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A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Heterozygous for F508del and an MF mutation (F/MF genotypes)
  • Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age, sex, and height

  • Abnormal glucose tolerance determined by an OGTT as either:

    • Impaired glucose tolerance (IGT)
    • CF-related diabetes (CFRD)

Key Exclusion Criteria:

  • Clinically significant liver cirrhosis with or without portal hypertension
  • Solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Type 1 or Type 2 diabetes
  • Duration of CFRD ≥5 years

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • Royal Adelaide Hospital
  • The Prince Charles Hospital
  • Alfred Hospital
  • Telethon Kids Institute
  • The Royal Children's Hospital
  • Sydney Children's Hospital
  • Mater Adult Hospital
  • Queensland Children's Hospital
  • Westmead Hospital
  • Cliniques Universitaires de Bruxelles Hopital Erasme
  • Universitair Ziekenhuis Gent
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Klinika Nemoci Plicnich a Tuberkulozy
  • Fakultni nemocnice v Motole
  • Centre Hospitalier Intercommunal Creteil
  • CHRU de Lille - Hopital Albert Calmette
  • CHU Marseille - Hopital Nord
  • Hopital Arnaud de Villeneuve
  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec
  • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
  • Centre Hospitalier Lyon Sud
  • CHU de Rouen - Hopital Charles Nicolle
  • Hopitaux Universitaires de Strasbourg
  • Hopital Foch (Suresnes), Hopital Foch, Adultes
  • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
  • Azienda Ospedaliera Universitaria Policlinico G. Martino
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Azienda Ospedaliero Universitaria Federico II Napoli
  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
  • Academisch Medisch Centrum (Academic Medical Centre)
  • HagaZiekenhuis van den Haag
  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
  • Erasmus Medical Center
  • Hospital Saint Joan de Deu
  • Hospital Universitari Vall d Hebron
  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
  • Hospital Universitario 12 de Octubre
  • Hospital Universitario Ramon y Cajal
  • Hospital Virgen de la Arrixaca
  • Hospital Universitario Virgen del Rocio

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening.

Outcomes

Primary Outcome Measures

Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours.

Secondary Outcome Measures

Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Baseline dysglycemia category was defined as the most recent non-missing measurement before the first dose of study drug in the treatment period. Improvement in dysglycemia is a change from cystic fibrosis-related diabetes (CFRD) at baseline to impaired glucose tolerance (IGT)/normal glucose tolerance (NGT) at Week 48 OR change from IGT at baseline to NGT at Week 48. CFRD: 2-hour post-OGTT blood glucose level ≥200 mg/dL or fasting blood glucose level ≥126 mg/dL; IGT: 2-hour post-OGTT blood glucose level ≥140 to <200 mg/dL and fasting blood glucose level <126 mg/dL; NGT: 2 hour post-OGTT blood glucose level <140 mg/dL and fasting blood glucose level <126 mg/dL.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
October 21, 2020
Last Updated
July 13, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04599465
Brief Title
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
Official Title
A Phase 3b Open-label Study to Assess the Effect of Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) on Glucose Tolerance in Cystic Fibrosis Subjects With Abnormal Glucose Metabolism
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
January 15, 2021 (Actual)
Primary Completion Date
July 14, 2022 (Actual)
Study Completion Date
July 14, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
69 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed dose combination (FDC) tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablets for oral administration.
Primary Outcome Measure Information:
Title
Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
Description
Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours.
Time Frame
Baseline, Week 36 and 48
Secondary Outcome Measure Information:
Title
Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Description
Baseline dysglycemia category was defined as the most recent non-missing measurement before the first dose of study drug in the treatment period. Improvement in dysglycemia is a change from cystic fibrosis-related diabetes (CFRD) at baseline to impaired glucose tolerance (IGT)/normal glucose tolerance (NGT) at Week 48 OR change from IGT at baseline to NGT at Week 48. CFRD: 2-hour post-OGTT blood glucose level ≥200 mg/dL or fasting blood glucose level ≥126 mg/dL; IGT: 2-hour post-OGTT blood glucose level ≥140 to <200 mg/dL and fasting blood glucose level <126 mg/dL; NGT: 2 hour post-OGTT blood glucose level <140 mg/dL and fasting blood glucose level <126 mg/dL.
Time Frame
Baseline, Week 48
Title
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Heterozygous for F508del and an MF mutation (F/MF genotypes) Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age, sex, and height Abnormal glucose tolerance determined by an OGTT as either: Impaired glucose tolerance (IGT) defined as 2 hour post OGTT blood glucose level ≥140 to <200 mg/dL (≥7.77 to <11.10 mmol/L) and fasting blood glucose level <126 mg/dL (<7.00 mmol/L) CF-related diabetes (CFRD) defined as either fasting hyperglycemia (blood glucose level ≥126 mg/dL [≥7.00 mmol/L] after an 8-hour fast) or 2-hour post OGTT blood glucose level ≥200 mg/dL (≥11.10 mmol/L) Key Exclusion Criteria: Clinically significant liver cirrhosis with or without portal hypertension Solid organ or hematological transplantation Lung infection with organisms associated with a more rapid decline in pulmonary status Type 1 or Type 2 diabetes Duration of CFRD ≥5 years Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
Royal Adelaide Hospital
City
Adelaide
Country
Australia
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne
Country
Australia
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville, VIC
Country
Australia
Facility Name
Sydney Children's Hospital
City
Randwick
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
Klinika Nemoci Plicnich a Tuberkulozy
City
Brno
Country
Czechia
Facility Name
Fakultni nemocnice v Motole
City
Praha 5
Country
Czechia
Facility Name
Centre Hospitalier Intercommunal Creteil
City
Créteil
Country
France
Facility Name
CHRU de Lille - Hopital Albert Calmette
City
Lille
Country
France
Facility Name
CHU Marseille - Hopital Nord
City
Marseille
Country
France
Facility Name
Hopital Arnaud de Villeneuve
City
Montpellier Cedex 5
Country
France
Facility Name
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
City
Nantes
Country
France
Facility Name
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
City
Nice
Country
France
Facility Name
Centre Hospitalier Lyon Sud
City
Pierre-Bénite
Country
France
Facility Name
CHU de Rouen - Hopital Charles Nicolle
City
Rouen Cedex, Seine Maritime
Country
France
Facility Name
Hopitaux Universitaires de Strasbourg
City
Strasbourg
Country
France
Facility Name
Hopital Foch (Suresnes), Hopital Foch, Adultes
City
Suresnes
Country
France
Facility Name
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
City
Ancona
Country
Italy
Facility Name
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
City
Genova
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Policlinico G. Martino
City
Messina
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Federico II Napoli
City
Naples
Country
Italy
Facility Name
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
City
Verona
Country
Italy
Facility Name
Academisch Medisch Centrum (Academic Medical Centre)
City
Amsterdam
Country
Netherlands
Facility Name
HagaZiekenhuis van den Haag
City
Den Haag
Country
Netherlands
Facility Name
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
City
Heidelberglaan
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
Hospital Saint Joan de Deu
City
Barcelona
Country
Spain
Facility Name
Hospital Universitari Vall d Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
Country
Spain
Facility Name
Hospital Virgen de la Arrixaca
City
Murcia
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Learn more about this trial

A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)

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