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NCT Number: NCT02722057

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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Sex eligibility

All sexes

Study type

Observational

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  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

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2019
Study completion
2019
First posted
Mar 29, 2016
Registry last updated
Feb 5, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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