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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

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ClinicalTrials.gov Identifier: NCT02722057
Recruitment Status : Active, not recruiting
First Posted : March 29, 2016
Last Update Posted : August 5, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief 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. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.

Condition or disease
Cystic Fibrosis

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.

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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Other
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
Study Start Date : December 2015
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Group/Cohort
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.



Primary Outcome Measures :
  1. 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

  2. Pulmonary exacerbations, use of IV antibiotics [ Time Frame: 36 Months ]
    Pulmonary exacerbation data will be collected as recorded in the registry.

  3. 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

  4. Death or transplantation [ Time Frame: 36 months ]
    Death will be collected from the registry database.

  5. Hospitalizations [ Time Frame: 36 Months ]
    Hospitalizations will be collected from the registry database.

  6. 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

  7. Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus) [ Time Frame: 36 Months ]
    Data on microorganisms as recorded in the registry will be evaluated



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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
Criteria

Inclusion Criteria:

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

  • 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

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02722057     History of Changes
Other Study ID Numbers: VX15-770-122
First Posted: March 29, 2016    Key Record Dates
Last Update Posted: August 5, 2019
Last Verified: August 2019

Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action