A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
- Registration Number
- NCT04599465
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 69
-
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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA IVA Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening. ELX/TEZ/IVA ELX/TEZ/IVA Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening.
- Primary Outcome Measures
Name Time Method Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48 Baseline, Week 36 and 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
Name Time Method Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48 Baseline, 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) Day 1 up to Week 52
Trial Locations
- Locations (41)
Hopital Arnaud de Villeneuve
🇫🇷Montpellier Cedex 5, France
Cliniques Universitaires de Bruxelles Hopital Erasme
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
CHRU de Lille - Hopital Albert Calmette
🇫🇷Lille, France
Telethon Kids Institute
🇦🇺Nedlands, Australia
The Royal Children's Hospital
🇦🇺Parkville, VIC, Australia
Sydney Children's Hospital
🇦🇺Randwick, Australia
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
Westmead Hospital
🇦🇺Westmead, Australia
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
🇳🇱Heidelberglaan, Netherlands
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
🇪🇸Barcelona, Spain
CHU Marseille - Hopital Nord
🇫🇷Marseille, France
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
🇮🇹Verona, Italy
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Saint Joan de Deu
🇪🇸Barcelona, Spain
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Hospital Universitari Vall d Hebron
🇪🇸Barcelona, Spain
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Azienda Ospedaliero Universitaria Federico II Napoli
🇮🇹Naples, Italy
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
🇫🇷Nice, France
Hopitaux Universitaires de Strasbourg
🇫🇷Strasbourg, France
Hopital Foch (Suresnes), Hopital Foch, Adultes
🇫🇷Suresnes, France
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
🇮🇹Ancona, Italy
CHU de Rouen - Hopital Charles Nicolle
🇫🇷Rouen Cedex, Seine Maritime, France
Erasmus Medical Center
🇳🇱Rotterdam, Netherlands
Royal Adelaide Hospital
🇦🇺Adelaide, Australia
The Prince Charles Hospital
🇦🇺Chermside, Australia
Alfred Hospital
🇦🇺Melbourne, Australia
Mater Adult Hospital
🇦🇺South Brisbane, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Klinika Nemoci Plicnich a Tuberkulozy
🇨🇿Brno, Czechia
Centre Hospitalier Intercommunal Creteil
🇫🇷Créteil, France
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, France
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
HagaZiekenhuis van den Haag
🇳🇱Den Haag, Netherlands
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
🇫🇷Nantes, France
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Academisch Medisch Centrum (Academic Medical Centre)
🇳🇱Amsterdam, Netherlands
Azienda Ospedaliera Universitaria Policlinico G. Martino
🇮🇹Messina, Italy