A Phase 3 2-part Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis Homozygous for F508del
Study on Safety and Effects of Investigational Medications in Young Children with Cystic Fibrosis
Brief description of study.
Detailed description of study
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: cystic fibrosis
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Age: 1 years - 2 years
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Gender: All
Inclusion Criteria
Male and female subjects 1 to 2 years of age on Day 1 with cystic fibrosis, homozygous for F508del mutation
Weight at the Screening Visit must be within the weight limits as defined for the study drug
dose levels
Exclusion Criteria
History of any comorbidity reviewed at the Screening Visit that, in the opinion of the
investigator, might confound the results of the study or pose an additional risk in
administering LUM/IVA to the subject
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 (first dose of
LUM/IVA)
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
Use of restricted medication or food within specified duration before the first dose of
LUM/IVA
An adequate slit-lamp examination could not be conducted at the Screening Visit
ophthalmologic examination
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
The subject or a close relative of the subject is the investigator or a subinvestigator, research
assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of
the study
This study investigates the safety and effects of a combination of investigational medications in young children with Cystic Fibrosis, a genetic disorder that affects the lungs and digestive system. The purpose of this study is to evaluate how these medications are processed in the body of children aged 1 to less than 2 years, who have a specific genetic makeup known as homozygous for F508del.
Participants in the study will undergo various procedures to assess the effects and safety of the investigational medications. These procedures may include regular health check-ups, laboratory tests, and monitoring of any side effects to ensure the well-being of the participants.
- Who can participate: Children who are 1 to less than 2 years old with Cystic Fibrosis and have the F508del mutation can participate. Participants must meet specific weight requirements and should not have significant health issues that could affect the study results.
- Study details: Participants will receive the investigational medications and be monitored for safety and effectiveness. A placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine, will not be used in this study.
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