Ivacaftor or Elexacaftor/Tezacaftor/Ivacaftor for Sinus Disease in Children with Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores how a new treatment for cystic fibrosis (CF) affects sinus problems and the sense of smell in young children. It involves two groups of children with CF: one group will begin the new CF treatment, while the other will not. The goal is to determine if the treatment improves sinus health and quality of life over two years. Children with CF who frequently experience sinus issues and are either starting or not using the new treatment could be suitable candidates for this trial. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important findings.
Do I have to stop taking my current medications for the trial?
The trial requires that you stop taking ivacaftor or elexacaftor/tezacaftor/ivacaftor and chronic oral corticosteroids at least 28 days before the first visit. If you are using an investigational drug, you must stop 28 days before the first visit as well.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that ivacaftor is safe for children with cystic fibrosis (CF) who are at least 1 month old and have specific gene mutations. Past studies indicate that children tolerate it well, but it does not work for those with two copies of the F508del mutation.
For the combination of elexacaftor, tezacaftor, and ivacaftor, studies have found it safe for people with CF who have at least one F508del mutation. However, some cases of serious liver damage have occurred, so discussing this with a doctor is important. This combination is approved for patients aged 12 and older, but its safety for children under 2 remains uncertain.
Both treatments have received FDA approval for certain age groups and CF mutations, supporting their safety. Always consult a healthcare provider about any concerns and to determine if these treatments are suitable for your child.12345Why are researchers excited about this trial?
Most treatments for sinus disease in children with cystic fibrosis focus on managing symptoms with antibiotics, decongestants, or nasal steroids. However, the treatments being studied here, ivacaftor and elexacaftor/tezacaftor/ivacaftor, work directly on the underlying genetic cause of cystic fibrosis by targeting and improving the function of the CFTR protein. This approach is unique because instead of just alleviating symptoms, it aims to correct the malfunctioning protein responsible for the condition. Researchers are excited because this could lead to more effective and long-lasting improvements in sinus health for these young patients.
What evidence suggests that highly effective modulator therapy could be effective for chronic rhinosinusitis and olfactory dysfunction in young children with cystic fibrosis?
Research shows that ivacaftor improves lung function in people with cystic fibrosis (CF), including young children. Studies have found that it leads to lasting improvements in lung function and overall health in CF patients. In this trial, some participants will join the HEMT Group, starting ivacaftor or the combination of elexacaftor, tezacaftor, and ivacaftor. This combination therapy significantly improves health outcomes, lowering sweat chloride levels and reducing lung symptom flare-ups by 63%. Both treatments effectively manage cystic fibrosis symptoms. Meanwhile, the Non-HEMT/Control Group will include children with CF not on these CFTR modulator therapies.678910
Who Is on the Research Team?
Daniel M Beswick, MD
Principal Investigator
University of California, Los Angeles
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Initial assessments including MRI scans, olfactory tests, and quality of life surveys
1-Year Follow-up
Participants undergo assessments to evaluate changes in sinus and olfactory health
2-Year Follow-up
Final assessments to evaluate long-term changes in sinus and olfactory health
What Are the Treatments Tested in This Trial?
Interventions
- Elexacaftor/Tezacaftor/Ivacaftor
- Ivacaftor
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the non-HEMT group of this study may enroll into the HEMT cohort if they become eligible for these CFTR modulator therapies and plan to start them.
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, Los Angeles
Lead Sponsor
University of Vermont
Collaborator
Children's Hospital Medical Center, Cincinnati
Collaborator
University of Kansas Medical Center
Collaborator
University of Iowa
Collaborator
Children's Hospital Colorado
Collaborator
University of Virginia
Collaborator
University of North Carolina, Chapel Hill
Collaborator
Citations
LONGITUDE: An observational study of the long-term ...
LONGITUDE is an observational, registry-based cohort study using data from the UK CF Registry to evaluate outcomes of ELX/TEZ/IVA in people aged ≥6 years.
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news.vrtx.com
news.vrtx.com/news-releases/news-release-details/vertex-presents-new-data-across-portfolio-cystic-fibrosisVertex Presents New Data Across Portfolio of Cystic Fibrosis ...
ALYFTREK has shown greater reductions in sweat chloride compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Phase 3 trials ...
Real‐World Evaluation of Outcomes and Safety ...
Elexacaftor/Tezacaftor/Ivacaftor (ETI) therapy has significantly improved clinical outcomes in people with cystic fibrosis (PwCF) carrying ...
Real-world outcomes and direct care cost before and after ...
With the most recent approval of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), approximately 90% of the CF population is now eligible to ...
Studies and Results | TRIKAFTA® (elexacaftor/tezacaftor ...
Through 24 weeks, the number of pulmonary exacerbations significantly decreased by 63% for people taking TRIKAFTA compared with placebo.
Safety Profile | TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor ...
Safety data from 510 patients aged 12 years and older with CF with at least one F508del mutation in 2 double‑blind, controlled, Phase 3 trials of 24 weeks ...
Important Safety Information
TRIKAFTA can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a ...
Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta) - NCBI
Data from the Canadian Cystic Fibrosis Registry on people in Canada with CF who do not currently have a Health Canada–based indication for modulators show ...
Real-world safety and effectiveness of elexacaftor ...
Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in people with cystic fibrosis (CF) with ≥1 ...
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) ... It is not known if TRIKAFTA is safe and effective in children under 2 years ...
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