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Observational Completed with Results
Observational Study in Children 6 Years and Up who have the R117H or other non-G551D gating mutation (GOAL-OB-11 Expansion)
These observational cohorts were a part of the expansion of the GOAL-e2 Study (GOAL-OB-11 Expanded and Extended). In these cohorts, data were collected from people with CF who have the R117H and other non-G551D gating CFTR mutations and had never taken ivacaftor (Kalydeco®).
Clinical information (including height, weight, lung function, BMI, sweat chloride, and patient reported outcomes) was collected at four time points during the study: before and one, three, and six months after participants started ivacaftor. Information from the CF Foundation Patient Registry (CFFPR) (including hospitalization and P. aeruginosa culture results) was also collected and merged with the clinical data. Additionally, samples (including plasma, serum, buffy coat, urine and sputum) were collected to store and use for future research.
Eligibility
See other primary eligibility criteria for more information.
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Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No 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
Must have following CFTR mutations: For Cohort 2: a) R117H on at least 1 allele b) Any known or unknown mutation on the second allele except G551D For Cohort 3: a) A Non-G551D gating mutation on one allele: (G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D) b) Any known or unknown mutation on the second allele except G551D OR R117H Cannot be participating in the VX-770-105, VX-770-106, VX-770-108, VX-770-110, VX-770-111, VX-770-112, or VX-770-113 study, VX-770 Extended Access Program or use of ivacaftor within 6 months prior to Visit 1.
Study Results
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What We Learned:
This study showed people with CF who have the R117H and other non-G551D gating CFTR mutations treated with ivacaftor had significant improvements in lung function, nutritional status, sweat chloride, and patient reported respiratory symptoms when compared to those who were not treated with ivacaftor.
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Primary Findings:
Effectiveness:
Safety:
This study was conducted between February 2014 and January 2016. Of the 23 participants enrolled in the study, 21 were prescribed ivacaftor as part of the study and 86% completed the entire study including follow-up. Significant improvements in lung function (as measured by FEV1) were observed after taking ivacaftor at one month (FEV1% increase of 9.3%, p=0.0011), at three months (FEV1% increase of 8.4%, p=0.0214) and at six months (FEV1% increase of 10.9%, p=0.0134). Significant improvements in nutritional status (as measured by BMI and weight) were also seen (mean BMI increase of 1.4 kg/m2, p<0.001). Similarly, overall sweat chloride significantly decreased throughout the study (1 month= -53.5 mEq/L, p<0.0001; 3 months= -51.4 mEq/L, p<0.0001; and 6 months= -48.6 mEq/L, p<0.0001). Patient reported outcomes (as measured by the CF Questionnaire-Revised) were significantly improved at six months (improvement of 14.2 points, p=0.0366).
Extracted from the CFFPR, hospitalizations significantly decreased after participants initiated ivacaftor (rate reduction of 0.71 hospitalizations per participant-year, 95% CI, p=0.004). P. aeruginosa positive cultures did not show a significant decrease.
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Citation:
J Cyst Fibros ;DOI 10.1016/j.jcf.2018.04.004 [Epub ahead of print]
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Observational -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
6 months -
Number of Study Visits:
5
Additional Information
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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:
6 Years and Older -
Mutation(s):
One Copy F508del or No 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
Must have following CFTR mutations: For Cohort 2: a) R117H on at least 1 allele b) Any known or unknown mutation on the second allele except G551D For Cohort 3: a) A Non-G551D gating mutation on one allele: (G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D) b) Any known or unknown mutation on the second allele except G551D OR R117H Cannot be participating in the VX-770-105, VX-770-106, VX-770-108, VX-770-110, VX-770-111, VX-770-112, or VX-770-113 study, VX-770 Extended Access Program or use of ivacaftor within 6 months prior to Visit 1.
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