Symdeko for Cystic Fibrosis
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop your current medications, but it excludes those taking medications with significant drug interactions with CFTR modulators. It's best to discuss your specific medications with the study team.
What data supports the effectiveness of the drug Symdeko for treating cystic fibrosis?
Research shows that the combination of tezacaftor and ivacaftor, which is part of Symdeko, is effective in improving lung function and quality of life in people with cystic fibrosis. It has a favorable safety profile and is particularly beneficial for those with specific genetic mutations related to cystic fibrosis.12345
Is Symdeko (tezacaftor/ivacaftor) safe for people with cystic fibrosis?
Symdeko (tezacaftor/ivacaftor) has been studied for safety in people with cystic fibrosis, especially those who had to stop another treatment due to breathing problems. It generally has a good safety profile, but some people may experience side effects like rash or headache, and liver function should be monitored.45678
How is the drug Symdeko different from other cystic fibrosis treatments?
Symdeko, which combines tezacaftor and ivacaftor, is unique because it targets the underlying cause of cystic fibrosis by helping the defective protein function better, specifically for those with certain genetic mutations. This approach is different from treatments that only manage symptoms, offering a more targeted therapy for improving lung function and overall health in eligible patients.5791011
What is the purpose of this trial?
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.
Eligibility Criteria
This trial is for individuals with Cystic Fibrosis, aged 6 or older, who have specific CFTR mutations that might respond to certain drugs not yet approved for their condition. They must be on a stable CF treatment and able to give consent. People can't join if they have severe liver or kidney disease, had an organ transplant, are currently in another drug study, smoked recently, or take medications that don't mix well with the trial drugs.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive off-label CFTR modulators such as Symdeko, Orkambi, or Ivacaftor based on their CFTR mutation response
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Symdeko
Symdeko is already approved in United States, Canada, European Union for the following indications:
- Cystic fibrosis in people ages 12 and older with certain mutations in the CFTR gene
- Cystic fibrosis in people with certain mutations in the CFTR gene
- Cystic fibrosis in people with certain mutations in the CFTR gene
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Alabama at Birmingham
Lead Sponsor