A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation



Status:Recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:Any
Updated:5/30/2018
Start Date:August 16, 2017
End Date:June 7, 2021
Contact:Medical Information
Email:medicalinfo@vrtx.com
Phone:617-341-6777

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A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an
observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in
subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have
an approved Ivacaftor-Responsive mutation


Inclusion Criteria:

Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:

- Subjects transitioning from Study 124 Part B must have completed the last study visit
of Study 124 Part B.

- As judged by the investigator, parent or legal guardian must be able to understand
protocol requirements, restrictions, and instructions; and must sign the informed
consent form (ICF).

Ivacaftor Arm: Subjects Not From Study 124 Part B:

- Confirmed diagnosis of CF, or 2 CF-causing mutations.

- An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible
in countries/regions where ivacaftor is approved for use in subjects 2 years of age
and older.

- As judged by the investigator, parent or legal guardian must be able to understand
protocol requirements, restrictions, and instructions; and must sign the ICF.

Observational Arm:

- Received ivacaftor treatment in Study 124 Part B and elected not to enroll or
ineligible to enroll in the ivacaftor arm of Study 126.

Exclusion Criteria:

Ivacaftor Arm: Subjects From Study 124 Part B:

- 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
ivacaftor to the subject.

- Subjects receiving commercially available ivacaftor treatment

Ivacaftor Arm: Subjects Not From Study 124 Part B:

- 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
ivacaftor to the subject

- An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes
in therapy for pulmonary disease within 4 weeks of Day 1

- Abnormal liver function at screening

- Hemoglobin <9.5 g/dL at screening

- History of solid organ or hematological transplantation

- Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1

Observational Arm:

- Receiving ivacaftor treatment

Other protocol defined Inclusion/Exclusion criteria may apply.
We found this trial at
17
sites
6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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1600 Rockland Road
Wilmington, Delaware 19803
(302) 651-4200
Alfred I. duPont Hospital for Children Nemours began more than 70 years ago with the...
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Atlanta, GA
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Baltimore, Maryland 21287
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Chicago, IL
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Indianapolis, IN
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Madison, Wisconsin 53792
(608) 263-2400
University of Wisconsin In achievement and prestige, the University of Wisconsin–Madison has long been recognized...
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Palo Alto, California 94304
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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501 Stanley Street
South Brisbane, 4101
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