A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04353817
• Other study ID #: VX19-445-116
• Secondary ID: 2019-003554-86
• Status: Completed
• Phase: Phase 3
• Start date: June 19, 2020
• Est. completion date: May 17, 2021

Study information

• Verified date: July 2022
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 121
• Est. completion date: May 17, 2021
• Est. primary completion date: May 17, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 11 Years

Eligibility

Key Inclusion Criteria:

• Heterozygous for the F508del mutation (F/MF)

• Forced expiratory volume in 1 second (FEV1) value greater than equal to(=) 70%

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension

• Lung infection with organisms associated with a more rapid decline in pulmonary status

• Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration qd in the morning.
• IVA
Tablet for oral administration qd in the evening.

Other:
• Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
• Placebo (matched to IVA)
Placebo matched to IVA for oral administration qd in the evening.

Locations

Country	Name	City	State
Australia	Telethon Kids Institute	Nedlands
Australia	Queensland Children's Hospital	South Brisbane
Australia	The Children's Hospital at Westmead	Westmead
Canada	McGill University Health Centre, Glen Site, Montreal Children's Hospital	Montréal
Canada	The Hospital for Sick Children	Toronto
Canada	British Columbia Children's Hospital	Vancouver
Denmark	Juliane Marie Center, Rigshospitalet	Copenhagen
France	Groupe Hospitaler Pellegrin, CHU De Bordeaux	Bordeaux cedex
France	CHU Lyon - Hopital Femme Mere-Enfant	Bron Cedex
France	Hopital Robert Debre	Paris
France	Hopital Necker, Enfants Malades	Paris Cedex 15
France	Centre de Perharidy	Roscoff cedex
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Universitätsklinikum Essen	Essen
Germany	Johann Wolfgang Goethe University	Frankfurt
Germany	Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin	Gießen
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin	Heidelberg
Germany	Universitaetsklinkum Koeln, CF-Studienzentrum	Koeln
Israel	Hadassah University Hospital Mount Scopus	Jerusalem
Israel	Schneider Children's Medical Center	Petah Tikva
Netherlands	Universitair Medisch Centrum Groningen	Groningen
Netherlands	Erasmus Medical Center / Sophia Children's Hospital	Rotterdam
Spain	Hospital Universitari Vall d Hebron	Barcelona
Spain	Hospital Virgen de la Arrixaca	Murcia
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zurich
United Kingdom	University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital	Bristol
United Kingdom	Children's Hospital of Wales	Cardiff
United Kingdom	Royal Hospital for Sick Children	Edinburgh
United Kingdom	Alder Hey Children's NHS Foundation Trust	Liverpool
United Kingdom	Great Ormond Street Hospital for Sick Children	London
United Kingdom	Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital	London
United Kingdom	Southampton General Hospital	Southampton

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Canada,  Denmark,  France,  Germany,  Israel,  Netherlands,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change in Lung Clearance Index 2.5 (LCI2.5)	The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.	From Baseline Through Week 24
Secondary	Absolute Change in Sweat Chloride (SwCl)	Sweat samples were collected using an approved collection device.	From Baseline Through Week 24
Secondary	Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		Day 1 up to Week 28