Exploring new treatments for cystic fibrosis using existing medications
Personalized Theratyping Trial
This study is testing if existing cystic fibrosis medications can help improve lung function in patients with specific gene mutations who are at least 6 years old.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | University of Alabama at Birmingham Academic / other |
| Locations | 1 site (Birmingham, Alabama) |
| Trial ID | NCT03587961 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are cystic fibrosis patients aged 6 years and older with specific CFTR mutations that may respond to off-label CFTR modulators.
Not a fit: Patients who have severe health conditions that preclude the use of CFTR modulators or those currently involved in other CFTR modulator studies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide new treatment options for cystic fibrosis patients with specific mutations, potentially improving their quality of life and health outcomes.
How similar studies have performed: Other studies have shown promise in using CFTR modulators for various mutations, but this specific approach to off-label use is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Birmingham, Alabama
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.