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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04599465




Registration number
NCT04599465
Ethics application status
Date submitted
21/10/2020
Date registered
22/10/2020

Titles & IDs
Public title
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
Scientific 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
Secondary ID [1] 0 0
2020-003170-44
Secondary ID [2] 0 0
VX19-445-117
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA

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


Treatment: Drugs: ELX/TEZ/IVA
Fixed dose combination (FDC) tablets for oral administration.

Treatment: Drugs: IVA
Tablets for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
Timepoint [1] 0 0
Baseline, Week 36 and 48
Secondary outcome [1] 0 0
Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48
Timepoint [1] 0 0
Baseline, Week 48
Secondary outcome [2] 0 0
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [2] 0 0
Day 1 up to Week 52

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/01/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/07/2022
Sample size
Target
Accrual to date
Final
69
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [4] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [5] 0 0
The Royal Children's Hospital - Parkville, VIC
Recruitment hospital [6] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [7] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [8] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [9] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment postcode(s) [5] 0 0
- Parkville, VIC
Recruitment postcode(s) [6] 0 0
- Randwick
Recruitment postcode(s) [7] 0 0
- South Brisbane
Recruitment postcode(s) [8] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Belgium
State/province [2] 0 0
Gent
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Czechia
State/province [4] 0 0
Brno
Country [5] 0 0
Czechia
State/province [5] 0 0
Praha 5
Country [6] 0 0
France
State/province [6] 0 0
Créteil
Country [7] 0 0
France
State/province [7] 0 0
Lille
Country [8] 0 0
France
State/province [8] 0 0
Marseille
Country [9] 0 0
France
State/province [9] 0 0
Montpellier Cedex 5
Country [10] 0 0
France
State/province [10] 0 0
Nantes
Country [11] 0 0
France
State/province [11] 0 0
Nice
Country [12] 0 0
France
State/province [12] 0 0
Pierre-Bénite
Country [13] 0 0
France
State/province [13] 0 0
Rouen Cedex, Seine Maritime
Country [14] 0 0
France
State/province [14] 0 0
Strasbourg
Country [15] 0 0
France
State/province [15] 0 0
Suresnes
Country [16] 0 0
Italy
State/province [16] 0 0
Ancona
Country [17] 0 0
Italy
State/province [17] 0 0
Genova
Country [18] 0 0
Italy
State/province [18] 0 0
Messina
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Italy
State/province [20] 0 0
Naples
Country [21] 0 0
Italy
State/province [21] 0 0
Verona
Country [22] 0 0
Netherlands
State/province [22] 0 0
Amsterdam
Country [23] 0 0
Netherlands
State/province [23] 0 0
Den Haag
Country [24] 0 0
Netherlands
State/province [24] 0 0
Heidelberglaan
Country [25] 0 0
Netherlands
State/province [25] 0 0
Rotterdam
Country [26] 0 0
Spain
State/province [26] 0 0
Barcelona
Country [27] 0 0
Spain
State/province [27] 0 0
Madrid
Country [28] 0 0
Spain
State/province [28] 0 0
Murcia
Country [29] 0 0
Spain
State/province [29] 0 0
Sevilla

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04599465