Elexacaftor/Ivacaftor/Tezacaftor for Cystic Fibrosis

(ENACT Trial)

This trial tests the effectiveness of a combination of three drugs—Elexacaftor, Ivacaftor, and Tezacaftor—for treating cystic fibrosis (CF) in children. Researchers aim to predict which patients will benefit from these drugs, potentially avoiding unnecessary side effects and costs for those who won't. The trial involves a single group of participants, focusing on determining the right dose to minimize side effects. Children aged 2 and older with a stable CF diagnosis and no recent lung issues may be suitable for this study. As a Phase 4 trial, this research seeks to understand how the already FDA-approved and effective treatment benefits more patients.

The trial requires that you stay on a stable dose of your current CFTR modulator therapy for at least two weeks before starting the study. It doesn't specify stopping other medications, so you should discuss this with the trial team.

Research shows that combining elexacaftor, tezacaftor, and ivacaftor is generally safe for people with cystic fibrosis. Studies have found positive results even in children as young as six. Side effects have been minor, with improvements in lung function and overall health. For example, one study showed that participants breathed better and experienced fewer lung infections. Additionally, the FDA has approved this treatment for cystic fibrosis, indicating its safety for this condition.¹²³⁴⁵

Researchers are excited about Elexacaftor/Ivacaftor/Tezacaftor for cystic fibrosis because it targets the root cause of the disease, unlike many treatments that only manage symptoms. This combination therapy works by correcting the malfunctioning CFTR protein, which is the underlying issue in most cystic fibrosis cases. By improving the protein's function, these drugs have the potential to significantly enhance lung function and overall health, offering a more comprehensive approach than traditional therapies like antibiotics or mucus thinners.

Research has shown that the combination of Elexacaftor, Ivacaftor, and Tezacaftor effectively treats cystic fibrosis. Studies have found that these drugs significantly improve lung function and overall health. Patients on this treatment experience fewer hospital and emergency room visits and a slower decline in lung function compared to those not receiving the treatment. In the long term, these drugs are linked to increased survival rates. Participants in this trial will join a single-arm study to assess the feasibility of adjusting doses to maintain concentrations within an estimated effective range, with side effects evaluated as described in the protocol.¹³⁶⁷⁸