Clinical trial NCT03277196: A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2017-09-08
Enrollment: 86
Sex: ALL
Minimum age (years): 0
Maximum age (years): 24
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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 participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.


Eligibility criteria: Inclusion Criteria:

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

* Participants 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: Participants 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. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 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: Participants 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 participant.
* Participants receiving commercially available ivacaftor treatment

Ivacaftor Arm: Participants 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 participant
* 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.

Primary outcomes:
measure: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
timeFrame: Day 1 up to Week 120
Secondary outcomes:
measure: Absolute Change in Sweat Chloride
timeFrame: From Baseline at Week 96
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Wilmington
country: United States
facility: Alfred I DuPont Hospital for Children
state: Delaware
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Center for Advanced Pediatrics
state: Georgia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University Health
state: Indiana
city: Baltimore
country: United States
facility: John Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: Childrens's Hospitals and Clinics of Minnnesota
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Billings
country: United States
facility: Billings Clinic Hospital
state: Montana
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
city: Dublin
country: Ireland
facility: Children's Health Ireland at Crumlin
city: Dublin
country: Ireland
facility: Children's University Hospital Temple Street
city: Limerick
country: Ireland
facility: University Hospital Limerick
city: Edinburgh
country: United Kingdom
facility: Paediatric Clinical Research Facility
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's Alder Hey Children's NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Sick Children
city: London
country: United Kingdom
facility: Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Trust, John Radcliffe Hospital
Study officials:
References: