Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating 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: NCT01946412
Recruitment Status : Completed
First Posted : September 19, 2013
Results First Posted : February 1, 2017
Last Update Posted : February 1, 2017
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
Study Start Date : December 2013
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
No Intervention: Observational Arm
Experimental: Ivacaftor

Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:

  • 50 mg q12h for subjects 2 to <6 years of age and <14 kg,
  • 75 mg q12h for subjects 2 to <6 years of age and ≥ 14 kg, or
  • 150 mg q12h for subjects ≥ 6 years of age.
Drug: Ivacaftor
Other Names:
  • VX-770
  • Kalydeco




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing) ]
    AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent.


Secondary Outcome Measures :
  1. Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 [ Time Frame: Baseline (study 108), Week 24, 48, 72 and 84 (study 109) ]
    Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).

  2. Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84 [ Time Frame: Baseline (study 109), Week 24, 48, 72 and 84 (study 109) ]
    Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of >=15 microliter was required for determination of sweat chloride. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).

  3. Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145)

  4. Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).

  5. Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).

  6. Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).

  7. Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).

  8. Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria (Ivacaftor Arm):

  1. Completed the last study visit of the treatment period of the previous study (NCT01705145)
  2. Hematology, serum chemistry, and vital signs results on Day 1 with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
  3. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned

Inclusion Criteria (Observational Arm):

1. Subjects who completed their assigned study drug treatment in the previous study (NCT01705145) and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.

Exclusion Criteria (Ivacaftor Arm):

  1. Subjects who prematurely discontinued from the previous study
  2. History of any illness or 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
  3. Subjects with a history of study treatment intolerance as observed in their previous study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
  4. Subjects receiving commercially-available ivacaftor treatment
  5. Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study

Exclusion Criteria: (Observational Arm)

1. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.


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): NCT01946412


Locations
Layout table for location information
United States, Alabama
Birmingham, Alabama, United States
United States, Colorado
Denver, Colorado, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, Michigan
Grand Rapids, Michigan, United States
United States, Minnesota
Minneapolis, Minnesota, United States
United States, Missouri
Kansas city, Missouri, United States
United States, Nebraska
Omaha, Nebraska, United States
United States, Utah
Salt Lake City, Utah, United States
United States, Virginia
Richmond, Virginia, United States
United States, Washington
Seattle, Washington, United States
Canada, British Columbia
Vancouver, British Columbia, Canada
United Kingdom
Edinburgh, United Kingdom
Liverpool, United Kingdom
London, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation
Investigators
Layout table for investigator information
Study Director: Adebayo Lawal, M.D. Vertex Pharmaceuticals Incorporated

Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01946412     History of Changes
Other Study ID Numbers: VX11-770-109
First Posted: September 19, 2013    Key Record Dates
Results First Posted: February 1, 2017
Last Update Posted: February 1, 2017
Last Verified: December 2016
Keywords provided by Vertex Pharmaceuticals Incorporated:
Cystic Fibrosis
Additional relevant MeSH terms:
Layout table for MeSH terms
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