Clinical trial NCT04599465: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2020-10-22
Enrollment: 69
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
timeFrame: Baseline, Week 36 and 48
Secondary outcomes:
measure: Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
timeFrame: Baseline, Week 48
measure: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 52
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Chermside
country: Australia
facility: The Prince Charles Hospital
city: Melbourne
country: Australia
facility: Alfred Hospital
city: Nedlands
country: Australia
facility: Telethon Kids Institute
city: Parkville, VIC
country: Australia
facility: The Royal Children's Hospital
city: Randwick
country: Australia
facility: Sydney Children's Hospital
city: South Brisbane
country: Australia
facility: Mater Adult Hospital
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Westmead
country: Australia
facility: Westmead Hospital
city: Brussels
country: Belgium
facility: Cliniques Universitaires de Bruxelles Hopital Erasme
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
city: Brno
country: Czechia
facility: Klinika Nemoci Plicnich a Tuberkulozy
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Créteil
country: France
facility: Centre Hospitalier Intercommunal Creteil
city: Lille
country: France
facility: CHRU de Lille - Hopital Albert Calmette
city: Marseille
country: France
facility: CHU Marseille - Hopital Nord
city: Montpellier Cedex 5
country: France
facility: Hopital Arnaud de Villeneuve
city: Nantes
country: France
facility: Centre Hospitalier Universitaire De Nantes - G. R. Laennec
city: Nice
country: France
facility: Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
city: Rouen Cedex, Seine Maritime
country: France
facility: CHU de Rouen - Hopital Charles Nicolle
city: Strasbourg
country: France
facility: Hopitaux Universitaires de Strasbourg
city: Suresnes
country: France
facility: Hopital Foch (Suresnes), Hopital Foch, Adultes
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
city: Genova
country: Italy
facility: IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico G. Martino
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Naples
country: Italy
facility: Azienda Ospedaliero Universitaria Federico II Napoli
city: Verona
country: Italy
facility: Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum (Academic Medical Centre)
city: Den Haag
country: Netherlands
facility: HagaZiekenhuis van den Haag
city: Heidelberglaan
country: Netherlands
facility: University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center
city: Barcelona
country: Spain
facility: Hospital Saint Joan de Deu
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Murcia
country: Spain
facility: Hospital Virgen de la Arrixaca
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
Study officials:
References: