Triple Therapy Modulator for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new triple-therapy medication for individuals with Cystic Fibrosis (CF) who experienced mental health or liver issues with a previous treatment. The goal is to determine if the new drug combination (vanzacaftor, tezacaftor, deutivacaftor) is safe and effective for those who had to stop or change their old medication due to these side effects. Participants should have experienced new or worsening mental health symptoms or liver problems with the prior treatment and intend to start the new therapy. It suits those who can delay starting the new treatment and have a smart device to receive surveys. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important CF advancements.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop your current medications. However, it mentions that participants should be willing to delay the first dose of the new treatment for a short period to complete baseline assessments.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that the medication vanzacaftor-tezacaftor-deutivacaftor (VTD) is safe and well tolerated for people with cystic fibrosis. Studies have found that it works as effectively as another treatment, elexacaftor-tezacaftor-ivacaftor (ETI), improving lung function and other symptoms.
Trials have proven VTD safe and effective for children as young as 6 years old and for older patients, ages 12 and up. People taking VTD generally did not experience more side effects than those taking ETI.
For those concerned about side effects, it is reassuring that VTD has undergone testing for long-term safety. Researchers are closely monitoring any potential issues, and so far, the treatment is considered safe for people with cystic fibrosis.12345Why are researchers excited about this trial?
Researchers are excited about the new combination of vanzacaftor/tezacaftor/deutivacaftor (VTD) because it offers potential benefits for people with cystic fibrosis (CF) who experience side effects from the current standard treatment, elexacaftor/tezacaftor/ivacaftor (ETI). The VTD combination is being explored for its ability to reduce mental health and neurocognitive symptoms, like depression and brain fog, which some patients experience on ETI. Additionally, VTD may offer a safer profile for individuals who have had drug-induced liver issues with ETI, potentially lowering the risk of liver injury. This makes VTD an exciting prospect as it could provide a more tolerable and effective treatment option for individuals with challenging side effects from existing therapies.
What evidence suggests that this trial's treatments could be effective for Cystic Fibrosis?
This trial will evaluate the treatment vanzacaftor-tezacaftor-deutivacaftor (VTD) for cystic fibrosis in various contexts. Research has shown that VTD is promising for cystic fibrosis, with studies indicating it matches elexacaftor-tezacaftor-ivacaftor (ETI) in improving lung function for individuals aged 12 and older. It is also safe and effective for children aged 6 to 11. Participants in the RETRIAL-Mental Health arm will include those with a history of mental health symptoms while on ETI. The RETRIAL-Neuro arm will focus on individuals with neurocognitive symptoms, and the RETRIAL-Liver arm will include participants with liver-related issues attributed to ETI. While ETI can sometimes cause liver problems, VTD might offer a safer option with fewer liver-related side effects. Overall, VTD is well-tolerated and effective for managing cystic fibrosis.36789
Who Is on the Research Team?
Anna Georgiopoulos, MD
Principal Investigator
Massachusetts General Hospital
CJ Bathgate, PhD
Principal Investigator
National Jewish Health
Janis Stoll, MD
Principal Investigator
Washington University at St. Louis
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
Treatment
Participants start taking vanzacaftor/tezacaftor/deutivacaftor (VTD) and are monitored for mental health and liver-related adverse events
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Vanzacaftor/Tezacaftor/Deutivacaftor (VTD)
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
People with CF from RETRIAL-Mental Health who experienced new/worsening neurocognitive symptoms (such as brain fog or memory problems) while taking elexacaftor/tezacaftor/ivacaftor (ETI).
People with CF ages 6 and up with a history of new or worsening mental health symptoms (such as depression, anxiety, mood, sleep) while on elexacaftor/tezacaftor/ivacaftor (ETI) requiring discontinuation or change from standard dosing who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
People with Cystic Fibrosis ages 6 and up with a history of drug-induced liver injury (such as elevated liver enzymes) attributed to elexacaftor/tezacaftor/ivacaftor (ETI) requiring dose modification or discontinuation who start taking vanzacaftor/tezacaftor/deutivacaftor (VTD).
Find a Clinic Near You
Who Is Running the Clinical Trial?
Boston Children's Hospital
Lead Sponsor
Cystic Fibrosis Foundation
Collaborator
National Jewish Health
Collaborator
Washington University School of Medicine
Collaborator
Massachusetts General Hospital
Collaborator
University of Kansas Medical Center
Collaborator
Johns Hopkins University
Collaborator
Children's Hospital Colorado
Collaborator
Indiana University
Collaborator
Citations
Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor– ...
These results show that vanzacaftor–tezacaftor–deutivacaftor treatment might lead to more people with cystic fibrosis attaining levels of CFTR function either ...
Trial 3 | ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor)
One patient had 2 serious adverse events: infective pulmonary exacerbation of cystic fibrosis and failure to thrive. 2. Liver function test elevations1,4 ...
Study Results
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 ...
4.
investors.vrtx.com
investors.vrtx.com/news-releases/news-release-details/vertex-presents-new-data-across-portfolio-cystic-fibrosisVertex Presents New Data Across Portfolio of Cystic ...
TRIKAFTA can cause serious and potentially fatal drug-induced liver injury. ... Liver injury has been reported within the first month of therapy ...
5.
clinicaltrials.gov
clinicaltrials.gov/study/NCT05844449?term=deutivacaftor%20AND%20tezacaftor%20AND%20vanzacaftor&rank=2Evaluation of Long-Term Safety and Efficacy of Vanzacaftor ...
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor / tezacaftor / deutivacaftor (VNZ/TEZ/D-IVA) in ...
Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 ...
Vanzacaftor-tezacaftor-deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator.
Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor ...
Elexacaftor–tezacaftor–ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele.
Vanzacaftor–Tezacaftor–Deutivacaftor Noninferior to ETI
Vanzacaftor–tezacaftor–deutivacaftor was noninferior to elexacaftor–tezacaftor–ivacaftor among patients aged 12 years and older with cystic fibrosis.
Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor ...
Vanzacaftor-tezacaftor-deutivacaftor is non-inferior to elexacaftor-tezacaftor-ivacaftor in terms of FEV 1 % predicted, and is safe and well tolerated.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.