Cystic Fibrosis Foundation distributed the following clinical trial updates in October. Click the trial title to get more details.
The following alert was issued on October 2:
Status: Enrolling
Description: This study is taking place at multiple care centers across the U.S. It will evaluate the safety of the drug lumacaftor/ivacaftor (Orkambi®) and its effect on the body. It is for babies ages 1 to less than 2 years old who have cystic fibrosis and two copies of the F508del CFTR mutation.
Age: 1 Years to 2 Years
Mutation: Two Copies F508del
Fev1% Predicted: No FEV1 Limit
Number of Visits: 11
Length of Participation: 38 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03601637
The following alerts were issued on October 2:
Phase 3 study of ivacaftor in babies 12-24 month old who have a CFTR gating mutation
Status: Completed
Description: This study evaluated the safety of ivacaftor (Kalydeco®) as well as how the body processes the drug in babies who have at least one copy of a CFTR gating mutation.
Age: 12 Months to 24 Months
Mutation: One Copy F508del or No Copies F508del
Fev1% Predicted: No FEV1 Limit
Number of Visits: 8
Length of Participation: 24 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02725567
ASSURE: Study of Relizorb in people with CF who receive enteral tube feeding
Status: Completed
Description: This study evaluated the safety, tolerability and effectiveness of RELiZORB® cartridge. RELiZORB® cartridge is a digestive enzyme cartridge for people using enteral nutrition (EN) tube feedings that breaks down fats in enteral nutrition tube feeding formula.
Age: 4 Years and Older
Mutation: No Mutation Requirement
Fev1% Predicted: Less than 100%
Number of Visits: 5
Length of Participation: 4 months
ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT02750501
Phase 3 study of lumacaftor/ivacaftor in children with CF
Status: Completed
Description: This study evaluated the safety and effectiveness of lumacaftor in combination with ivacaftor (Orkambi®) in people who have two copies of the F508del mutation.
Age: 2 Years to 5 Years
Mutation: Two Copies F508del
Fev1% Predicted: 40% or greater
Number of Visits: 11
Length of Participation: 32 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02797132
Phase 2 study of VX-371 in people with CF who are currently taking lumacaftor/ivacaftor
Status: Completed
Description: This study evaluated the safety and effectiveness of the inhaled drug VX-371 (formerly P-1037) in combination with hypertonic saline in participants who have two copies of the F508del CFTR mutation and are taking lumacaftor/ivacaftor (Orkambi®).
Age: 12 Years and Older
Mutation: Two Copies F508del
Fev1% Predicted: 40 to 90%
Number of Visits: 12
Length of Participation: 98 days
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02709109
Status: Completed
Description: This study evaluated the safety and effectiveness of the drug VX-661 in combination with ivacaftor (Kalydeco®) in people already taking ivacaftor (Kalydeco®).
Age: 12 Years and Older
Mutation: One Copy F508del
Fev1% Predicted: 40 to 90%
Number of Visits: 8
Length of Participation: 13 weeks
ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT02412111