Study of New Drug Dosing and Genetic Factors in Children with Cystic Fibrosis Using Triple Combination Therapy

What this Trial Is About

Researchers are studying how several new drugs affect children with cystic fibrosis CF and how genetic differences influence their response to treatments. The study aims to identify who is most likely to benefit from existing therapies, potentially reducing side effects and high costs associated with ineffective treatments. Understanding how these drugs work and how best to study them can help expand their use to all patients with CF. The study involves participants who may join an observational one-visit study to examine drug concentration and side effects. Those experiencing side effects can join a single arm study focused on adjusting their dose to keep drug levels within an effective range. Once the optimal dose is reached, no further adjustments occur. The study evaluates side effects and dosing strategies specifically for the triple combination CFTR modulator therapy consisting of elexacaftor, tezacaftor, and ivacaftor. Participants will undergo assessments of drug concentration, mental and neuropsychological health, and treatment responses over 6 to 12 months. The study monitors side effects and the feasibility of dose adjustments to manage neuropsychological side effects. Data collection includes up to six concentration assessments and ongoing evaluation throughout the treatment period. The total involvement duration varies but includes careful follow-up to understand treatment effects and safety.

Ensuring Access to Optimal Therapy in CF: The ENACT Study