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Trial record 1 of 1 for:    VX16-152-102
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A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

This study is currently recruiting participants.
See Contacts and Locations
Verified January 2017 by Vertex Pharmaceuticals Incorporated
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02951195
First received: October 26, 2016
Last updated: April 12, 2017
Last verified: January 2017
  Purpose
This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in Triple Combination (TC) with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF), or who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del).

Condition Intervention Phase
Cystic Fibrosis Drug: VX-152 Drug: Tezacaftor Drug: Ivacaftor Drug: Triple Placebo Drug: Placebo for VX-152 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double Blind, Controlled Study to Evaluate the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline up to 8 Weeks ]

Secondary Outcome Measures:
  • Absolute change in sweat chloride concentrations [ Time Frame: from baseline at Day 15 ]
  • Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline at Day 15 ]
  • Relative change in ppFEV1 [ Time Frame: from baseline at Day 15 ]
  • Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Day 15 ]
  • Maximum observed concentration (Cmax) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (μg/mL) [ Time Frame: Day 1 through Day 15 ]
  • Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (μg.h/mL) [ Time Frame: Day 1 through Day 15 ]
  • Observed pre-dose concentration (Ctrough) of VX-152, TEZ, M1-TEZ, IVA, and M1-IVA (μg/mL) [ Time Frame: Day 1 through Day 15 ]

Estimated Enrollment: 60
Study Start Date: November 2016
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Heterozygous F508del/MF Cohort 1A: Triple Combination (TC)

VX-152 100 milligrams (mg) administered every 12 hours (q12h). TEZ 100 mg once daily (qd). IVA 150 mg q12h.

Morning Dose: VX-152 + TEZ/IVA

Evening Dose: VX-152 + IVA

Drug: VX-152 Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Placebo Comparator: Heterozygous F508del/MF Cohort 1A: TC
Placebos matched to VX-152, TEZ/IVA, and IVA.
Drug: Triple Placebo
Experimental: Heterozygous F508del/MF Cohort 1B: TC

To be initiated after blinded review of Cohort 1A, if supported by safety and PK Data.

The dosage of VX-152 may be adjusted based on data from Cohort 1A.

Morning Dose: VX-152 + TEZ/IVA

Evening Dose: VX-152 + IVA

Drug: VX-152 Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Placebo Comparator: Heterozygous F508del/MF Cohort 1B: Triple Placebo
Placebos matched to VX-152, TEZ/IVA, and IVA.
Drug: Triple Placebo
Experimental: Heterozygous F508del/MF Cohort 1C: TC

To be initiated after blinded review of Cohort 1B, if supported by safety and PK Data.

The dosage of VX-152 to be determined based on data from Cohort 1B.

Morning Dose: VX-152 + TEZ/IVA

Evening Dose: VX-152 + IVA

Drug: VX-152 Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Placebo Comparator: Heterozygous F508del/MF Cohort 1C: Triple Placebo
Placebos matched to VX-152, TEZ/IVA, and IVA.
Drug: Triple Placebo
Experimental: Homozygous F508del/F508del Cohort 2A: TC

To be initiated after blinded review of Cohort 1A or Cohort 1B, if supported by safety and PK Data.

The dosage of VX-152 to be determined based on data from Cohort 1A (and from Cohort 1B, if applicable.)

Morning Dose: VX-152 + TEZ/IVA

Evening Dose: VX-152 + IVA

The experimental period will be preceded by a 4-week run-in period and followed by a 2-week washout period, during both of which subjects will receive:

TEZ 100 mg administered once daily (qd). IVA 150 mg q12h.

Drug: VX-152 Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Active Comparator: Homozygous F508del/F508del Cohort 2A: TEZ/IVA

To be initiated after blinded review of Cohort 1A or Cohort 1B, if supported by safety and PK Data.

Morning Dose: Placebo + TEZ/IVA

Evening Dose: Placebo + IVA

The active comparator period will be preceded by a 4-week run-in period and followed by a 2-week washout period, during both of which subjects will receive:

TEZ 100 mg administered once daily (qd). IVA 150 mg q12h.

Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Drug: Placebo for VX-152
Experimental: Homozygous F508del/F508del Cohort 2B: TC

To be initiated after blinded review of Cohort 2A, if supported by safety and PK Data.

