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Completed

NCT Number: NCT01946412

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Vancouver, British Columbia, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

(Ivacaftor Arm):

  • Completed the last study visit of the treatment period of the previous study (NCT01705145)
  • 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
  • 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):

  • 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):

  • Subjects who prematurely discontinued from the previous study
  • 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
  • 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
  • Subjects receiving commercially-available ivacaftor treatment
  • Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study

Exclusion criteria

(Observational Arm)

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

Treatment and study plan

Ivacaftor

Drug

Other names: VX-770, Kalydeco

Primary outcomes

  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 outcomes

  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).

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Collaborators

  • Cystic Fibrosis Foundation

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Sep 19, 2013
Registry last updated
Feb 1, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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