Cystic Fibrosis (CF) is a disease affecting the lungs and digestive system. It is caused by mutations in the CFTR gene, which leads to thick, sticky mucus in the body. This study tests a new treatment, VX-121/Tezacaftor/Deutivacaftor (also called VX-121/TEZ/D-IVA), for children aged 1 to 11 with CF who have a specific type of gene mutation called TCR mutation. This study checks how the body processes the medicine, its safety, and if it helps CF symptoms.
- Participation Requirements: Children must have stable CF and at least one TCR mutation.
- Exclusions: Children with certain health issues, like past organ transplants or severe liver problems, cannot participate.
- Study Details: The study checks how safe and effective the treatment is for young CF patients.
If your child has CF and meets the criteria, participating could help in understanding new treatments for CF. Always discuss with your doctor to understand potential benefits and risks. The study's goal is to find better treatments for CF, especially for young children with specific gene mutations.