Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04537793
• Other study ID #: VX20-445-111
• Secondary ID: 2020-002251-38
• Status: Completed
• Phase: Phase 3
• Start date: November 19, 2020
• Est. completion date: June 3, 2022

Study information

• Verified date: June 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination therapy in CF subjects 2 through 5 years of age.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 83
• Est. completion date: June 3, 2022
• Est. primary completion date: June 3, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 5 Years

Eligibility

Key Inclusion Criteria:

• Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)

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 granules for oral administration.
• IVA
Granules for oral administration.

Locations

Country	Name	City	State
Australia	Telethon Kids Institute	Nedlands
Australia	The Royal Children's Hospital	Parkville, VIC
Australia	Queensland Children's Hospital	South Brisbane
Canada	The Hospital for Sick Children	Toronto
Canada	British Columbia Children's Hospital	Vancouver
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie	Essen
United Kingdom	Alder Hey Children's NHS Foundation Trust	Liverpool
United Kingdom	Royal Brompton Hospital	London
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	NC TraCS Institute - CTRC University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Stanford University	Palo Alto	California
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University School of Medicine / St. Louis Children's Hospital	Saint Louis	Missouri
United States	Seattle Children's Hospital	Seattle	Washington
United States	Banner University of Arizona Medical Center	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites		From Day 1 through Day 15
Primary	Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		From Day 1 up to Day 43
Primary	Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		From Day 1 up to Week 28
Secondary	Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites		From Day 1 through Week 16
Secondary	Part B: Absolute Change in Sweat Chloride (SwCl)	Sweat samples were collected using an approved collection device.	From Baseline through Week 24
Secondary	Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)	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