Page Title
Clinical Trial Finder
Observational Completed with Results
PROSPECT Part B (PROSPECT-OB-14 - Part B )
Part B of the PROSPECT study evaluated the effectiveness of lumacaftor/ivacaftor (Orkambi®) and collected biospecimens and clinical data from people who have two copies of the F508del CFTR mutation both before and after treatment with lumacaftor/ivacaftor.
Participants participated in at least one study visit (baseline visit) prior to being prescribed lumacaftor/ivacaftor. Additional study visits were conducted after participants had taken lumacaftor/ivacaftor for 1, 3, 6 and 12 months. At each visit, researchers measured lung function, body weight and sweat chloride concentration.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
12 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants in Part B of the PROSPECT study were required to have two copies of the DF508 gene and their physicians planned to treat them with lumacaftor/ivacaftor.
Study Results
-
What We Learned:
This study found that participants taking lumacaftor/ivacaftor did not have a significant change in lung function (measured by FEV1) but had a significant decrease in sweat chloride and improvements in nutritional status.
-
Primary Findings:
Effectiveness:
Safety:
This study was conducted between March 2015 and April 2018. The study enrolled 196 participants. 193 participants started taking lumacaftor/ivacaftor and 164 completed the study through 1 year.
Lung function (FEV1) did not significantly change from baseline after taking lumacaftor/ivacaftor for 12 months (baseline FEV1= 85%, mean change = -0.3 (95%CI: -1.8, 1.2)).
Nutritional status (as measured by BMI) improved from baseline to 12 months (mean change=0.8 kg/m2, p<0.001). Sweat chloride improved significantly from baseline to 1 month (mean change= -18.5 mmol/L, 95% CI = (-20.7, -16.3), p<0.001), and these reductions were sustained through the study period. There were no significant changes in hospitalization rate for pulmonary exacerbations and Pseudomonas aeruginosa infection status with treatment.
-
Citation:
Ann Amer Thorac Soc ;DOI 10.1513/AnnalsATS.202002-144OC
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
-
Study Type: ?more info
Observational -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
1 years -
Number of Study Visits:
5
Additional Information
-
Phase: ?more info
Not Applicable -
Study Sponsor: ?more info
Rowe, Steven -
Study Drugs:
N/A
Eligibility
See other primary eligibility criteria for more information.
-
Age:
12 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
Other Primary Eligibility Criteria
Participants in Part B of the PROSPECT study were required to have two copies of the DF508 gene and their physicians planned to treat them with lumacaftor/ivacaftor.
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Check the Drug Development Pipeline
We’re attacking CF from every angle. Learn about the status of CF drugs in development.
Learn More