NCT02445053

Brief Summary

To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
75

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Apr 2015

Longer than P75 for all trials

Geographic Reach
3 countries

15 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2015

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

May 11, 2015

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 15, 2015

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2020

Completed
Last Updated

November 13, 2020

Status Verified

November 1, 2020

Enrollment Period

5.5 years

First QC Date

May 11, 2015

Last Update Submit

November 12, 2020

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis

Outcome Measures

Primary Outcomes (8)

  • Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment

    48 Months

  • Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment

    48 Months

  • Respiratory Microbiology - Percentage of patients with cultures positive for bacteria other than Pseudomonas aeruginosa and for fungi during Kalydeco treatment compared to the period before Kalydeco treatment

    48 Months

  • FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment

    48 Months

  • Measures of nutritional status: Absolute change in weight, weight-for-age Z score, body mass index (BMI), and BMI-for-age Z-score during Kalydeco treatment

    48 Months

  • Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment

    48 Months

  • Mortality: Incidence and cause of deaths

    48 Months

  • Organ transplantation: Incidence and reason for organ transplantations

    48 Months

Other Outcomes (1)

  • To explore the effect of Kalydeco treatment on Health-Related Quality of Life (HRQoL) in patients with CF and in caregivers of pediatric patients enrolled in the study.

    48 Months

Interventions

ivacaftorDRUG
Also known as: Kalydeco, VX-770

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Male and female patients with CF, age 6 years or older, who have 1 of the following CFTR mutations on at least 1 allele: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D

You may qualify if:

  • Male or female with confirmed diagnosis of CF16
  • At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D
  • Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent
  • Signed ICFs and, where appropriate, signed Assent Form
  • Able to understand the study requirements and comply with study data collection procedures

You may not qualify if:

  • Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment
  • Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation
  • History of organ transplantation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Unknown Facility

Bari, Italy

Location

Unknown Facility

Messina, Italy

Location

Unknown Facility

Milan, Italy

Location

Unknown Facility

Milan, Italy

Location

Unknown Facility

Naples, Italy

Location

Unknown Facility

Potenza, Italy

Location

Unknown Facility

Verona, Italy

Location

Unknown Facility

The Hague, South Holland, Netherlands

Location

Unknown Facility

Amsterdam, Netherlands

Location

Unknown Facility

Gronningen, Netherlands

Location

Unknown Facility

Heidelberglaan, Netherlands

Location

Unknown Facility

Nijmegen, Netherlands

Location

Unknown Facility

Rotterdam, Netherlands

Location

Unknown Facility

London, Greater London, United Kingdom

Location

Unknown Facility

Birmingham, West Midlands, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 11, 2015

First Posted

May 15, 2015

Study Start

April 1, 2015

Primary Completion

October 1, 2020

Study Completion

October 1, 2020

Last Updated

November 13, 2020

Record last verified: 2020-11

Locations

GB(2)IT(7)NL(6)