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A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

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ClinicalTrials.gov Identifier: NCT02742519
Recruitment Status : Terminated (Low enrollment.)
First Posted : April 19, 2016
Last Update Posted : October 6, 2017
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
Study Start Date : May 2016
Actual Primary Completion Date : August 2017
Actual Study Completion Date : August 2017

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: Part 1-Sequence 1
ivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2
Drug: ivacaftor
Other Name: VX-770

Drug: Placebo
Experimental: Part 1 - Sequence 2
placebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2
Drug: ivacaftor
Other Name: VX-770

Drug: Placebo
Experimental: Part 2: ivacaftor
open label period
Drug: ivacaftor
Other Name: VX-770




Primary Outcome Measures :
  1. Part 1: Absolute change from baseline in LCI2.5 through 8 weeks of treatment [ Time Frame: through 8 weeks ]

Secondary Outcome Measures :
  1. Part 1: Absolute change from baseline in serum levels of immunoreactive trypsinogen at 8 weeks of treatment [ Time Frame: at 8 Weeks ]
  2. Part 1: Absolute change from baseline in fecal elastase-1 at 8 weeks of treatment [ Time Frame: at 8 Weeks ]
  3. Part 1: Absolute change from baseline in weight at 8 weeks of treatment [ Time Frame: at 8 weeks ]
  4. Absolute change from baseline in body mass index (BMI) at 8 weeks of treatment [ Time Frame: at 8 weeks ]
  5. Parts 1 and 2: Safety, as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: at Weeks 8, 48, 96, and 144 ]
  6. Part 2: Absolute change from baseline in LCI2.5 [ Time Frame: at Weeks 48, 96, 144 ]
  7. Par 2: Absolute change from baseline in CT scan [ Time Frame: at Week 96 ]
  8. Part 2: Absolute change from baseline in serum levels of immunoreactive trypsinogen [ Time Frame: at Weeks 48, 96, and 144 ]
  9. Part 2: Absolute change from baseline in fecal elastase-1 [ Time Frame: at Weeks 48, 96, and 144 ]
  10. Part 2: Absolute change from baseline in weight-for-age z-score [ Time Frame: at Weeks 48, 96, and 144 ]
  11. Part 2: Absolute change from baseline in height-for-age z-score [ Time Frame: at Weeks 48, 96, and 144 ]
  12. Part 2: Absolute change from baseline in BMI-for-age z-score [ Time Frame: at Weeks 48, 96, and 144 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female with confirmed diagnosis of CF.
  • Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
  • Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.

Exclusion Criteria:

  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
  • Abnormal liver function, at Screening, defined as ≥3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
  • History of solid organ or hematological transplantation
  • Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
  • Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
  • Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening

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): NCT02742519


Locations
Australia, Victoria
Parkville, Victoria, Australia
Australia
South Brisbane, Australia
Subiaco, Australia
Westmead, Australia
Canada, Ontario
Toronto, Ontario, Canada
United Kingdom
London, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02742519     History of Changes
Other Study ID Numbers: VX15-770-123
2015-001267-39 ( EudraCT Number )
First Posted: April 19, 2016    Key Record Dates
Last Update Posted: October 6, 2017
Last Verified: October 2017

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