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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03068312
Recruitment Status : Completed
First Posted : March 1, 2017
Results First Posted : January 6, 2020
Last Update Posted : February 26, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Ivacaftor Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
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
IVA 150 mg tablet.
Other Names:
  • VX-770
  • IVA

Drug: Placebo
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.
Drug: Ivacaftor
IVA 150 mg tablet.
Other Names:
  • VX-770
  • IVA

Drug: Placebo
Placebo matched to IVA tablet.




Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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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
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.

Information from the National Library of Medicine

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


Locations
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Israel
Hadassah Medical Organization
Jerusalem, Israel
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] October 20, 2016
Statistical Analysis Plan  [PDF] October 19, 2018


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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03068312    
Other Study ID Numbers: VX16-770-127
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

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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
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action