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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

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: NCT03601637
Recruitment Status : Completed
First Posted : July 26, 2018
Last Update Posted : November 29, 2021
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE July 18, 2018
First Posted Date  ICMJE July 26, 2018
Last Update Posted Date November 29, 2021
Actual Study Start Date  ICMJE August 23, 2018
Actual Primary Completion Date October 29, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 15, 2020)
  • Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of LUM and IVA [ Time Frame: from baseline through Day 15 ]
  • 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) ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 18, 2018)
  • 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) ]
  • 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) ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 15, 2020)
  • 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) ]
  • Part A: Average observed pre-dose concentrations (Ctrough) of LUM and IVA metabolites [ Time Frame: from baseline through Day 15 ]
  • Part B: Absolute change in sweat chloride [ Time Frame: from baseline at Week 24 ]
  • Part B: Average observed pre-dose concentration (Ctrough) of LUM, IVA, and their respective metabolites [ Time Frame: from baseline through Week 24 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 18, 2018)
  • 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) ]
  • 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) ]
  • Part B: Absolute change in sweat chloride [ Time Frame: from baseline at Week 24 ]
  • 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) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • Experimental: Part A Cohort 1 [aged 18 to <24 months]
    Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
    Interventions:
    • Drug: LUM
    • Drug: IVA
  • Experimental: Part A Cohort 2 [12 to <18months]
    Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
    Interventions:
    • Drug: LUM
    • Drug: IVA
  • Experimental: Part B
    Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
    Interventions:
    • Drug: LUM
    • Drug: IVA
Publications * Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12:CD010966. doi: 10.1002/14651858.CD010966.pub3.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 27, 2021)
60
Original Estimated Enrollment  ICMJE
 (submitted: July 18, 2018)
40
Actual Study Completion Date  ICMJE October 29, 2021
Actual Primary Completion Date October 29, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Months to 23 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03601637
Other Study ID Numbers  ICMJE VX16-809-122
2017-004794-13 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date November 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP