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Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

NCT ID: NCT02797132

Last Updated: 2018-10-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-31

Study Completion Date

2017-09-30

Brief Summary

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This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.

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A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lumacaftor/Ivacaftor (LUM/IVA)

Part A (\<14 kg): Participants weighing less than (\<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part A (\>=14 kg): Participants weighing greater than or equal to (\>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A.

Part B (\<14 kg): Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Part B (\>=14 kg): Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

Interventions

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LUM/IVA

Intervention Type DRUG

Other Intervention Names

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Orkambi VX-809+VX-770

Eligibility Criteria

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Inclusion Criteria

* Subjects who weigh ≥8 kilogram (kg) without shoes and wearing light clothing at the Screening Visit
* Subjects with confirmed diagnosis of CF at the Screening Visit
* Subjects who are homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation

Exclusion Criteria

* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
* A standard 12-lead ECG demonstrating QTc \>450 millisecond (msec) at the Screening Visit.
* History of solid organ or hematological transplantation.
* Ongoing or prior participation in an investigational drug study (including studies investigating LUM and/or IVA) within 30 days of the Screening Visit.
* History of cataract/lens opacity or evidence of cataract/lens opacity determined to be clinically significant by a licensed ophthalmologist during the ophthalmologic examination at the Screening Visit
Minimum Eligible Age

2 Years

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Palo Alto, California, United States

Site Status

Aurora, Colorado, United States

Site Status

Chicago, Illinois, United States

Site Status

Indianapolis, Indiana, United States

Site Status

Boston, Massachusetts, United States

Site Status

Minneapolis, Minnesota, United States

Site Status

Kansas City, Missouri, United States

Site Status

Buffalo, New York, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Cleveland, Ohio, United States

Site Status

Columbus, Ohio, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Charleston, South Carolina, United States

Site Status

Houston, Texas, United States

Site Status

Norfolk, Virginia, United States

Site Status

Seattle, Washington, United States

Site Status

Vancouver, British Columbia, Canada

Site Status

Toronto, Ontario, Canada

Site Status

Montreal, , Canada

Site Status

Countries

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United States Canada

References

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McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24.

Reference Type DERIVED
PMID: 30686767 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-001004-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX15-809-115

Identifier Type: -

Identifier Source: org_study_id

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