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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04853368
Registration number
NCT04853368
Ethics application status
Date submitted
19/04/2021
Date registered
21/04/2021
Titles & IDs
Public title
Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis
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Scientific title
A Phase 2 Study of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
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Secondary ID [1]
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2020-005805-25
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Secondary ID [2]
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M19-771
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis (CF)
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Cystic fibrosis
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Respiratory
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Other respiratory disorders / diseases
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Inflammatory and Immune System
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Connective tissue diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ABBV-576
Treatment: Drugs - Galicaftor
Treatment: Drugs - Placebo
Treatment: Drugs - Navocaftor
Treatment: Drugs - ABBV-119
Experimental: F508del Homozygous Cystic Fibrosis (CF) Participants - F508del homozygous cystic fibrosis (CF) participants receive galicaftor/navocaftor dual combination (28 days) followed by galicaftor/navocaftor/ABBV-119 triple combination therapy (28 days).
Experimental: F508del Heterozygous CF Participants (Active Drug Group) - F508del heterozygous CF participants receive galicaftor/navocaftor/ABBV-119 combination therapy (28 days).
Placebo comparator: F508del Heterozygous CF Participants (Placebo Group) - F508del heterozygous CF participants receive placebo (28 days).
Experimental: F508del Homozygous and Heterozygous CF Participants - F508del homozygous and heterozygous CF participants receive galicaftor/navocaftor/ABBV-576 triple combination therapy for 28 days.
Treatment: Drugs: ABBV-576
Oral capsules
Treatment: Drugs: Galicaftor
Oral capsules
Treatment: Drugs: Placebo
Oral capsules
Treatment: Drugs: Navocaftor
Oral capsules
Treatment: Drugs: ABBV-119
Oral capsules
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration and is used as a measure of lung function. Mixed-effect model with repeated measures (MMRM) was used for the analyses.
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Timepoint [1]
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Day 1 (Baseline) through Day 29
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Primary outcome [2]
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Cohort 3: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
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Assessment method [2]
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Sweat collection was performed to evaluate sweat chloride concentration. SwCl is a biomarker of cystic fibrosis transmembrane conductance regulator (CFTR) activity. Persons with CF have higher levels of chloride in their sweat. MMRM was used for the analysis.
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Timepoint [2]
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Day 1 (Baseline) through Day 29
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Secondary outcome [1]
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Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
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Assessment method [1]
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Sweat collection was performed to evaluate sweat chloride concentration. SwCl is a biomarker of CFTR activity. Persons with CF have higher levels of chloride in their sweat. MMRM was used for the analysis.
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Timepoint [1]
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Day 1 (Baseline) through Day 29
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Secondary outcome [2]
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Absolute Change From Baseline Through Day 29 in Forced Vital Capacity (FVC)
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Assessment method [2]
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FVC is the total amount of air exhaled during forced expiratory volume (FEV) test and is a lung function test that is measured during spirometry. MMRM was used for the analyses.
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Timepoint [2]
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Day 1 (Baseline) through Day 29
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Secondary outcome [3]
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Absolute Change From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
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Assessment method [3]
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FEF25-75 is a lung function test that is measured during spirometry, and is defined as the forced expiratory flow between 25% and 75% of vital capacity (mid-lung capacity). MMRM was used for analyses.
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Timepoint [3]
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Day 1 (Baseline) through Day 29
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Secondary outcome [4]
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Relative Changes From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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Assessment method [4]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration and is used as a measure of lung function. MMRM was used for the analyses. Note: The primary analysis of ppFEV1 using MMRM excludes data that are inconsistent with baseline in terms of the timing of bronchodilator or airway clearance regimen.
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Timepoint [4]
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Day 1 (Baseline) through Day 29
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Secondary outcome [5]
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Relative Changes From Baseline Through Day 29 in Forced Vital Capacity (FVC)
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Assessment method [5]
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FVC is the total amount of air exhaled during FEV test and is a lung function test that is measured during spirometry. MMRM was used for the analyses.
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Timepoint [5]
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Day 1 (Baseline) through Day 29
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Secondary outcome [6]
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Relative Changes From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
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Assessment method [6]
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FEF25-75 is a lung function test that is measured during spirometry, and is defined as the forced expiratory flow between 25% and 75% of vital capacity (mid-lung capacity). MMRM was used for analyses.
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Timepoint [6]
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Day 1 (Baseline) through Day 29
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Secondary outcome [7]
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Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline.
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Assessment method [7]
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The CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score is designed for use in participants with a diagnosis of cystic fibrosis and is designed to measure impact on overall health, daily life, perceived well-being, and symptoms. CFQ-R has a total of 50 questions. Questions 40, 41, 42, 44, 45, 46, scored 1, 2, 3, or 4, from worst to best, were used to calculate the respiratory domain score. The scaled score for the domain is calculated as 100 × (mean scores of all non-missing questions - 1) / 3, ranging from 0 to 100. If more than 3 questions in the domain have missing scores, the scaled score was set as missing. Note: The LS mean is estimated using the linear regression on the change in CFQ-R from baseline to day 29. The higher CFQ-R respiratory score indicates better health. A positive change in CFQ-R respiratory score since baseline indicates improved health quality, while a negative change indicates a decreased health quality.
