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Oct

Clinical Trial Alerts – October 2018

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:

Phase 3 study of lumacaftor/ivacaftor (Orkambi®) in babies with two copies of the F508del CFTR mutation

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

Phase 3 study of Vertex 661 and ivacaftor in people with CF who have one copy of the F508del-CFTR mutation and a second CFTR mutation with a gating defect responsive to ivacaftor

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


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