A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Details
Therapeutic approach
The type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise
A Phase 2, Randomized, Double-blind Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Study details
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561
Study results
This study assessed the safety and effectiveness of deutivacafter monotherapy (VX-561) in people with CF aged 18 years and older who have a gating CFTR gene variant and were previously taking ivacaftor.
A total of 77 people took part in the study, and they were randomly assigned to one of five study groups. Group 1 (the control group) continued taking Ivacaftor. Groups 2 to 5 were assigned varying doses of deutivacaftor starting at 25mg daily to a maximum of 250mg daily.
Groups 2 and 3 (25mg and 50mg doses) were discontinued by the trial sponsor, Vertex, when participants in these groups showed worsening lung function. Analysis of the results was only done on groups 1, 4, and 5 (Ivacaftor, 150mg, and 250mg doses).
The results of this study showed that deutivacaftor was safe and well-tolerated, the deutivacaftor safety profile was consistent with the safety profile of Ivacaftor treatment. The participants in the deutivacaftor groups also showed improvements in lung function and decrease in sweat chloride levels. These results indicated further research into deutivacaftor therapy as a CFTR modulator.
Phase
The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use
Phase 2
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial
Who can take part?
Age range
18 years and older
Including people
Must have one of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
On Ivacaftor therapy
FEV1 value ≥40% and ≤100%
Excluding people
History of clinically significant cirrhosis with or without portal hypertension
History of solid organ or hematological transplantation
Lung infection with organisms associated with a more rapid decline in pulmonary status
