Back to resultsA Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 3
Locations
Israel
Study Type
Interventional
Intervention
Ivacaftor
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
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:
- 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.
Sites / Locations
- Hadassah Medical Organization
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Sequence 1: First Ivacaftor (IVA) Then Placebo
Sequence 2: First Placebo Then IVA
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Full Information
NCT ID
NCT03068312
First Posted
February 24, 2017
Last Updated
February 13, 2020
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT03068312
Brief Title
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
Official Title
A Randomized, Double-blind, Placebo-controlled, Crossover Study to Evaluate the Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older and Have Either a 3849 + 10KB C→T or D1152H-CFTR Mutation
Study Type
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
July 18, 2017 (Actual)
Primary Completion Date
December 18, 2018 (Actual)
Study Completion Date
December 18, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
38 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Sequence 1: First Ivacaftor (IVA) Then Placebo
Arm Type
Experimental
Arm Description
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.
Arm Title
Sequence 2: First Placebo Then IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Ivacaftor
Other Intervention Name(s)
VX-770, IVA
Intervention Description
IVA 150 mg tablet.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to IVA tablet.
Primary Outcome Measure Information:
Title
Change in Lung Clearance Index 2.5 (LCI2.5)
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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:
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.
Facility Information:
Facility Name
Hadassah Medical Organization
City
Jerusalem
Country
Israel
12. IPD Sharing Statement
Citations:
PubMed Identifier
33095038
Citation
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.
Results Reference
derived
Learn more about this trial
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
We'll reach out to this number within 24 hrs