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

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ClinicalTrials.gov Identifier: NCT03601637
Recruitment Status : Completed
First Posted : July 26, 2018
Results First Posted : January 6, 2023
Last Update Posted : January 6, 2023
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 61 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 : September 7, 2018
Actual Primary Completion Date : October 29, 2021
Actual Study Completion Date : October 29, 2021

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: Part A: LUM/IVA
Participants weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
 Drug: LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
Other Names:
  • lumacaftor
  • VX-809

Drug: IVA
FDC granules (LUM/IVA).
Other Names:
  • ivacaftor
  • VX-770
Experimental: Part B: LUM/IVA
Participants weighing 7 to <9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
 Drug: LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
Other Names:
  • lumacaftor
  • VX-809

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


Outcome Measures
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Primary Outcome Measures :
  1. Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA [ Time Frame: Day 1 and Day 15 ]
  2. Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA [ Time Frame: Pre-dose at Day 8 and Day 15 ]
  3. Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: From Day 1 up to Week 26 ]

Secondary Outcome Measures :
  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: From Day 1 up to Day 25 ]
  2. Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA) [ Time Frame: Pre-dose at Day 8 and Day 15 ]
  3. Part B: Absolute Change in Sweat Chloride [ Time Frame: From Baseline at Week 24 ]
  4. Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA) [ Time Frame: Pre-dose at Day 15, Week 4, Week 12 and Week 24 ]

Eligibility Criteria
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Information from the National Library of Medicine

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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:

  • Participants 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 participants
  • 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): NCT03601637


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] July 29, 2020
Statistical Analysis Plan  [PDF] May 19, 2021

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: NCT03601637    
Other Study ID Numbers: VX16-809-122
2017-004794-13 ( EudraCT Number )
First Posted: July 26, 2018    Key Record Dates
Results First Posted: January 6, 2023
Last Update Posted: January 6, 2023
Last Verified: December 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