A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
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|ClinicalTrials.gov Identifier: NCT03068312|
Recruitment Status : Completed
First Posted : March 1, 2017
Results First Posted : January 6, 2020
Last Update Posted : February 26, 2020
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|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Ivacaftor Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||38 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Actual Study Start Date :||July 18, 2017|
|Actual Primary Completion Date :||December 18, 2018|
|Actual Study Completion Date :||December 18, 2018|
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.
IVA 150 mg tablet.
Placebo matched to IVA tablet.
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.
IVA 150 mg tablet.
Placebo matched to IVA tablet.
- Change in Lung Clearance Index 2.5 (LCI2.5) [ Time Frame: From baseline through 8 weeks ]LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
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|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03068312
|Hadassah Medical Organization|
Documents provided by Vertex Pharmaceuticals Incorporated:
|Responsible Party:||Vertex Pharmaceuticals Incorporated|
|Other Study ID Numbers:||
2017-000457-39 ( EudraCT Number )
|First Posted:||March 1, 2017 Key Record Dates|
|Results First Posted:||January 6, 2020|
|Last Update Posted:||February 26, 2020|
|Last Verified:||February 2020|
|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|
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action