Symdeko for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to explore how certain drugs might help individuals with specific genetic mutations linked to cystic fibrosis (CF). Researchers are testing treatments such as Symdeko (a combination of tezacaftor and ivacaftor), Ivacaftor, and Orkambi to assess their impact on the function of CFTR, a protein involved in CF. Individuals with cystic fibrosis and a mutation that might respond to these drugs could be suitable candidates, particularly if they have managed the condition without recent severe flare-ups. As a Phase 1 trial, the research focuses on understanding how the treatment works in participants.
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.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Previous studies have used the medications Symdeko, Ivacaftor, and Orkambi to treat certain types of cystic fibrosis (CF). Symdeko has been tested in over 1,000 patients and is generally well-tolerated, with most people not experiencing serious side effects. It is approved for patients aged 6 and older with specific gene mutations. Ivacaftor aids CF patients with a different type of mutation, and research shows it improves lung function and is safe for many. Orkambi combines two drugs to assist patients with two copies of a specific mutation. Studies in both young children and older patients have shown it is generally safe.
These treatments have been tested in various patient groups, and results indicate they are mostly well-tolerated. Prospective trial participants should remember that while side effects can occur, they are often manageable.12345Why are researchers excited about this trial's treatments?
Researchers are excited about these treatments for cystic fibrosis because they offer targeted approaches based on specific genetic mutations. Symdeko and Orkambi are both CFTR correctors, which help correct the malfunctioning protein caused by cystic fibrosis mutations. Ivacaftor, on the other hand, is a potentiator that enhances the function of the CFTR protein, especially for mutations similar to the wild type. Unlike standard treatments that may not account for individual genetic differences, these treatments are tailored to the patient's specific mutation, potentially leading to more effective and personalized care. This precision in targeting the underlying genetic causes of cystic fibrosis is what makes these treatments stand out.
What evidence suggests that this trial's treatments could be effective for cystic fibrosis?
In this trial, participants will receive one of the following treatments: Symdeko, Ivacaftor, or Orkambi. Research has shown that Symdeko, a combination of tezacaftor and ivacaftor, can significantly improve lung function in people with cystic fibrosis. Studies have found that it enhances lung performance, lowers sweat chloride levels, and improves quality of life. Ivacaftor alone has also proven effective, with evidence showing it reduces lung issues and hospital visits for cystic fibrosis patients. Clinical trials have demonstrated that Orkambi, which combines lumacaftor and ivacaftor, benefits patients with certain genetic mutations by improving lung function and reducing disease severity. All these treatments aim to enhance lung health and overall well-being in cystic fibrosis patients.56789
Are You a Good Fit for This Trial?
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 for a Trial Participant
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
What Are the Treatments Tested in This Trial?
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
George Solomon
Lead Sponsor