CompletedNCT03068312Updated Feb 26, 2020Results posted

A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

A Phase 3 interventional study of Ivacaftor and Placebo in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 1 site in Israel. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2020-02-26.

Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 38
Allocation: Randomized
Ages: 6 Years and older
Sex: All

Study summary

This study will evaluate the efficacy of ivacaftor treatment in subjects with CF 6 years of age and older who have a 3849 + 10KB C→T or D1152H CFTR mutation.

Conditions studied

Cystic Fibrosis

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In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 38 is close to the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.

Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 6 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Confirmed diagnosis of CF based on protocol-specified clinical features and at least one of the following: increased sweat chloride level, identification of 2 CF causing mutations, or demonstration of abnormal nasal epithelial ion transport.
A 3849 + 10KB C→T or D1152H mutation on at least 1 CFTR allele.
FEV1 ≥40% of predicted and ≤105% of predicted at screening.

Exclusion criteria

Exclusion Criteria:

A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation.
History of any illness or any clinical condition 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 subject.
Ongoing or prior participation in an investigational drug study within 30 days before the Screening Visit.
Protocol-specified abnormal laboratory values at the Screening Visit
For subjects \<18 years of age at the Screening Visit, evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination (OE) at the Screening Visit.
Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1.
Pregnant, breastfeeding, or planning to become pregnant during the study.
Sexually active subjects of reproductive potential must be willing to use appropriate contraception.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 38 participants (actual)

Study arms

Experimental
Sequence 1: First Ivacaftor (IVA) Then Placebo
Participants received IVA 150 milligram (mg) every 12 hours (q12h) for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.
Drug: Ivacaftor · Drug: Placebo
Experimental
Sequence 2: First Placebo Then IVA
Participants received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA 150 mg q12h for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.
Drug: Ivacaftor · Drug: Placebo

Interventions

DrugIvacaftor
IVA 150 mg tablet.
Also known as: VX-770, IVA
DrugPlacebo
Placebo matched to IVA tablet.

What researchers measure

Primary outcomes

Change in Lung Clearance Index 2.5 (LCI2.5)
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Time frame: From baseline through 8 weeks

Results

Posted Jan 6, 2020

Participant flow

Participant flow — Overall Study
Milestone	Sequence 1: First Ivacaftor (IVA) Then Placebo	Sequence 2: First Placebo Then IVA
Started	19	19
Completed	19	19
Not completed	0	0

Outcome measures

PrimaryChange in Lung Clearance Index 2.5 (LCI2.5)

LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Time frame:: From baseline through 8 weeks
Reported as:: Least squares mean · lung clearance index

Change in Lung Clearance Index 2.5 (LCI2.5)
lung clearance index	Placebo	Ivacaftor
Change in Lung Clearance Index 2.5 (LCI2.5)	0.20 ± 0.19	-0.46 ± 0.19

Statistical analysis

Placebo vs Ivacaftor · Least squares mean difference: -0.66 · 95% CI -1.10 to -0.21

Adverse events

Collected over From first dose of study drug up to safety follow-up visit (up to Week 28). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Placebo	0/38 (0%)	2/38 (5.3%)	19/38 (50%)
Ivacaftor	0/38 (0%)	1/38 (2.6%)	12/38 (31.6%)

Most frequent serious events

Most frequent serious events
Event	Placebo	Ivacaftor
Abortion spontaneousPregnancy, puerperium and perinatal conditions	0/38	1/38
PancreatitisGastrointestinal disorders	1/38	0/38
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations	1/38	0/38

Most frequent other events

Most frequent other events
Event	Placebo	Ivacaftor
Viral upper respiratory tract infectionInfections and infestations	9/38	1/38
Upper respiratory tract infectionInfections and infestations	6/38	3/38
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations	2/38	5/38
HaemoptysisRespiratory, thoracic and mediastinal disorders	2/38	3/38
HeadacheNervous system disorders	2/38	1/38
PyrexiaGeneral disorders	1/38	2/38
MalaiseGeneral disorders	2/38	0/38
Aphthous ulcerGastrointestinal disorders	2/38	0/38

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Sequence 1: First IVA Then Placebo	Sequence 2: First Placebo Then IVA	Total
Mean	32.6 ± 15.3	32.1 ± 15.6	32.3 ± 15.2

Sex: Female, Male
Sex: Female, Male(Participants)	Sequence 1: First IVA Then Placebo	Sequence 2: First Placebo Then IVA	Total
Female	10	10	20
Male	9	9	18

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Sequence 1: First IVA Then Placebo	Sequence 2: First Placebo Then IVA	Total
Hispanic or Latino	0	0	0
Not Hispanic or Latino	19	19	38
Unknown or Not Reported	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Sequence 1: First IVA Then Placebo	Sequence 2: First Placebo Then IVA	Total
American Indian or Alaska Native	0	0	0
Asian	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	0	0	0
White	19	19	38
More than one race	0	0	0
Unknown or Not Reported	0	0	0

Lung Clearance Index 2.5 (LCI2.5)
Lung Clearance Index 2.5 (LCI2.5)(lung clearance index)	Sequence 1: First IVA Then Placebo	Sequence 2: First Placebo Then IVA	Total
Mean	12.74 ± 4.04	13.19 ± 5.45	12.96 ± 4.74

Study locations

1 site

Hadassah Medical Organization
Jerusalem, Israel

References and documents

Publications

Kerem E, Cohen-Cymberknoh M, Tsabari R, Wilschanski M, Reiter J, Shoseyov D, Gileles-Hillel A, Pugatsch T, Davies JC, Short C, Saunders C, DeSouza C, Sullivan JC, Doyle JR, Chandarana K, Kinnman N. Ivacaftor in People with Cystic Fibrosis and a 3849+10kb C-->T or D1152H Residual Function Mutation. Ann Am Thorac Soc. 2021 Mar;18(3):433-441. doi: 10.1513/AnnalsATS.202006-659OC. PubMed 33095038 ↗

Study documents

Study protocol · Oct 20, 2016
Statistical analysis plan · Oct 19, 2018

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Feb 26, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT03068312
Lead sponsor: Vertex Pharmaceuticals Incorporated
Responsible party: Sponsor
First posted: Mar 1, 2017
Start date: Jul 18, 2017
Primary completion: Dec 18, 2018
Completion: Dec 18, 2018
Results posted: Jan 6, 2020
Last update: Feb 26, 2020

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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