Cystic FibrosisPhase 3NCT03601637
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
A Phase 3 study testing LUM and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 27 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 to 23 months
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- LUM and IVA
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 27 sitesin 2 countries
- Study dates
- 7 Sep 2018to 29 Oct 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 23 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
27 sites in 2 countries.
27 sites in 2 countries
United States24 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Medical Center of Dallas
Dallas, Texas, 75235
Cook Children's Medical Center
Fort Worth, Texas, 76104
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
The Children's Mercy Hospital
Kansas City, Missouri, 64108
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Yale New Haven Medical Center
New Haven, Connecticut, 06511
Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, 63104
Nemours / Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803
Wake Forest University School of Medicine - Brenner Children's Hospital
Winston-Salem, North Carolina, 27157
Canada3 sites
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal
The Hospital for Sick Children
Toronto
British Columbia's Children's Hospital
Vancouver
About this study
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
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).
- Conditions
- Cystic Fibrosis
- Being tested
- LUM, IVA
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUMDrug
Also called lumacaftor, VX-809
Fixed Dose Combination (FDC) granules (LUM/IVA).
IVADrug
Also called ivacaftor, VX-770
FDC granules (LUM/IVA).
| Study group | Role | What they receive |
|---|---|---|
| Part A: LUM/IVA | Experimental | 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. Receives: Drug: LUM; Drug: IVA |
| Part B: LUM/IVA | Experimental | 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. Receives: Drug: LUM; Drug: IVA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Measured: Day 1 and Day 15
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Measured: Pre-dose at Day 8 and Day 15
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 up to Week 26
Secondary outcomes (4)
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 up to Day 25
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Measured: Pre-dose at Day 8 and Day 15
Part B: Absolute Change in Sweat Chloride
Measured: From Baseline at Week 24
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Measured: Pre-dose at Day 15, Week 4, Week 12 and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2018
- Study start7 Sep 2018
- Primary completion29 Oct 2021
- Study completion29 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03601637
- Sponsor's protocol ID
- VX16-809-122
- Other IDs
- 2017-004794-13, 2017
- Status last verified
- Dec 2022
- First submitted
- 18 Jul 2018
- Last updated
- 6 Jan 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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
Publications
Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PubMed 37983082Rayment 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.
PubMed 35771568Southern 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.
PubMed 33331662
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03601637
- Acronym / sponsor ID
- VX16-809-122
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 7 Sep 2018 (actual)
- Primary completion
- 29 Oct 2021 (actual)
- Study completion
- 29 Oct 2021 (actual)
- First posted
- 26 Jul 2018 (actual)
- Results
- Posted 6 Jan 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part A: LUM/IVA | Experimental | Drug: LUM, Drug: IVAParticipants 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. |
| Part B: LUM/IVA | Experimental | Drug: LUM, Drug: IVAParticipants 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. |
Who may qualify
- Ages
- 12 to 23 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- 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 (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LUMAlso: lumacaftor, VX-809Fixed Dose Combination (FDC) granules (LUM/IVA). | Drug | Part A: LUM/IVA, Part B: LUM/IVA |
| IVAAlso: ivacaftor, VX-770FDC granules (LUM/IVA). | Drug | Part A: LUM/IVA, Part B: LUM/IVA |
Primary outcome
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Time frame: Day 1 and Day 15
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Time frame: Pre-dose at Day 8 and Day 15
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
4 secondary outcomes
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
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
Part B: Absolute Change in Sweat Chloride
Time frame: From Baseline at Week 24
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37983082Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Derived from this study: PubMed 35771568Rayment 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.
- Derived from this study: PubMed 33331662Southern 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.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing LUM and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 27 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jan 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA (time frame: Day 1 and Day 15). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03601637, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2023.