Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

NCT01707290 | Completed Phase 3 Results

• 2 other identifiers: VX12-770-1122012-000389-39
• Study Type: interventional
• Target: 125
• Locations: 4 countries
• Sites: 38

Brief Summary

The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm

  AE: any untoward medical occurrence in a participants 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 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, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.

  Day 1 up to Week 108 (Study 112)

Secondary Outcomes (6)

• Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104

  Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

• Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104

  Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

• Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104

  Baseline, Week 2, 24, 48 and 104 (Study 112)

• Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104

  Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)

• Number of Pulmonary Exacerbations Events

  Through Week 104 (Study 112)

Study Arms (2)

Ivacaftor

EXPERIMENTAL

Participants who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to Ivacaftor tablet orally, every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks.

Drug: Ivacaftor

Observational

NO INTERVENTION

Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years.

Interventions

Ivacaftor DRUG

150 mg tablet, oral use, every 12 hours (q12h)

Also known as: Kalydeco, VX-770

Ivacaftor

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study.
• Participants from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study.
• Participants of childbearing potential entering the ivacaftor arm must not be pregnant.
• Participants entering the ivacaftor arm must be willing to comply with contraception requirements.

You may not qualify if:

• History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the Participant.
• Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A.
• Evidence of cataract or lens opacity at or before the Day 1 Visit.

Sponsors & Collaborators

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

Study Sites (38)

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Birmingham, Alabama, United States

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Palo Alto, California, United States

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Denver, Colorado, United States

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Hartford, Connecticut, United States

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Tampa, Florida, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Iowa City, Iowa, United States

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Lexington, Kentucky, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Grand Rapids, Michigan, United States

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Minneapolis, Minnesota, United States

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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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New York, New York, United States

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Syracuse, New York, United States

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Valhalla, New York, United States

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Chapel Hill, North Carolina, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Charleston, South Carolina, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Fort Worth, Texas, United States

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Houston, Texas, United States

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Salt Lake City, Utah, United States

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Richmond, Virginia, United States

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Seattle, Washington, United States

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Morgantown, West Virginia, United States

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Madison, Wisconsin, United States

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Leuven, Belgium

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Montpellier, France

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Paris, France

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Belfast, United Kingdom

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Edinburgh, United Kingdom

Location

Related Publications (2)

• Pilewski JM, De Boeck K, Nick JA, Tian S, DeSouza C, Higgins M, Moss RB. Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations. Pulm Ther. 2020 Dec;6(2):303-313. doi: 10.1007/s41030-020-00129-2. Epub 2020 Sep 23.

  PMID: 32965659 DERIVED

• Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

  PMID: 26070913 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

• Joseph Pilewski, MD

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

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: October 9, 2012
• First Posted: October 16, 2012
• Study Start: February 1, 2013
• Primary Completion: April 1, 2016
• Study Completion: April 1, 2016
• Last Updated: May 12, 2017
• Results First Posted: May 12, 2017

Record last verified: 2017-04

Locations

US (33); BE (1); FR (2); GB (2)