Cystic FibrosisNCT02722057
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
An observational study of Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated.
Trial summary
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 368 participants
- Where
- No sites listed yet
- Study dates
- Dec 2015to Dec 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Non Interventional Cohort - Patients Enrolled in the US CFF Patient Registry Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009
Who may be able to join
- Non Interventional Cohort
- Male or female with confirmed diagnosis of CF
- Must have at least 1 allele of the R117H-CFTR mutation
- Enrolled in the US CFF Patient Registry
- With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016
- Historical Cohort
Show all 9Show fewer
- Patients with CF in the CFF Patient Registry as of 01 January 2009
- Must have at least 1 allele of the R117H-CFTR mutation
- Patients with no evidence of any prior Kalydeco exposure
Who cannot join
None listed.
About this study
Official title: A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
Read the detailed description
Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. The study also includes retrieval of retrospective data entered into the registry for 36 months before the initiation of Kalydeco treatment, from patients matched for Non-Interventional Cohorts. This will permit a within-group comparison of outcomes before and after Kalydeco treatment for effectiveness and safety. The interventional cohort will not be utilized.
- Conditions
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - Interventional | The Interventional cohort will not be utilized. | |
| Cohort 2 - Non Interventional | A Non-Interventional Cohort comprising pediatric (<18 years of age) and adult R117H-CFTR patients treated with commercially-available Kalydeco. | |
| Cohort 3 - Historical | A Historical Cohort comprising data from an earlier time period for pediatric (<18 years of age) and adult patients with the R117H-CFTR mutation who have never been exposed to Kalydeco and matched on age, gender, and lung function to patients in the Non-Interventional Cohort. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC
Measured: 36 Months
Pulmonary exacerbations, use of IV antibiotics
Pulmonary exacerbation data will be collected as recorded in the registry.
Measured: 36 Months
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)
Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived
Measured: 36 Months
Death or transplantation
Death will be collected from the registry database.
Measured: 36 months
Hospitalizations
Hospitalizations will be collected from the registry database.
Measured: 36 Months
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)
Information for the above shown CF-related complications as recorded in the registry will be evaluated
Measured: 36 Months
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)
Data on microorganisms as recorded in the registry will be evaluated
Measured: 36 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2016 (estimated)
- Study startDec 2015
- Primary completionDec 2019
- Study completionDec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 368 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
- NCT02722057
- Sponsor's protocol ID
- VX15-770-122
- Status last verified
- Feb 2020
- First submitted
- 22 Mar 2016
- Last updated
- 5 Feb 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort
- ClinicalTrials.gov ID
- NCT02722057
- Acronym / sponsor ID
- VX15-770-122
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 368 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Dec 2015 (actual)
- Primary completion
- Dec 2019 (actual)
- Study completion
- Dec 2019 (actual)
- First posted
- 29 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
Detailed description
Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. The study also includes retrieval of retrospective data entered into the registry for 36 months before the initiation of Kalydeco treatment, from patients matched for Non-Interventional Cohorts. This will permit a within-group comparison of outcomes before and after Kalydeco treatment for effectiveness and safety. The interventional cohort will not be utilized.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - Interventional | – | –The Interventional cohort will not be utilized. |
| Cohort 2 - Non Interventional | – | –A Non-Interventional Cohort comprising pediatric (<18 years of age) and adult R117H-CFTR patients treated with commercially-available Kalydeco. |
| Cohort 3 - Historical | – | –A Historical Cohort comprising data from an earlier time period for pediatric (<18 years of age) and adult patients with the R117H-CFTR mutation who have never been exposed to Kalydeco and matched on age, gender, and lung function to patients in the Non-Interventional Cohort. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Non Interventional Cohort - Patients Enrolled in the US CFF Patient Registry Historical Cohort - Patients with CF in the CFF Patient Registry as of 01 January 2009
Key inclusion criteria (9 listed)
- Non Interventional Cohort
- Male or female with confirmed diagnosis of CF
- Must have at least 1 allele of the R117H-CFTR mutation
- Enrolled in the US CFF Patient Registry
- With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
Time frame: 36 Months
Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC
Pulmonary exacerbations, use of IV antibiotics
Time frame: 36 Months
Pulmonary exacerbation data will be collected as recorded in the registry.
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)
Time frame: 36 Months
Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived
Death or transplantation
Time frame: 36 months
Death will be collected from the registry database.
Hospitalizations
Time frame: 36 Months
Hospitalizations will be collected from the registry database.
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)
Time frame: 36 Months
Information for the above shown CF-related complications as recorded in the registry will be evaluated
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)
Time frame: 36 Months
Data on microorganisms as recorded in the registry will be evaluated
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2020.
Related trials for Cystic Fibrosis
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 2
Phase 2 Study to Assess the Safety and Efficacy of ANG003
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Recruiting
Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
Cystic Fibrosis (CF), Bronchiectasis, Non-Cystic Fibrosis
Not yet recruitingObservational study
Vascular Health and Function in Cystic Fibrosis
Cystic Fibrosis, Endothelial Dysfunction
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
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.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated.
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 0 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 5 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC]) (time frame: 36 Months). The study lists 7 primary outcomes in all. 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 5 Feb 2020.