ELX/TEZ/IVA for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\<) 24 months of age.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinator or your doctor for guidance.
How is the drug ELX/TEZ/IVA unique for treating cystic fibrosis?
ELX/TEZ/IVA is unique because it combines three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor) to improve the function of the CFTR protein in cystic fibrosis patients, especially those with the F508del mutation. This combination has shown significant improvements in lung function and other health outcomes compared to previous treatments.12345
Are You a Good Fit for This Trial?
This trial is for young children aged 12 to less than 24 months with Cystic Fibrosis who have at least one F508del mutation or another mutation responsive to the study drugs. Children should not have other illnesses that could confuse the results or increase risks from the study drugs.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Participants receive ELX/TEZ/IVA in the morning and IVA in the evening
Treatment Part B
Participants receive ELX/TEZ/IVA in the morning and IVA in the evening with doses based on Part A outcomes
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ELX/TEZ/IVA
- IVA
ELX/TEZ/IVA is already approved in United States, European Union, Canada for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
Dr. David Altshuler
Vertex Pharmaceuticals Incorporated
Chief Medical Officer since 2020
MD, PhD
Dr. Reshma Kewalramani
Vertex Pharmaceuticals Incorporated
Chief Executive Officer since 2020
MD, trained in internal medicine and nephrology