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Timepoint [7]
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Day 1 (Baseline) through Day 29
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Secondary outcome [8]
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Cohort 3: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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Assessment method [8]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration and is used as a measure of lung function. MMRM was used for the analyses. Note: The primary analysis of ppFEV1 using MMRM excludes data that are inconsistent with baseline in terms of the timing of bronchodilator or airway clearance regimen.
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Timepoint [8]
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Day 1 (Baseline) through Day 29
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Eligibility
Key inclusion criteria
* Confirmed clinical diagnosis of cystic fibrosis (CF).
* Arm 1 participants with genotype homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation and not receiving elexacaftor/tezacaftor/ivacaftor (ETI) treatment .
* Arm 2 and 3 participants with genotype heterozygous for the F508del CFTR mutation and a minimal function mutation and not receiving ETI treatment.
* Arm 4 participants with genotype either homozygous or heterozygous for the F508del mutation. Participants must be receiving stable ETI treatment.
* Percent predicted forced expiratory volume in 1 second (ppFEV1) >= 40% and <=90% of predicted normal for age, gender and height at screening.
* For arms 1 and 2: sweat chloride (SwCl) >= 60 mmol/L at screening. For participants who participated in Study M19-530, it is acceptable to use a SwCl value that the central lab provided in Study M19-530 to establish eligibility.
* Weight >= 35 kg at screening and Day -28 for arm 1 or day 1 for arms 2 to 4.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Clinically significant laboratory values at screening that would pose undue risk for the participant or interfere with safety assessments (per the investigator).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
20/09/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
5/06/2023
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Sample size
Target
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Accrual to date
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Final
48
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
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Recruitment hospital [1]
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Royal Prince Alfred Hospital /ID# 228781 - Camperdown
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Recruitment hospital [2]
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Westmead Hospital /ID# 227281 - Westmead
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Recruitment hospital [3]
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Mater Misericordiae Limited /ID# 227279 - South Brisbane
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Recruitment hospital [4]
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Royal Adelaide Hospital /ID# 228486 - Adelaide
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Recruitment hospital [5]
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Alfred Health /ID# 227283 - Melbourne
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Recruitment hospital [6]
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Royal Children's Hospital /ID# 227280 - Parkville
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Recruitment hospital [7]
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Institute for Respiratory Health /ID# 227624 - Nedlands
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4101 - South Brisbane
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Recruitment postcode(s) [4]
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5000 - Adelaide
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Recruitment postcode(s) [5]
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3004 - Melbourne
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Recruitment postcode(s) [6]
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3052 - Parkville
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Recruitment postcode(s) [7]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Alabama
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California
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Florida
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Kansas
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Massachusetts
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Michigan
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Missouri
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Slovakia
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Banska Bystrica
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Slovakia
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Bratislava
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
AbbVie
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Address
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Ethics approval
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Summary
Brief summary
Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40. The main objective of this study is to assess how safe and effective is the combination therapy of galicaftor/navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 in adult participants with CF who are homozygous or heterozygous for the F508del mutation in each arm. Galicaftor/Navocaftor/ABBV-119 combination therapy and Galicaftor/Navocaftor/ABBV-576 is being developed as an investigational drug for the treatment of CF. Study doctors place participants in 1 of the 4 groups, called treatment arms. Each group receives a different treatment. Around 90 adult participants with a diagnosis of CF will be enrolled in the study around approximately 35 sites worldwide. Participants in arm 1 will receive oral capsules of galicaftor/navocaftor dual combination for 28 days followed by galicaftor/navocaftor/ABBV-119 triple combination for 28 days. Participants in arms 2 and 3 will receive the galicaftor/navocaftor/ABBV-119 triple combination or placebo for 28 days. Participants in arm 4 will receive galicaftor/navocaftor/ABBV-576 triple combination therapy for 28 days. For all study arms, ABBV-576, galicaftor, navocaftor, will be given once daily and ABBV-119 twice a day. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
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Trial website
https://clinicaltrials.gov/study/NCT04853368
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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ABBVIE INC.
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Address
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AbbVie
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols, analyses plans, clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
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When will data be available (start and end dates)?
For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
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Available to whom?
Access to this clinical trial data can be requested by any qualified researchers who engage in rigorous independent scientific research, and will be provided following review and approval of a research proposal and statistical analysis plan and execution of a data sharing statement. Data requests can be submitted at any time after approval in the US and/or EU and a primary manuscript is accepted for publication. For more information on the process, or to submit a request, visit the following link https://www.abbvieclinicaltrials.com/hcp/data-sharing/
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/abbvie/
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What supporting documents are/will be available?
No Supporting Document Provided
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Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/68/NCT04853368/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/68/NCT04853368/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04853368