NCT02797132

Brief Summary

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
62

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 2016

Shorter than P25 for phase_3

Geographic Reach
2 countries

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2016

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

May 25, 2016

Completed
19 days until next milestone

First Posted

Study publicly available on registry

June 13, 2016

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2017

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

October 30, 2018

Completed
Last Updated

October 30, 2018

Status Verified

September 1, 2018

Enrollment Period

1.3 years

First QC Date

May 25, 2016

Results QC Date

September 30, 2018

Last Update Submit

September 30, 2018

Conditions

Cystic Fibrosis

Keywords

CF

Outcome Measures

Primary Outcomes (2)

  • Part A: Pre-dose Concentration (Ctrough) of LUM and IVA

    Day 15

  • Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Day 1 up to Week 26

Secondary Outcomes (21)

  • Part A: Pre-dose Concentration (Ctrough) of LUM and IVA Metabolites

    Day 15

  • Part A: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Day 1 up to Day 25

  • Part B: Absolute Change From Baseline in Sweat Chloride at Week 24

    Baseline, Week 24

  • Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24

    Baseline, Week 24

  • Part B: Absolute Change From Baseline in Body Mass Index (BMI) For-Age Z-Score at Week 24

    Baseline, Week 24

  • +16 more secondary outcomes

Study Arms (1)

Lumacaftor/Ivacaftor (LUM/IVA)

EXPERIMENTAL

Part A (\<14 kg): Participants weighing less than (\<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (\>=14 kg): Participants weighing greater than or equal to (\>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (\<14 kg): Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (\>=14 kg): Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B.

Drug: LUM/IVA

Interventions

LUM/IVADRUG
Also known as: Orkambi, VX-809+VX-770
Lumacaftor/Ivacaftor (LUM/IVA)

Eligibility Criteria

Age2 Years - 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Subjects who weigh ≥8 kilogram (kg) without shoes and wearing light clothing at the Screening Visit
  • Subjects with confirmed diagnosis of CF at the Screening Visit
  • Subjects who are homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation

You may not qualify if:

  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
  • A standard 12-lead ECG demonstrating QTc \>450 millisecond (msec) at the Screening Visit.
  • History of solid organ or hematological transplantation.
  • Ongoing or prior participation in an investigational drug study (including studies investigating LUM and/or IVA) within 30 days of the Screening Visit.
  • History of cataract/lens opacity or evidence of cataract/lens opacity determined to be clinically significant by a licensed ophthalmologist during the ophthalmologic examination at the Screening Visit

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Unknown Facility

Palo Alto, California, United States

Location

Unknown Facility

Aurora, Colorado, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Indianapolis, Indiana, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

Kansas City, Missouri, United States

Location

Unknown Facility

Buffalo, New York, United States

Location

Unknown Facility

Chapel Hill, North Carolina, United States

Location

Unknown Facility

Cincinnati, Ohio, United States

Location

Unknown Facility

Cleveland, Ohio, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, United States

Location

Unknown Facility

Charleston, South Carolina, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Norfolk, Virginia, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Montreal, Canada

Location

Related Publications (1)

  • McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24.

    PMID: 30686767DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated
medicalinfo@vrtx.com
617-341-6777

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 25, 2016

First Posted

June 13, 2016

Study Start

May 1, 2016

Primary Completion

September 1, 2017

Study Completion

September 1, 2017

Last Updated

October 30, 2018

Results First Posted

October 30, 2018

Record last verified: 2018-09

Locations

US(17)CA(3)