Exploring new treatments for cystic fibrosis using existing medications

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates the use of off-label CFTR modulators to improve CFTR function in patients with specific cystic fibrosis mutations that are not currently approved for these drugs. Participants will receive treatments such as Symdeko, Orkambi, or Ivacaftor based on their mutation's response to these medications as determined by in vitro studies. The trial aims to assess the efficacy of these treatments in a population that may benefit from them, particularly focusing on patients aged 6 years and older. The study will exclude individuals with certain health conditions or those currently participating in other CFTR modulator studies.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Diagnosis of CF
* Age ≥6 y.o.
* CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators
* Informed Consent/Assent
* Stable CF pulmonary regimen

Exclusion Criteria:

* Exacerbation requiring antibiotic or steroids for \>28 days before trial entry
* Ongoing participation in a CFTR modulator study
* Active smoking in the past 6 months
* History of solid organ transplant
* Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)
* Any condition that precludes the patient from participation in the opinion of the investigator
* Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators

Where it's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting

Study contacts

• Study coordinator: Heather Hathorne, PhD
• Email: hhathorne@peds.uab.edu
• Phone: 205-638-9568

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Inhaled CFTR mRNA for cystic fibrosis in Chinese patients aged 12 and older

  This trial tests inhaled BMD003, a CFTR mRNA therapy, to see if it's safe and shows early signs of benefit in Chinese people 12 years and up with cystic fibrosis.

  Recruiting Early Phase 1 Peking Union Medical College Hospital · Beijing, Beijing Municipality · NCT07485543

• VX-272 dose escalation in healthy adults

  This will test single and multiple increasing doses of the experimental drug VX-272 in healthy adults to see how safe it is and how the body handles it.

  Recruiting Phase 1 Vertex Pharmaceuticals Incorporated · Tempe, Arizona · NCT07437105

• Prevalence of smoking, passive smoke exposure, and vaping among adults with cystic fibrosis

  This project will see how common smoking, exposure to secondhand smoke, and vaping are among adults with cystic fibrosis followed at the Strasbourg Adult CF Center.

  Recruiting University Hospital, Strasbourg, France · Strasbourg · NCT07342920

• Dose escalation of VX-581 to check safety and drug levels in healthy adults

  The trial will test increasing doses of VX-581 in healthy adults to see if it is safe and to measure how the body absorbs and clears the drug.

  Recruiting Phase 1 Vertex Pharmaceuticals Incorporated · Overland Park, Kansas · NCT07283770

• PALMIRE cohort: phenotyping inflammatory lung diseases to enable personalized care

  This project will collect clinical, imaging, and biological data from adults with asthma, COPD, bronchiectasis, cystic fibrosis, primary ciliary dyskinesia, or interstitial lung disease to try to identify disease subtypes and markers that could guide more personalized care.

  Recruiting CHU de Reims · Reims · NCT07274631

• Collecting sputum samples at home for cystic fibrosis patients

  This study is testing if collecting sputum samples at home can help people with cystic fibrosis better identify lung infections compared to traditional methods done at the clinic.

  Recruiting at 4 sites University of Pennsylvania · Denver, Colorado and 3 other locations · NCT06950892

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.