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 |
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
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
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University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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Alfred I. duPont Hospital for Children Nemours began more than 70 years ago with the...
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Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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University of Wisconsin In achievement and prestige, the University of Wisconsin–Madison has long been recognized...
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Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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