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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02565914




Registration number
NCT02565914
Ethics application status
Date submitted
18/09/2015
Date registered
1/10/2015
Date last updated
28/09/2023

Titles & IDs
Public title
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
Scientific title
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Secondary ID [1] 0 0
2014-004827-29
Secondary ID [2] 0 0
VX14-661-110
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - TEZ/IVA
Treatment: Drugs - IVA

Experimental: TEZ/IVA - Part A: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 milligram (mg)/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks.

Part B: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108,109,112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks.

Part C: Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks.


Treatment: Drugs: TEZ/IVA
Fixed dose tablet for oral administration.

Treatment: Drugs: IVA
Tablet for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Day 1 up to Week 100
Primary outcome [2] 0 0
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs
Timepoint [2] 0 0
Day 1 up to Week 100
Primary outcome [3] 0 0
Part C: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [3] 0 0
Day 1 up to Week 196
Secondary outcome [1] 0 0
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set
Timepoint [1] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [2] 0 0
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set
Timepoint [2] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [3] 0 0
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set
Timepoint [3] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [4] 0 0
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set
Timepoint [4] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [5] 0 0
Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set
Timepoint [5] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [6] 0 0
Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set
Timepoint [6] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [7] 0 0
Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set
Timepoint [7] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [8] 0 0
Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set
Timepoint [8] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [9] 0 0
Part A: Number of Pulmonary Exacerbation (PEx) Events for 106/110 PEx Analysis Set
Timepoint [9] 0 0
From Baseline up to Study 110 Week 96
Secondary outcome [10] 0 0
Part A: Number of Pulmonary Exacerbation (PEx) Events for 108/110 PEx Analysis Set
Timepoint [10] 0 0
From Baseline up to Week 96
Secondary outcome [11] 0 0
Part A: Absolute Change in Body Mass Index (BMI) for 106/110 Efficacy Set
Timepoint [11] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [12] 0 0
Part A: Absolute Change in Body Mass Index (BMI) for 108/110 Efficacy Set
Timepoint [12] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [13] 0 0
Part A: Absolute Change in Body Mass Index (BMI) for 103/110 Efficacy Set
Timepoint [13] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [14] 0 0
Part A: Absolute Change in Body Mass Index (BMI) for Study 111/110 Efficacy Set
Timepoint [14] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [15] 0 0
Part A: Absolute Change in BMI Z-score for 106/110 Efficacy Set
Timepoint [15] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [16] 0 0
Part A: Absolute Change in BMI Z-score for 108/110 Efficacy Set
Timepoint [16] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [17] 0 0
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 106/110 Efficacy Set
Timepoint [17] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [18] 0 0
Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 108/110 Efficacy Set
Timepoint [18] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [19] 0 0
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/110 Efficacy Set
Timepoint [19] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [20] 0 0
Part A: Absolute Change in Body Weight for Study 106/110 Efficacy Set
Timepoint [20] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [21] 0 0
Part A: Absolute Change in Body Weight for 108/110 Efficacy Set
Timepoint [21] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [22] 0 0
Part A: Absolute Change in Body Weight for 103/110 Efficacy Set
Timepoint [22] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [23] 0 0
Part A: Absolute Change in Body Weight for 111/110 Efficacy Set
Timepoint [23] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [24] 0 0
Part A: Absolute Change in Body Weight Z-score for 106/110 Efficacy Set
Timepoint [24] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [25] 0 0
Part A: Absolute Change in Body Weight Z-score for 108/110 Efficacy Set
Timepoint [25] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [26] 0 0
Part A: Absolute Change in Height Z-score for 106/110 Efficacy Set
Timepoint [26] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [27] 0 0
Part A: Absolute Change in Height Z-score for 108/110 Efficacy Set
Timepoint [27] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [28] 0 0
Part A: Time-to-first Pulmonary Exacerbation (PEx) for 106/110 PEx Analysis Set
Timepoint [28] 0 0
96 weeks
Secondary outcome [29] 0 0
Part A: Time-to-first Pulmonary Exacerbation (PEx) for 108/110 PEx Analysis Set
Timepoint [29] 0 0
96 weeks
Secondary outcome [30] 0 0
Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA)
Timepoint [30] 0 0
Week 24
Secondary outcome [31] 0 0
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [31] 0 0
From Baseline at Study 110 Week 96
Secondary outcome [32] 0 0
Part B: Absolute Change in Body Mass Index (BMI)
Timepoint [32] 0 0
From Baseline at Week 96
Secondary outcome [33] 0 0
Part B: Absolute Change in BMI Z-score
Timepoint [33] 0 0
From Baseline at Week 96
Secondary outcome [34] 0 0
Part B: Number of Pulmonary Exacerbation (PEx) Events
Timepoint [34] 0 0
From Baseline up to Week 96

