples collected
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.