Recruiting
Early Phase 1

CFTR Modulators

Sponsor:

George Solomon

Code:

NCT03587961

Conditions

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

Symdeko

Study Details

Brief summary:

The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

Conditions

Cystic Fibrosis

Study ID

NCT03587961

Start date

Aug 1, 2019

Status verified date

Jan, 2026

Completion date

Sep 1, 2027

Anticipated

Primary completion date

Jan 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

20 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Symdeko

Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern

experimental: Ivacaftor

Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.

experimental: Orkambi

Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern

Interventions

Symdeko

explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.

Primary outcome measure

  • spirometry [ Time Frame: 32 weeks ]

Central Contacts and Locations

Central contacts

Heather Hathorne, PhD

205-638-9568hhathorne@peds.uab.edu

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Heather Hathorne, PhD

205-638-9568hhathorne@peds.uab.edu

Ginger Reeves, BS

205-638-5970greeves@peds.uab.edu

Principal Investigator:

George Solomon, MD

More Information

Sponsor

George Solomon

Last update posted

Feb 2, 2026

Last verified

Jan, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by George Solomon on 2026-02-02.