A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

NCT ID: NCT03911713

Last Updated: 2022-01-25

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

77 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-17

Study Completion Date

2020-08-20

Brief Summary

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The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Ivacaftor

Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.

Group Type ACTIVE_COMPARATOR

IVA

Intervention Type DRUG

150-mg film-coated tablet for oral administration.

Placebo

Intervention Type DRUG

Placebos matched to VX-561.

VX-561: 25 mg

Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.

Group Type EXPERIMENTAL

VX-561

Intervention Type DRUG

VX-561 tablets for oral administration.

Placebo

Intervention Type DRUG

Placebo matched to IVA.

VX-561: 50 mg

Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.

Group Type EXPERIMENTAL

VX-561

Intervention Type DRUG

VX-561 tablets for oral administration.

Placebo

Intervention Type DRUG

Placebo matched to IVA.

VX-561: 150 mg

Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.

Group Type EXPERIMENTAL

VX-561

Intervention Type DRUG

VX-561 tablets for oral administration.

Placebo

Intervention Type DRUG

Placebo matched to IVA.

VX-561: 250 mg

Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.

Group Type EXPERIMENTAL

VX-561

Intervention Type DRUG

VX-561 tablets for oral administration.

Placebo

Intervention Type DRUG

Placebo matched to IVA.

Interventions

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VX-561

VX-561 tablets for oral administration.

Intervention Type DRUG

IVA

150-mg film-coated tablet for oral administration.

Intervention Type DRUG

Placebo

Placebo matched to IVA.

Intervention Type DRUG

Placebo

Placebos matched to VX-561.

Intervention Type DRUG

Other Intervention Names

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CTP-656 Deutivacaftor (D-IVA) VX-770 Ivacaftor

Eligibility Criteria

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Inclusion Criteria

* Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
* On ivacaftor therapy
* FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height

Exclusion Criteria

* History of clinically significant cirrhosis with or without portal hypertension
* History of solid organ or hematological transplantation
* Lung infection with organisms associated with a more rapid decline in pulmonary status
Minimum Eligible Age

18 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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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Banner University of Arizona Medical Center

Tucson, Arizona, United States

Site Status

Miller Children's Hospital / Long Beach Memorial

Long Beach, California, United States

Site Status

UCSF Gateway Medical Center

San Francisco, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status

Central Florida Pulmonary Group, PA

Orlando, Florida, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Cardinal Glennon Children's Hospital

St Louis, Missouri, United States

Site Status

Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

University of North Carolina Hospitals

Chapel Hill, North Carolina, United States

Site Status

UC Health Holmes

Cincinnati, Ohio, United States

Site Status

ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center

Toledo, Ohio, United States

Site Status

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Dell Children's Medical Group

Austin, Texas, United States

Site Status

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

University of Utah / Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, United States

Site Status

The Prince Charles Hospital

Chermside, , Australia

Site Status

Alfred Hospital

Melbourne, VIC, , Australia

Site Status

Mater Adult Hospital

South Brisbane, , Australia

Site Status

Westmead Hospital

Westmead, , Australia

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

Erlangen, , Germany

Site Status

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

Site Status

Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie

Halle, , Germany

Site Status

Pneumologisches Studienzentrum Muenchen-West

München, , Germany

Site Status

University Hospital Wuerzburg

Würzburg, , Germany

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Cork University Hospital

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

University Hospital Limerick (Adults)

Limerick, , Ireland

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status

St. James University Hospital

Leeds, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

Countries

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United States Australia Belgium Germany Ireland Netherlands United Kingdom

References

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Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23.

Reference Type DERIVED
PMID: 36842446 (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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2018-003970-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX18-561-101

Identifier Type: -

Identifier Source: org_study_id

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