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

NCT01946412 | Completed Phase 3 Results DMC

• 1 other identifier: VX11-770-109
• Study Type: interventional
• Target: 33
• Locations: 3 countries
• Sites: 16

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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  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.

  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)

Secondary Outcomes (8)

• Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84

  Baseline (study 108), Week 24, 48, 72 and 84 (study 109)

• Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84

  Baseline (study 109), Week 24, 48, 72 and 84 (study 109)

• Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84

  Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)

• Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84

  Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)

• Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84

  Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)

Study Arms (2)

Observational Arm

NO INTERVENTION

Ivacaftor

EXPERIMENTAL

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

Interventions

Ivacaftor DRUG

Also known as: VX-770, Kalydeco

Ivacaftor

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• \. 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.

You may not qualify if:

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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (16)

Unknown Facility

Birmingham, Alabama, United States

Location

Unknown Facility

Denver, Colorado, United States

Location

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Indianapolis, Indiana, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Grand Rapids, Michigan, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

Kansas City, Missouri, United States

Location

Unknown Facility

Omaha, Nebraska, United States

Location

Unknown Facility

Salt Lake City, Utah, United States

Location

Unknown Facility

Richmond, Virginia, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Unknown Facility

Edinburgh, United Kingdom

Location

Unknown Facility

Liverpool, United Kingdom

Location

Unknown Facility

London, United Kingdom

Location

Related Publications (1)

• Rosenfeld M, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Chilvers M, Higgins M, Tian S, Cooke J, Davies JC; KLIMB study group. An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB). J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30.

  PMID: 31053538 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Study Officials

• Adebayo Lawal, M.D.

  Vertex Pharmaceuticals Incorporated

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2013
• First Posted: September 19, 2013
• Study Start: December 1, 2013
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: February 1, 2017
• Results First Posted: February 1, 2017

Record last verified: 2016-12

Locations

GB (3); US (12); CA (1)