Cystic FibrosisPhase 3NCT03277196
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
A Phase 3 study testing IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 29 sites in 6 countries.
Trial summary
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation. Read more about this study
- Who is it for?
- Ages 0 to 24 months
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- IVA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 86 participants
- Where
- 29 sitesin 6 countries
- Study dates
- 16 Aug 2017to 2 Oct 2023
- 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 0 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Ivacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B:
- Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
- As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).
- Ivacaftor Arm: Participants Not From Study 124 Part B:
- Confirmed diagnosis of CF, or 2 CF-causing mutations.
- An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 2 years of age and older.
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- As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.
- Observational Arm:
- Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.
Who cannot join
- Ivacaftor Arm: Participants From Study 124 Part B:
- History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant.
- Participants receiving commercially available ivacaftor treatment
- Ivacaftor Arm: Participants Not From Study 124 Part B:
- History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant
- An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
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- Abnormal liver function at screening
- Hemoglobin <9.5 g/dL at screening
- History of solid organ or hematological transplantation
- Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1
- Observational Arm:
- Receiving ivacaftor treatment
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
29 sites in 6 countries.
29 sites in 6 countries
United States17 sites
Center for Advanced Pediatrics
Atlanta, Georgia, 30329
John Hopkins Hospital
Baltimore, Maryland, 21287
Billings Clinic Hospital
Billings, Montana, 59101
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Northwestern University
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202
Children's Mercy Hospital
Kansas City, Missouri, 64108
University of Wisconsin
Madison, Wisconsin, 53792
Childrens's Hospitals and Clinics of Minnnesota
Minneapolis, Minnesota, 55404
Nemours Children's Hospital
Orlando, Florida, 32827
Stanford University
Palo Alto, California, 94034
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Seattle Children's Hospital
Seattle, Washington, 98105
Alfred I DuPont Hospital for Children
Wilmington, Delaware, 19803
United Kingdom6 sites
Paediatric Clinical Research Facility
Edinburgh
Alder Hey Children's Alder Hey Children's NHS Foundation Trust
Liverpool
Great Ormond Street Hospital for Sick Children
London
Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital
London
Royal Manchester Children's Hospital
Manchester
Oxford University Hospitals NHS Trust, John Radcliffe Hospital
Oxford
Ireland3 sites
Children's Health Ireland at Crumlin
Dublin
Children's University Hospital Temple Street
Dublin
University Hospital Limerick
Limerick
Germany1 site
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
Heidelberg
About this study
Official title: A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.
- Conditions
- Cystic Fibrosis
- Being tested
- 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.
IVADrug
Also called VX-770, ivacaftor
Granules for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor Arm | Experimental | Participants less than (<) 24 months of age and weighing 5 to less than (<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to <14 kg received 50 mg IVA q12h, and those weighing 14 to <25 kg received 75 mg IVA q12h.
Participants more than or equal (>=) 24 months of age and weighing <14 kg received 50 mg IVA q12h, and those weighing more than or equal to (>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks.
Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight. Receives: Drug: IVA |
| Observational Arm | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Measured: Day 1 up to Week 120
Secondary outcomes (1)
Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Measured: From Baseline at Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2017
- Study start16 Aug 2017
- Primary completion2 Oct 2023
- Study completion2 Oct 2023
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.
- 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 86 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
- NCT03277196
- Sponsor's protocol ID
- VX15-770-126
- Other IDs
- 2017-001379-21, 2017
- Status last verified
- Oct 2024
- First submitted
- 6 Sep 2017
- Last updated
- 23 Oct 2024
- 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
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
- ClinicalTrials.gov ID
- NCT03277196
- Acronym / sponsor ID
- VX15-770-126
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 16 Aug 2017 (actual)
- Primary completion
- 2 Oct 2023 (actual)
- Study completion
- 2 Oct 2023 (actual)
- First posted
- 8 Sep 2017 (actual)
- Results
- Posted 23 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor Arm | Experimental | Drug: IVAParticipants less than (<) 24 months of age and weighing 5 to less than (<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to <14 kg received 50 mg IVA q12h, and those weighing 14 to <25 kg received 75 mg IVA q12h. Participants more than or equal (>=) 24 months of age and weighing <14 kg received 50 mg IVA q12h, and those weighing more than or equal to (>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks. Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight. |
| Observational Arm | No intervention | – |
Who may qualify
- Ages
- 0 to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
- As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).
- Confirmed diagnosis of CF, or 2 CF-causing mutations.
- An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 2 years of age and older.
- As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.
Key exclusion criteria (13 listed)
- History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant.
- Participants receiving commercially available ivacaftor treatment
- History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant
- An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
- Abnormal liver function at screening
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 |
|---|---|---|
| IVAAlso: VX-770, ivacaftorGranules for oral administration. | Drug | Ivacaftor Arm |
Primary outcome
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Day 1 up to Week 120
1 secondary outcome
Absolute Change in Sweat Chloride
Time frame: From Baseline at Week 96
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
29 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 29 sites in 6 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 9 inclusion and 13 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 23 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, United Kingdom, Ireland, Australia, Canada and 1 more country. 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 23 Oct 2024). 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?
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs (time frame: Day 1 up to Week 120). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Oct 2024.