Active not recruitingPHASE3INTERVENTIONAL

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

This study is investigating a new medication called VX-121/TEZ/D-IVA for young individuals with cystic fibrosis (CF) between the ages of 1 and 11. The main goal is to understand how well the medicine works, how safe it is, and how the body processes it. We are looking for participants who have a specific genetic change (mutation) in their CFTR gene that is known to respond to triple combination treatments. This research aims to find better ways to manage CF and improve the health of those affected. It will help us determine if this new medicine could be a good option for this age group.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

210

Start

21 Jun 2022

Estimated completion

30 Jun 2030

Cystic Fibrosis

What is this study about?

Cystic fibrosis (CF) is a condition that affects many parts of the body, especially the lungs and digestive system. It's caused by a faulty gene. Researchers are always looking for new and better ways to help people with CF live healthier lives. This particular study is focusing on a new medicine called VX-121/TEZ/D-IVA.

The main purpose of this study is to carefully look at how this new medicine works in children and young people with CF aged between 1 and 11 years old. We want to understand several things: how safe the medicine is, how well it is tolerated (meaning if it causes many side effects), how the body uses and processes the medicine (pharmacokinetics), and most importantly, if it helps improve the health markers for CF (efficacy).

This is a Phase 3 study, which means it's one of the final steps before a medicine might be made available to everyone. The information we gather will be crucial in helping doctors and regulators decide if this new treatment is a valuable option for children with CF who have certain genetic changes.

Key takeaways

This study is testing a new medicine for cystic fibrosis in children aged 1 to 11.
It aims to check the safety, tolerability, and effectiveness of VX-121/TEZ/D-IVA.
Participants must have a specific genetic change (mutation) in their CFTR gene.
The study is in Phase 3, a late stage of clinical research.
Participation involves taking the study medicine and regular health checks at clinic visits.

Who may be eligible?

To join this study, children must have been diagnosed with cystic fibrosis and need to have a specific genetic change (called a 'mutation') in their CFTR gene that is known to respond to certain types of medication. They should also be generally stable in their CF condition at the time of joining the study. This means their CF shouldn't be getting worse quickly or causing serious problems that need urgent attention.

There are also some reasons why a child might not be able to participate. For instance, if they have had an organ transplant, a blood cancer, or certain liver problems that are already moderate or severe. Children with certain lung infections that are known to make CF worse quickly would also not be able to join. These rules are in place to make sure the study is safe for everyone involved and that the results are clear.

Quick self-check

Is my child between 1 and 11 years old?
Does my child have cystic fibrosis?
Has my child's doctor confirmed they have a specific CF gene change that responds to certain 'triple combination' medicines?
Is my child's CF condition generally stable, without rapid worsening?
Does my child have any history of organ transplants, blood cancer, or significant liver problems?
Does my child have any lung infections that tend to severely worsen their CF?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child qualifies and you decide to take part, they will receive the study medicine, VX-121/TEZ/D-IVA. The study will involve regular visits to the clinic where doctors and nurses will do various health checks. These checks might include blood tests, breathing tests, and other assessments to monitor your child's health and see how they are responding to the medicine.

You and your child will be carefully looked after throughout the study. You will be asked about any side effects your child might experience. The total duration of participation will depend on the study design, but generally, Phase 3 studies involve ongoing treatment and regular follow-up for a period to gather enough information about the long-term effects of the medicine. All study-related medication and tests will be provided at no cost.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as access to a new medicine that could improve your child's cystic fibrosis before it's widely available. It also contributes valuable information to scientific understanding that could help many other children with CF in the future. However, there are also potential risks; the medicine might cause side effects, some of which could be unexpected or serious. All efforts will be made to monitor your child's health closely. You and your child have the right to withdraw from the study at any time, for any reason, without it affecting their future medical care.

Locations (38)

Children's Hospital of Orange County
Orange, United States
Stanford University Clinical and Translational Research Unit
Palo Alto, United States
Children's Hospital of Colorado
Aurora, United States
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
Atlanta, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, United States
Boston Children's Hospital
Boston, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, United States
The Children's Mercy Hospital
Kansas City, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, United States
Cohen Children's Medical Center
Lake Success, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, United States

+26 more sites — see the official record for the full list.

Common questions

What is VX-121/TEZ/D-IVA?

It's a new medicine being tested for children with cystic fibrosis. It's a combination of three different drugs designed to help the faulty gene that causes CF work better.

Who is funding this study?

The study is sponsored by a pharmaceutical company called Vertex, which develops medicines for conditions like cystic fibrosis.

Will my child know if they are getting the new medicine or a dummy pill (placebo)?

The study design will determine this. In some studies, participants know, and in others (called 'blinded' studies), neither the patient nor the doctor knows to keep the results fair. Your study team can provide more exact details.

How long will my child need to be in the study?

The exact duration can vary, but these types of studies often involve several months to over a year of follow-up. The study team will give you a specific timeline.

Will this study cure cystic fibrosis?

No, this study is not expected to cure cystic fibrosis. Instead, it aims to find new medicines that can help manage the condition more effectively and improve the health of those living with CF.