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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05076149




Registration number
NCT05076149
Ethics application status
Date submitted
29/09/2021
Date registered
13/10/2021

Titles & IDs
Public title
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
Scientific title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation
Secondary ID [1] 0 0
2021-000694-85
Secondary ID [2] 0 0
VX20-121-103
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 - VX-121/TEZ/D-IVA
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA
Treatment: Drugs - Placebo (matched to VX-121/TEZ/D-IVA)
Treatment: Drugs - Placebo (matched to ELX/TEZ/IVA)
Treatment: Drugs - Placebo (matched to IVA)

Active comparator: ELX/TEZ/IVA - Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.

Experimental: VX-121/TEZ/D-IVA - Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.


Treatment: Drugs: VX-121/TEZ/D-IVA
Fixed-dose combination tablets for oral administration.

Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.

Treatment: Drugs: IVA
Tablet for oral administration.

Treatment: Drugs: Placebo (matched to VX-121/TEZ/D-IVA)
Placebo matched to VX-121/TEZ/D-IVA for oral administration.

Treatment: Drugs: Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.

Treatment: Drugs: Placebo (matched to IVA)
Placebo matched to IVA for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1)
Timepoint [1] 0 0
From Baseline Through Week 24
Secondary outcome [1] 0 0
Absolute Change in Sweat Chloride (SwCl)
Timepoint [1] 0 0
From Baseline Through Week 24
Secondary outcome [2] 0 0
Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102)
Timepoint [2] 0 0
From Baseline Through Week 24
Secondary outcome [3] 0 0
Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102)
Timepoint [3] 0 0
From Baseline Through Week 24

Eligibility
Key inclusion criteria
Key

* Participant has one of the following genotypes:

* Homozygous for F508del;
* Heterozygous for F508del and a gating (F/G) mutation;
* Heterozygous for F508del and a residual function (F/RF) mutation;
* At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
* Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of solid organ or hematological transplantation
* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Pregnant or breast-feeding females

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
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
27/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/11/2023
Sample size
Target
Accrual to date
Final
597
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Alfred Hospital - Melbourne, VIC
Recruitment hospital [4] 0 0
Institute for Respiratory Health - Nedlands
Recruitment hospital [5] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [6] 0 0
The Royal Children's Hospital - Parkville, VIC
Recruitment hospital [7] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [8] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [9] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [10] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Camperdown
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- Chermside
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- Melbourne, VIC
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- Nedlands
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- Parkville, VIC
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- Randwick
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- South Brisbane
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- Westmead
Recruitment outside Australia
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Cambridge
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Penarth
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Southampton

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 not provided in https://clinicaltrials.gov/study/NCT05076149