Eligibility
Key inclusion criteria
Part A:

* Participants entering the Treatment Cohort must meet all of the following criteria:
* Elect to enroll in the Treatment Cohort
* Completed study drug Treatment Period in a parent study (NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111) or study drug treatment and the Safety Follow up Visit for participants from NCT02508207.
* Willing to remain on a stable CF regimen through the Safety Follow-up Visit.
* Participants re-enrolling in the Part A Treatment Cohort must meet all of the following criteria:

* Previously received at least 4 weeks of study drug before discontinuing in Part A of Study NCT02565914 to participate in another qualified Vertex study.
* Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part A Study NCT02565914.
* Participants entering the Part A Observational Cohort must meet the following criteria:

* <18 years of age (age on the date of informed consent/assent in the parent study)
* Completed study drug Treatment Period in a parent study or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207, but do not elect to enroll in the NCT02565914 Treatment Cohort; or
* Received at least 4 weeks of study drug treatment and completed visits up to the last scheduled visit of the Treatment Period of a parent study (and the Safety Follow up Visit for participants from NCT02508207), but do not meet eligibility criteria for enrollment into the Treatment Cohort

Part B:

Participants who meet all of the following inclusion criteria will be eligible for Part B.

* Did not withdraw consent from the parent study or Part A of Study NCT02565914.
* Completed study drug treatment during the Treatment Period in Part A of - Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Study NCT02565914.

Participants re enrolling in Part B must meet all of the following criteria:

* Previously received at least 4 weeks of study drug before discontinuing Study NCT02565914 to participate in another qualified Vertex study, which is defined as a Vertex study of investigational CFTR modulators that allows participation of participants in Study NCT02565914.
* Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part B.
* Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit in Part B.

Part C:

* Participants who meet all of the following inclusion criteria will be eligible for Part C.
* Did not withdraw consent from Part B of Study NCT02565914.
* Completed study drug treatment during Part B of NCT02565914.
* Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Part C.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 to the subject.
* Pregnant and nursing females.
* Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
* History of drug intolerance in the parent study that would pose an additional risk to the subject.
* Participation in an investigational drug trial (including studies investigating VX-661/ivacaftor or lumacaftor/ivacaftor) other than the parent studies of NCT02565914 or other eligible Vertex studies investigating VX-661 in combination with ivacaftor, or use of a commercially available CFTR modulator.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/08/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/12/2022
Sample size
Target
Accrual to date
Final
1131
Recruitment in Australia
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- Chermside
Recruitment hospital [2] 0 0
- Melbourne
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- South Brisbane
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- Westmead
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- Chermside
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- Melbourne
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- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
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Stoke-on-Trent

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744), VX14-661-106 (Study 106, NCT02347657), VX14-661-107 (Study 107, NCT02516410), VX14-661-108 (Study 108, NCT02392234), VX14-661-109 (Study 109, NCT02412111), VX14-661-111 (Study 111, NCT02508207), VX15-661-112 (NCT02730208), and VX16-661-114 (NCT03150719). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.
Trial website
https://clinicaltrials.gov/study/NCT02565914
Trial related presentations / publications
Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE; VX14-661-110 study group. Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study. Lancet Respir Med. 2021 Jul;9(7):733-746. doi: 10.1016/S2213-2600(20)30510-5. Epub 2021 Feb 10. Erratum In: Lancet Respir Med. 2021 Apr;9(4):e38. doi: 10.1016/S2213-2600(21)00122-3.
Public notes

Contacts
Principal investigator
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Contact person for public queries
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Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02565914