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Report for Clinical Trial NCT01531673

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase 2, Multicenter, Double-Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del-CFTR mutation.

NCT01531673 Cystic Fibrosis
MeSH:Cystic Fibrosis Fibrosis

4 Interventions

Name: VX-661

Type: Drug
Group Labels: 11

Group 1: VX-661 10 mg qd Group 2a: VX-661 30 mg qd Group 2b: VX-661 10 mg qd/Ivacaftor 150 mg q12h Group 3a: VX-661 100 mg qd Group 3b: VX-661 30 mg qd/Ivacaftor 150 mg q12h Group 4: VX-661 100 mg qd/Ivacaftor 150 mg q12h Group 5a: VX-661 150 mg qd Group 5b: VX-661 150 mg qd/Ivacaftor 150 mg q12h Group 6a: VX-661 100 mg qd/Ivacaftor 50 mg q12h Group 6d: VX-661 50 mg q12h/Ivacaftor 150 mg q12h Group 7: VX-661 100 mg qd

Name: Ivacaftor

Type: Drug
Group Labels: 8

Group 2b: VX-661 10 mg qd/Ivacaftor 150 mg q12h Group 3b: VX-661 30 mg qd/Ivacaftor 150 mg q12h Group 4: VX-661 100 mg qd/Ivacaftor 150 mg q12h Group 5b: VX-661 150 mg qd/Ivacaftor 150 mg q12h Group 6a: VX-661 100 mg qd/Ivacaftor 50 mg q12h Group 6d: VX-661 50 mg q12h/Ivacaftor 150 mg q12h Group 7: Placebo Group 7: VX-661 100 mg qd

Name: Placebo matched to VX-661

Type: Drug
Group Labels: 2

Group 1-6d Combined: Placebo Group 7: Placebo

Name: Placebo matched to ivacaftor

Type: Drug
Group Labels: 3

Group 1-6d Combined: Placebo Group 2a: VX-661 30 mg qd Group 3a: VX-661 100 mg qd


Primary Outcomes

Description: An AE is defined as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the Informed Consent Form is signed. AE includes serious as well as non-serious AEs. Serious Adverse Event (SAE) is any AE that results in any of the following: death; life-threatening condition; inpatient hospitalization or prolongation of hospitalization; persistent or significant disability or incapacity; congenital anomaly or birth defect; or other important medical event. Treatment-emergent adverse events are defined as adverse events that were reported or worsened on or after start of study drug through the Follow-up Visit (28 days after last dose of study drug) or premature discontinuation.

Measure: Safety as Determined by Adverse Events (AEs)

Time: Start of study drug through the Follow-up Visit (Up to Day 56)

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.

Measure: Change in Sweat Chloride From Baseline Through Study Day 28 for Group 1-5b

Time: Baseline through Day 28

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.

Measure: Change in Sweat Chloride From Baseline Through Study Day 28 for Group 6

Time: Baseline through Day 28

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.

Measure: Change in Sweat Chloride From Baseline Through Study Day 28 for Group 7

Time: Baseline through Day 28

Secondary Outcomes

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.

Measure: Change in Sweat Chloride From Baseline to Each Visit up to Study Day 28 for Group 1-5b

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.

Measure: Change in Sweat Chloride From Baseline to Each Visit up to Study Day 28 for Group 6

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.

Measure: Change in Sweat Chloride From Baseline to Each Visit up to Study Day 28 for Group 7

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Measure: Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 1-5b

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.

Measure: Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 6

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Measure: Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 7

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Measure: Change in FEV1 (Liter [L]) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 1-5b

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.

Measure: Change in FEV1 (L) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 6

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Measure: Change in FEV1 (L) From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 7

Time: Baseline, Day 7, Day 14, Day 21, Day 28

Description: The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

Measure: Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 1-5b

Time: Baseline, Day 14, Day 28

Description: The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.

Measure: Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 6

Time: Baseline, Day 14, Day 28

Description: The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

Measure: Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline to Each Visit and From Baseline Through Study Day 28 for Group 7

Time: Baseline, Day 14, Day 28

Description: Participants who received VX-661 monotherapy (Group 1, 2a, 3a and 5a) were analyzed for this outcome measure. PK analysis (AUC0-24h) was not planned for placebo reporting arms.

Measure: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661 After Administration of VX-661 Monotherapy

Time: Day 28

Description: Participants who received VX-661 in combination with Ivacaftor (Group 2b, 3b, 4, 5b, 6a, 6d and 7) were analyzed for this outcome measure. PK analysis was not planned for placebo reporting arms.

Measure: AUC0-24h of VX-661 and AUC0-12h of Ivacaftor After Administration of VX-661 in Combination With Ivacaftor

Time: Day 28

Purpose: Treatment

Allocation: Randomized

Parallel Assignment


There is one SNP

SNPs


1 G551D

Group 7 participants must have the F508del-CFTR mutation on 1 allele, and gating mutation G551D on the second allele and have been on their physician prescribed 150 mg KalydecoTM q12h (commercially available ivacaftor) for at least 28 days at the Screening Visit. --- G551D ---



HPO Nodes