CFF provided a series of clinical trial updates in January 2017. Click the trial title to get more details.
The following alerts were distributed on 01/19/2017:
Description: This was an open-label study designed to look at the safety of lumacaftor in combination with ivacaftor (Orkambi®). This study was for younger children with CF who have two copies of the F508del CFTR mutation.
Age: 6 Years to 11 Years
Mutation: Two Copies F508del
Fev1% Predicted: No FEV1 Limit
Number of Visits: 11
Length of Participation: 27 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/show/NCT01897233
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis
Description: This study looked at the safety and effectiveness of the drug VX-661 in combination with ivacaftor (Kalydeco®) compared to placebo. This study was for people with CF who have one copy of the F508del CFTR mutation and a second CFTR mutation that is not likely to respond to this drug therapy.
Age: 12 Years and Older
Mutation: One Copy F508del
Fev1% Predicted: 40 to 90%
Number of Visits: 7
Length of Participation: 20 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02516410
The following alerts were distributed on 01/13/2017:
Phase 2 study of inhaled Alpha-1 HC in people with CF
Description: This study assessed the safety and tolerability of two different doses of once daily inhaled Alpha-1 anti-trypsin in people with CF.
Age: 18 Years and Older
Mutation: No Mutation Requirement
Fev1% Predicted: 40 to 90%
Number of Visits: 5
Length of Participation: 9 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/show/NCT01684410
BONUS: Baby Observational and Nutrition Study
Description: This observational study was conducted to gain a better understanding of the factors that interfere with good growth in infants with CF. [NOTE: This study is complete.]
Age: Less than 15 Weeks
Mutation: No Mutation Requirement
Fev1% Predicted: No FEV1 Limit
Number of Visits: 9
Length of Participation: 12 months
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01424696
The following alert was distributed on 01/10/2017:
VX-661 alone and in combination with ivacaftor in people with cystic fibrosis
Description: This study looked at the safety and effectiveness of multiple dose levels of VX-661 alone and in combination with ivacaftor (KALYDECO®).
Age: 18 Years and Older
Mutation: Two Copies F508del
Fev1% Predicted: 40 to 90%
Number of Visits: 9
Length of Participation: 84 days
ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01531673
The following alert was distributed on 01/06/2017:
Description: This study evaluated the effects of an anti-oxidant enriched multivitamin supplement on inflammation. This study was for people with CF who are pancreatic insufficient.
Age: 10 Years and Older
Mutation: No Mutation Requirement
Fev1% Predicted: 40 to 100%
Number of Visits: 4
Length of Participation: 20 weeks
ClinicalTrials.gov link: https://clinicaltrials.gov/show/NCT01859390