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CFTR Modulator
Triple Combination Therapy for Cystic Fibrosis
Phase 3
Waitlist Available
Research Sponsored by Vertex Pharmaceuticals Incorporated
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Must not have
History of solid organ, hematological transplantation, or cancer
Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group
Summary
This trial is testing a combination of three medications to help people with cystic fibrosis. The goal is to see if these drugs are safe and effective over time. These medications work by improving the function of a faulty protein in cystic fibrosis patients, which can enhance their lung function and overall health. Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation of the CFTR gene.
Who is the study for?
This trial is for people with cystic fibrosis who finished treatment in a previous study (VX21-121-105). It's not open to those who've had organ transplants, cancer, drug intolerance during the prior study, or serious liver issues like cirrhosis with portal hypertension.
What is being tested?
The trial tests the long-term safety and effectiveness of a combination medication called vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) for individuals with cystic fibrosis.
What are the potential side effects?
While specific side effects are not listed here, common ones associated with CF treatments may include chest discomfort, coughing, digestive changes, and potential liver problems. The exact side effects will be monitored throughout the trial.
Eligibility Criteria
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have a history of organ transplant or cancer.
Select...
I have severe liver problems that could make taking new medications risky.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: VNZ/TEZ/D-IVAExperimental Treatment1 Intervention
Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
VNZ/TEZ/D-IVA
2024
Completed Phase 1
~50
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
CFTR modulators, such as ivacaftor (a potentiator) and lumacaftor, tezacaftor, and elexacaftor (correctors), work by enhancing the function and proper folding of the CFTR protein, which is defective in Cystic Fibrosis patients. Potentiators improve the activity of the CFTR protein at the cell surface, while correctors assist in the proper folding and trafficking of the protein to the cell surface.
These treatments are vital as they address the root cause of CF, leading to improved lung function, reduced symptoms, and overall better health outcomes for patients.
Small-molecule drugs for cystic fibrosis: Where are we now?Net benefit of ivacaftor during prolonged tezacaftor/elexacaftor exposure in vitro.Modulators of CFTR. Updates on clinical development and future directions.
Small-molecule drugs for cystic fibrosis: Where are we now?Net benefit of ivacaftor during prolonged tezacaftor/elexacaftor exposure in vitro.Modulators of CFTR. Updates on clinical development and future directions.
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Who is running the clinical trial?
Vertex Pharmaceuticals IncorporatedLead Sponsor
255 Previous Clinical Trials
34,505 Total Patients Enrolled
128 Trials studying Cystic Fibrosis
17,765 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You had a bad reaction to a drug in the previous study.I have a history of organ transplant or cancer.I have severe liver problems that could make taking new medications risky.
Research Study Groups:
This trial has the following groups:- Group 1: VNZ/TEZ/D-IVA
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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