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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
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
Secondary ID [1] 0 0
2020-005805-25
Secondary ID [2] 0 0
M19-771
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis (CF) 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 - 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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [1] 0 0
Day 1 (Baseline) through Day 29
Primary outcome [2] 0 0
Cohort 3: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
Timepoint [2] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [1] 0 0
Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
Timepoint [1] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [2] 0 0
Absolute Change From Baseline Through Day 29 in Forced Vital Capacity (FVC)
Timepoint [2] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [3] 0 0
Absolute Change From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
Timepoint [3] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [4] 0 0
Relative Changes From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [4] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [5] 0 0
Relative Changes From Baseline Through Day 29 in Forced Vital Capacity (FVC)
Timepoint [5] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [6] 0 0
Relative Changes From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
Timepoint [6] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [7] 0 0
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline.
Timepoint [7] 0 0
Day 1 (Baseline) through Day 29
Secondary outcome [8] 0 0
Cohort 3: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [8] 0 0
Day 1 (Baseline) through Day 29

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

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 analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/09/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/06/2023
Sample size
Target
Accrual to date
Final
48
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital /ID# 228781 - Camperdown
Recruitment hospital [2] 0 0
Westmead Hospital /ID# 227281 - Westmead
Recruitment hospital [3] 0 0
Mater Misericordiae Limited /ID# 227279 - South Brisbane
Recruitment hospital [4] 0 0
Royal Adelaide Hospital /ID# 228486 - Adelaide
Recruitment hospital [5] 0 0
Alfred Health /ID# 227283 - Melbourne
Recruitment hospital [6] 0 0
Royal Children's Hospital /ID# 227280 - Parkville
Recruitment hospital [7] 0 0
Institute for Respiratory Health /ID# 227624 - Nedlands
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4101 - South Brisbane
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment postcode(s) [6] 0 0
3052 - Parkville
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New Hampshire
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oklahoma
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Belgium
State/province [18] 0 0
Antwerpen
Country [19] 0 0
Belgium
State/province [19] 0 0
Bruxelles-Capitale
Country [20] 0 0
Belgium
State/province [20] 0 0
Oost-Vlaanderen
Country [21] 0 0
Belgium
State/province [21] 0 0
Vlaams-Brabant
Country [22] 0 0
Hungary
State/province [22] 0 0
Budapest
Country [23] 0 0
Netherlands
State/province [23] 0 0
Zuid-Holland
Country [24] 0 0
Netherlands
State/province [24] 0 0
Amsterdam
Country [25] 0 0
Netherlands
State/province [25] 0 0
Den Haag
Country [26] 0 0
New Zealand
State/province [26] 0 0
Auckland
Country [27] 0 0
New Zealand
State/province [27] 0 0
Canterbury
Country [28] 0 0
Slovakia
State/province [28] 0 0
Banska Bystrica
Country [29] 0 0
Slovakia
State/province [29] 0 0
Bratislava

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
AbbVie
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
ABBVIE INC.
Address 0 0
AbbVie
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)?
Yes
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)
When will data be available (start and end dates)?
For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
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/
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/abbvie/


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/NCT04853368