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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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
LUM
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Months - 23 Months (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at Birmingham
  • Arkansas Children's Hospital
  • Stanford University
  • Children's Hospital Colorado
  • Yale New Haven Medical Center
  • Nemours / Alfred I. duPont Hospital for Children
  • Children's Healthcare of Atlanta
  • Ann & Robert Lurie Children's Hospital of Chicago
  • Riley Hospital for Children at Indiana University Health
  • Johns Hopkins Hospital
  • Boston Children's Hospital
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
  • The Children's Mercy Hospital
  • Cardinal Glennon Children's Hospital - St. Louis University
  • University of Rochester Medical Center
  • University of North Carolina Hospitals
  • Wake Forest University School of Medicine - Brenner Children's Hospital
  • Cincinnati Children's Hospital Medical Center
  • Children's Hospital of Philadelphia
  • Children's Medical Center of Dallas
  • Cook Children's Medical Center
  • University of Utah / Primary Children's Medical Center
  • Seattle Children's Hospital
  • University of Wisconsin Hospital and Clinics
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • The Hospital for Sick Children
  • British Columbia's Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Part A: LUM/IVA

Part B: LUM/IVA

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Part B: Absolute Change in Sweat Chloride
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)

Full Information

First Posted
July 18, 2018
Last Updated
December 14, 2022
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT03601637
Brief Title
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Completed
Study Start Date
September 7, 2018 (Actual)
Primary Completion Date
October 29, 2021 (Actual)
Study Completion Date
October 29, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
61 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Part A: LUM/IVA
Arm Type
Experimental
Arm Description
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.
Arm Title
Part B: LUM/IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
LUM
Other Intervention Name(s)
lumacaftor, VX-809
Intervention Description
Fixed Dose Combination (FDC) granules (LUM/IVA).
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
ivacaftor, VX-770
Intervention Description
FDC granules (LUM/IVA).
Primary Outcome Measure Information:
Title
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Time Frame
Day 1 and Day 15
Title
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Time Frame
Pre-dose at Day 8 and Day 15
Title
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 Measure Information:
Title
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
Title
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
Title
Part B: Absolute Change in Sweat Chloride
Time Frame
From Baseline at Week 24
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
23 Months
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Yale New Haven Medical Center
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06511
Country
United States
Facility Name
Nemours / Alfred I. duPont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Ann & Robert Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Riley Hospital for Children at Indiana University Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Cardinal Glennon Children's Hospital - St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
University of North Carolina Hospitals
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Wake Forest University School of Medicine - Brenner Children's Hospital
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Medical Center of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University of Utah / Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montreal
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia's Children's Hospital
City
Vancouver
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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
Citations:
PubMed Identifier
35771568
Citation
Rayment JH, Asfour F, Rosenfeld M, Higgins M, Liu L, Mascia M, Paz-Diaz H, Tian S, Zahigian R, McColley SA. A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
Results Reference
derived
PubMed Identifier
33331662
Citation
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(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived

Learn more about this trial

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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