Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02070744
• Date of registration: 21/02/2014
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
• Scientific title: A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension
• Date of first enrolment: March 2014
• Target sample size: 40
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02070744
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Homozygous for the F508del CFTR mutation

- FEV1 =40% and =90% of predicted normal for age, sex, and height

- Stable CF disease as judged by the investigator

Exclusion Criteria:

- History of any comorbidity that, in the opinion of the investigator, might confound
the results of the study or pose an additional risk in administering study drug to the
participant

- Pregnant and nursing females: Females of childbearing potential must have a negative
pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE
Phase (whichever was applicable)

- Sexually active participants of reproductive potential who are not willing to follow
the contraception requirements

- The participant or a close relative of the participant is the investigator or sub
investigator, research assistant, pharmacist, study coordinator, or other staff
directly involved with the conduct of the study.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: VX-661

Drug: Ivacaftor

Drug: Placebo matched to Ivacaftor

Drug: Placebo matched to VX-661

Primary Outcome(s)

OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs [Time Frame: Baseline (OLE Phase) up to 364 days]

PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Baseline (PC Phase) up to 112 days]

Secondary Outcome(s)

OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40 [Time Frame: Baseline (OLE Phase), Through Week 40]

OLE Phase: Absolute Change From Baseline in Body Weight at Week 40 [Time Frame: Baseline (OLE Phase), Week 40]

PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12 [Time Frame: Baseline (PC Phase), Week 12]

PC Phase: Absolute Change From Baseline in Body Weight at Week 12 [Time Frame: Baseline (PC Phase), Week 12]

PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661 [Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85]

OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40 [Time Frame: Baseline (OLE Phase), Through Week 40]

PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12 [Time Frame: Baseline (PC Phase), Through Week 12]

PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA [Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85]

PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA [Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85]

OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40 [Time Frame: Baseline (OLE Phase), Through Week 40]

PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12 [Time Frame: Baseline (PC Phase), Through Week 12]

PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12 [Time Frame: Baseline (PC Phase), Through Week 12]

PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA [Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85]

OLE Phase: Absolute Change From Baseline BMI at Week 40 [Time Frame: Baseline (OLE Phase), Week 40]

OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40 [Time Frame: Baseline (OLE Phase), Through Week 40]

PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12 [Time Frame: Baseline (PC Phase), Through Week 12]

Secondary ID(s)

VX13-661-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 13/04/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02070744