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CFTR Modulator
VX-121 + Tezacaftor + Deutivacaftor for Cystic Fibrosis
Phase 3
Recruiting
Research Sponsored by Vertex Pharmaceuticals Incorporated
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Be younger than 18 years old
Must not have
Moderate hepatic impairment (Child Pugh Score 7 to 9)
History of solid organ, hematological transplantation, or cancer
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial
Summary
This trial tests a combination of three drugs for cystic fibrosis patients with a specific genetic mutation. The drugs work together to fix the faulty protein in their cells, helping their lungs and other organs function better. Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation in the CFTR gene.
Who is the study for?
This trial is for children aged 1-11 with stable cystic fibrosis who have at least one mutation responsive to the treatment. It's not suitable for those with a history of organ transplant, cancer, significant liver disease, or certain lung infections that worsen breathing.
What is being tested?
The study tests VX-121/tezacaftor/deutivacaftor in young CF patients to see how their bodies handle it and if it's safe and effective. The medication aims to improve lung function by targeting the defective protein causing CF.
What are the potential side effects?
While specific side effects aren't listed here, common ones for CF treatments include chest discomfort, coughing, digestive changes, and potential liver issues. Monitoring during the trial helps manage any adverse reactions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My cystic fibrosis is stable and I have at least one CFTR gene mutation.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My liver function is moderately impaired.
Select...
I have a history of organ transplant or cancer.
Select...
My liver is severely impaired.
Select...
I have a lung infection that worsens my breathing condition quickly.
Select...
I have liver cirrhosis with increased blood pressure in the liver.
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
Side effects data
From 2023 Phase 3 trial • 435 Patients • NCT0503308032%
Infective pulmonary exacerbation of cystic fibrosis
26%
COVID-19
20%
Cough
17%
Nasopharyngitis
13%
Upper respiratory tract infection
12%
Nasal congestion
11%
Blood creatine phosphokinase increased
11%
Oropharyngeal pain
11%
Headache
10%
Sputum increased
10%
Pyrexia
9%
Viral upper respiratory tract infection
8%
Fatigue
8%
Nausea
8%
Back pain
7%
Rhinorrhoea
7%
Diarrhoea
7%
Abdominal pain
6%
Respiratory tract infection
5%
Arthralgia
5%
Abdominal distension
5%
Haemoptysis
5%
Influenza
5%
Alanine aminotransferase increased
5%
Sinusitis
4%
Aspartate aminotransferase increased
4%
Rash
3%
Abdominal pain upper
2%
Productive cough
2%
Sinus congestion
1%
Constipation
100%
80%
60%
40%
20%
0%
Study treatment Arm
ELX/TEZ/IVA
VNZ/TEZ/D-IVA
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Part B: VX-121/TEZ/D-IVAExperimental Treatment1 Intervention
Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
Group II: Part A: VX-121/TEZ/D-IVAExperimental Treatment1 Intervention
Participants will receive VX-121/TEZ/D-IVA in the morning.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
VX-121/TEZ/D-IVA
2022
Completed Phase 3
~1070
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 those being studied in the VX-121/tezacaftor/deutivacaftor trial, target the defective CFTR protein caused by mutations in the CFTR gene, which is the root cause of Cystic Fibrosis. Correctors like tezacaftor help the CFTR protein fold correctly and reach the cell surface, while potentiators like ivacaftor enhance the function of the CFTR protein at the cell surface, improving chloride ion transport.
These treatments are crucial for CF patients as they address the underlying cause of the disease, leading to improved lung function, reduced pulmonary exacerbations, and overall better quality of life.
Find a Location
Who is running the clinical trial?
Vertex Pharmaceuticals IncorporatedLead Sponsor
255 Previous Clinical Trials
34,475 Total Patients Enrolled
128 Trials studying Cystic Fibrosis
17,735 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My liver function is moderately impaired.My cystic fibrosis is stable and I have at least one CFTR gene mutation.I have a history of organ transplant or cancer.My liver is severely impaired.I have a lung infection that worsens my breathing condition quickly.I have liver cirrhosis with increased blood pressure in the liver.
Research Study Groups:
This trial has the following groups:- Group 1: Part A: VX-121/TEZ/D-IVA
- Group 2: Part B: VX-121/TEZ/D-IVA
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
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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