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Long-term Safety of Lumacaftor/Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04235140
Recruitment Status : Active, not recruiting
First Posted : January 21, 2020
Last Update Posted : December 1, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This is a Phase 3, multicenter, open-label and roll-over study in subjects who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 52 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
Actual Study Start Date : February 24, 2020
Estimated Primary Completion Date : February 2024
Estimated Study Completion Date : February 2024

Resource links provided by the National Library of Medicine

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

Arms and Interventions
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Arm Intervention/treatment
Experimental: LUM/IVA
Subjects will receive LUM/IVA for 96 weeks.
 Drug: LUM/IVA
LUM/IVA granules for oral administration
Other Names:
  • lumacaftor/ivacaftor
  • VX-809/VX-770


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Up to 120 weeks ]

Secondary Outcome Measures :
  1. Absolute change in sweat chloride [ Time Frame: From Baseline at Week 96 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   12 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects From Study VX16-809-122 Part B (Study 122)

    • Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B

  • Subjects Not From Study 122

    • Subjects will be 1 to less than 2 years of age
    • Homozygous for the F508del mutation (F/F)

Key Exclusion Criteria:

  • Any clinically significant laboratory abnormalities 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.

Contacts and Locations
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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): NCT04235140


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04235140    
Other Study ID Numbers: VX19-809-124
First Posted: January 21, 2020    Key Record Dates
Last Update Posted: December 1, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.

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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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Cystic Fibrosis
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