The dosage of VX-152 to be determined based on data from Cohorts 1A, 1B, and 2B, as applicable.

Morning Dose: VX-152 + TEZ/IVA

Evening Dose: VX-152 + IVA

The experimental period will be preceded by a 4-week run-in period and followed by a 2-week washout period, during both of which subjects will receive:

TEZ 100 mg administered once daily (qd). IVA 150 mg q12h.

Drug: VX-152 Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Drug: Placebo for VX-152
Active Comparator: Homozygous F508del/F508del Cohort 2B: TEZ/IVA

To be initiated after blinded review of Cohort 1A or Cohort 1B, if supported by safety and PK Data.

Morning Dose: Placebo + TEZ/IVA Evening Dose: Placebo + IVA

The active comparator period will be preceded by a 4-week run-in period and followed by a 2-week washout period, during both of which subjects will receive:

TEZ 100 mg administered once daily (qd). IVA 150 mg q12h.

Drug: Tezacaftor
Other Names:
  • TEZ
  • VX-661
Drug: Ivacaftor
Other Names:
  • IVA
  • VX-770
  • Kalydeco
Drug: Placebo for VX-152

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Willing and able to comply with scheduled visits, treatment pan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
  • Body weight ≥35 kg.
  • Sweat chloride value ≥ 60 mmol/L from test results obtained during screening.
  • Subjects must have an eligible CFTR genotype:

    • Cohorts 1A, 1B, 1C: Heterozygous for F508del and a minimal function mutation known or predicted not to respond to TEZ and/or IVA.
    • Cohorts 2A, 2B: Homozygous for F508del.
  • Subjects must have an FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height at the Screening Visit.
  • Stable CF disease as judged by the investigator.
  • Willing to remain on a stable CF medication regimen through the planned end of treatment or if applicable the Safety Follow-up Visit.

Exclusion Criteria:

  • History of any comorbidity 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.
  • History of cirrhosis with portal hypertension.
  • Risk factors for Torsade de Pointes.
  • History of hemolysis.
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening.
  • Clinically significant abnormal laboratory values at screening.
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug.
  • Lung infection with organisms associated with a more rapid decline in pulmonary status.
  • An acute illness not related to CF within 14 days before the first dose of study drug.
  • A standard digital ECG demonstrating QTc >450 msec at screening.
  • History of solid organ or hematological transplantation.
  • History or evidence of cataract or lens opacity determined to be clinically significant by the ophthalmologist or optometrist, based on the ophthalmologic examination during the Screening Period.
  • History of alcohol or drug abuse in the past year, including but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator.
  • Ongoing or prior participation in an investigational drug study with certain exceptions.
  • Use of commercially available CFTR modulator within 14 days before screening (applies only to Cohorts 1A, 1B, and 1C).
  • Pregnant or nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02951195

Contacts
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

Locations
United States, Alabama
Recruiting
Birmingham, Alabama, United States
United States, California
Recruiting
La Jolla, California, United States
Recruiting
Los Angeles, California, United States
United States, Florida
Recruiting
Orlando, Florida, United States
United States, Illinois
Recruiting
Chicago, Illinois, United States
Recruiting
Peoria, Illinois, United States
United States, Missouri
Recruiting
Saint Louis, Missouri, United States
United States, North Carolina
Recruiting
Chapel Hill, North Carolina, United States
United States, Ohio
Recruiting
Akron, Ohio, United States
Recruiting
Cleveland, Ohio, United States
United States, Oregon
Recruiting
Portland, Oregon, United States
United States, Pennsylvania
Recruiting
Philadelphia, Pennsylvania, United States
United States, South Carolina
Recruiting
Charleston, South Carolina, United States
United States, Texas
Recruiting
Fort Worth, Texas, United States
Recruiting
Houston, Texas, United States
United States, Wisconsin
Recruiting
Madison, Wisconsin, United States
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02951195     History of Changes
Other Study ID Numbers: VX16-152-102
2016-003049-27 ( EudraCT Number )
Study First Received: October 26, 2016
Last Updated: April 12, 2017

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on August 18, 2017