A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03559062
• Other study ID #: VX16-661-115
• Secondary ID: 2016-004479-35
• Status: Completed
• Phase: Phase 3
• Start date: May 17, 2018
• Est. completion date: December 21, 2018

Study information

• Verified date: February 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 67
• Est. completion date: December 21, 2018
• Est. primary completion date: December 21, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 11 Years

Eligibility

Key Inclusion Criteria:

• Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).

• Participants with ppFEV1 of =70 percentage points adjusted for age, sex, height.

• Participants with a screening LCI2.5 result =7.5.

• Participants who are able to swallow tablets.

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension.

• Colonization 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:
• TEZ/IVA
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing =40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
• IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing =40 kg received IVA 150 mg tablet.
• Placebo
Placebo matched to TEZ/IVA FDC
• Placebo
Placebo matched to IVA

Locations

Country	Name	City	State
Australia	Hunter Medical Research Institute (HMRI)	New Lambton Heights
Australia	Princess Margaret Hospital for Children	Perth
Australia	Lady Cilento Children's Hospital	South Brisbane
Australia	The Children's Hospital at Westmead	Westmead
Belgium	Universitair Ziekenhuis Brussel - Campus Jette	Brussels
Belgium	Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg	Leuven
Denmark	University of Copenhagen Rigshospitalet	Copenhagen
France	Groupe Hospitalier Pellegrin - Hôpital des Enfants	Bordeaux Cedex
France	Hôpital Necker - Enfants Malades	Paris
Germany	Universitaetsklinikum Essen	Essen
Germany	Klinikum der Johann Wolfgang Goethe-Universitaet	Frankfurt
Germany	Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen	Giessen
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitaetsklinikum Heidelberg	Heidelberg
Germany	Universitaetsklinikum Jena	Jena
Germany	Universitaetsklinikum Koeln	Koeln
Germany	Universitaetsklinikum Tuebingen	Tuebingen
Ireland	Our Lady's Children's Hospital	Dublin
Ireland	University Hospital Limerick	Limerick
Poland	Klinika Mukowiscydozy, Oddzial Chorób Pluc SZP ZOZ	Dziekanow Lesny
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zuerich
United Kingdom	Royal Hospital for Sick Children	Edinburgh
United Kingdom	Leeds General Infirmary	Leeds
United Kingdom	Royal Brompton Hospital	London
United Kingdom	Nottingham University Hospital City Campus	Nottingham
United Kingdom	Southampton General Hospital	Southampton

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Belgium,  Denmark,  France,  Germany,  Ireland,  Poland,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8	LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.	From baseline through Week 8
Secondary	Absolute Change in Sweat Chloride At Week 8	Sweat samples were collected using an approved collection device.	From baseline at Week 8
Secondary	Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8	The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.	From baseline through Week 8
Secondary	Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit		From first dose of study drug up to safety follow-up visit (up to Week 12)