BridgeMD
New treatment option for Cystic Fibrosis
Official title Personalized Theratyping Trial
ClinicalTrials.gov ID: NCT03587961
What this study is testing
What is Symdeko?
Symdeko is an investigational medicine, being studied as a potential treatment for cystic fibrosis.
Also referred to as Orkambi, Ivacaftor.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.