A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02742519
• Other study ID #: VX15-770-123
• Secondary ID: 2015-001267-39
• Status: Terminated
• Phase: Phase 3
• Start date: May 2016
• Est. completion date: August 2017

Study information

• Verified date: October 2018
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 14
• Est. completion date: August 2017
• Est. primary completion date: August 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 5 Years

Eligibility

Inclusion Criteria:

• Male or female with confirmed diagnosis of CF.

• Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.

• Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.

Exclusion Criteria:

• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1

• Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)

• Abnormal liver function, at Screening, defined as =3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin

• History of solid organ or hematological transplantation

• Any clinically significant "non-CF-related" illness within 2 weeks before Day 1

• Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1

• Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ivacaftor

• Placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)	LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.	Baseline Through Week 8 for each treatment period, Up to 24 Weeks
Secondary	Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8	Serum samples were collected for evaluation of change in immunoreactive trypsinogen levels at Week 8.	Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary	Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8	Fecal elastase-1 was used clinically to diagnose pancreatic exocrine insufficiency in participants with cystic fibrosis.	Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary	Absolute Change From Baseline in Weight at Week 8		Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary	Absolute Change From Baseline in Body Mass Index (BMI) at Week 8	BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).	Baseline and Week 8 of each treatment period, Up to 24 Weeks
Secondary	Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)		Baseline up to Month 15