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Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

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ClinicalTrials.gov Identifier: NCT03601637
Recruitment Status : Recruiting
First Posted : July 26, 2018
Last Update Posted : February 4, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: LUM Drug: IVA Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 23, 2018
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: Part A Cohort 1 [aged 18 to <24 months]
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
  • lumacaftor
  • VX-809

Drug: IVA
FDC granule (LUM/IVA)
Other Names:
  • ivacaftor
  • VX-770

Experimental: Part A Cohort 2 [12 to <18months]
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
  • lumacaftor
  • VX-809

Drug: IVA
FDC granule (LUM/IVA)
Other Names:
  • ivacaftor
  • VX-770

Experimental: Part B
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
  • lumacaftor
  • VX-809

Drug: IVA
FDC granule (LUM/IVA)
Other Names:
  • ivacaftor
  • VX-770




Primary Outcome Measures :
  1. Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of LUM and IVA [ Time Frame: from baseline through safety follow-up (up to 10 days after the last dose) ]
  2. Part B: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 2 weeks after the last dose) ]

Secondary Outcome Measures :
  1. Part A: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 10 days after the last dose) ]
  2. Part A: Average observed pre-dose concentrations (Ctrough) of LUM and IVA metabolites [ Time Frame: from baseline through safety follow-up (up to 10 days after the last dose) ]
  3. Part B: Absolute change in sweat chloride [ Time Frame: from baseline at Week 24 ]
  4. Part B: Average observed pre-dose concentration (Ctrough) of LUM, IVA, and their respective metabolites [ Time Frame: from baseline through safety follow-up (up to 2 weeks after the last dose) ]


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Ages Eligible for Study:   12 Months to 23 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.
  • Homozygous for F508del (F/F).

Key Exclusion Criteria:

  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
  • Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03601637


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

Locations
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United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35294
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
United States, Illinois
Ann & Robert Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
United States, Indiana
Riley Hospital for Children at Indiana University Health Recruiting
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
United States, Missouri
The Children's Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
United States, New York
Albany Medical College Recruiting
Albany, New York, United States, 12208
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Children's Medical Center of Dallas Recruiting
Dallas, Texas, United States, 75235
Cook Children's Medical Center Recruiting
Fort Worth, Texas, United States, 76104
United States, Utah
University of Utah / Primary Children's Medical Center Recruiting
Salt Lake City, Utah, United States, 84132
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
United States, Wisconsin
University of Wisconsin Hospital and Clinics Recruiting
Madison, Wisconsin, United States, 53792
Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital Recruiting
Montreal, Canada
British Columbia's Children's Hospital Recruiting
Vancouver, Canada
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03601637     History of Changes
Other Study ID Numbers: VX16-809-122
2017-004794-13 ( EudraCT Number )
First Posted: July 26, 2018    Key Record Dates
Last Update Posted: February 4, 2019
Last Verified: February 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action