Cystic Fibrosis Foundation issued the following clinical trial alerts in the month of September.
September 8, 2020
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part A)
Status: Enrolling
Description: This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.
Age: 0 Years to 4 Years
Mutation: No Mutation Requirement
Fev1% Predicted: No FEV1 Limit
Number of Visits: 6
Length of Participation: 3 years
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04509050
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part B)
Status: Enrolling
Description: This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.
Age: Less than 6 Years
Mutation: No Mutation Requirement
Fev1% Predicted: No FEV1 Limit
Number of Visits: 6
Length of Participation: 2 years
ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT04509050
September 30, 2020
Status: Completed with results
Description: This study evaluated the safety and effectiveness of VX-445 (elexacaftor), tezacaftor, and ivacaftor in combination called TRIKAFTA®. This study was for people with CF 12 years and older with one copy of the F508del mutation and one copy of a gating or residual function mutation.
Age: 12 Years and Older
Mutation: One Copy F508del
Fev1% Predicted: 40 to 90%
Number of Visits: 8
Length of Participation: 20 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04058353