Cystic Fibrosis (CF)Phase 2NCT04853368

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

A Phase 2 study testing ABBV-576, Galicaftor and Navocaftor in people with Cystic Fibrosis (CF), run by AbbVie at 41 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Cystic Fibrosis (CF)
What is being tested?: ABBV-576, Galicaftor and Navocaftor
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 48 participants
Where: 41 sitesin 7 countries
Study dates: 20 Sep 2021to 5 Jun 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

Clinically significant laboratory values at screening that would pose undue risk for the participant or interfere with safety assessments (per the investigator).

Trial locations and contacts

41 sites in 7 countries.

Country

City, postcode or hospital

41 sites in 7 countries

United States22 sites

Albany Medical College-Pulmonary /ID# 248838
Albany, New York, 12208-3504
Boston Children's Hospital /ID# 248646
Boston, Massachusetts, 02115
Medical University of South Carolina /ID# 245403
Charleston, South Carolina, 29425
UH Cleveland Medical Center /ID# 245433
Cleveland, Ohio, 44106
Harper University Hospital /ID# 248917
Detroit, Michigan, 48201
Penn State Health /ID# 248585
Hershey, Pennsylvania, 17033
University of Kansas Health Sy /ID# 249056
Kansas City, Kansas, 66160-8500
Dartmouth-Hitchcock Medical Center /ID# 245706
Lebanon, New Hampshire, 03756
University of Southern California /ID# 249147
Los Angeles, California, 90030
Dartmouth Hitchcock Manchester /ID# 248795
Manchester, New Hampshire, 03104-4125
Medical College of Wisconsin - Plank Rd /ID# 249079
Milwaukee, Wisconsin, 53226-3548
Velocity Clinical Research /ID# 248675
Mobile, Alabama, 36608-1771
Vanderbilt University Medical Center /ID# 245400
Nashville, Tennessee, 37232
Northwell Health/Long Island Jewish Hospital /ID# 248916
New Hyde Park, New York, 11042
University of Oklahoma HSC /ID# 249190
Oklahoma City, Oklahoma, 73104-5410
Central FL Pulmonary Orlando /ID# 245432
Orlando, Florida, 32803
Children's Hospital of Richmond at VCU /ID# 248561
Richmond, Virginia, 23298
Washington University-School of Medicine /ID# 245393
St Louis, Missouri, 63110
ProMedica Toledo Harris McIntosh /ID# 248627
Toledo, Ohio, 43606-3845
The Univ Texas HSC at Tyler /ID# 248498
Tyler, Texas, 75708
New York Medical College /ID# 248640
Valhalla, New York, 10595
Ventura County Medical Center /ID# 248586
Ventura, California, 93003-1651

Other trials in United States

Australia7 sites

Royal Adelaide Hospital /ID# 228486
Adelaide, South Australia, 5000
Royal Prince Alfred Hospital /ID# 228781
Camperdown, New South Wales, 2050
Alfred Health /ID# 227283
Melbourne, Victoria, 3004
Institute for Respiratory Health /ID# 227624
Nedlands, Western Australia, 6009
Royal Children's Hospital /ID# 227280
Parkville, Victoria, 3052
Mater Misericordiae Limited /ID# 227279
South Brisbane, Queensland, 4101
Westmead Hospital /ID# 227281
Westmead, New South Wales, 2145

Other trials in Australia

Belgium4 sites

Uza /Id# 228533
Edegem, Antwerpen, 2650
UZ Gent /ID# 226605
Ghent, Oost-Vlaanderen, 9000
UZ Brussel /ID# 226607
Jette, Brussels Capital, 1090
Universitair Ziekenhuis Leuven /ID# 226608
Leuven, Vlaams-Brabant, 3000

Other trials in Belgium

Netherlands3 sites

Academisch Medisch Centrum /ID# 234253
Amsterdam, 1105 AZ
Erasmus Medisch Centrum /ID# 234254
Rotterdam, South Holland, 3015 GD
HagaZiekenhuis /ID# 234138
The Hague, 2545 AA

Other trials in Netherlands

New Zealand2 sites

Christchurch Hospital /ID# 227335
Christchurch, Canterbury, 8011
Greenlane Clinical Centre /ID# 227282
Epsom, Auckland, 1051

Other trials in New Zealand

Slovakia2 sites

Fakultna nemocnica s poliklinikou F.D. Roosevelta Banska Bystrica /ID# 228044
Banská Bystrica, 975 17
Univerzitna nemocnica Bratislava Nemocnica Ruzinov /ID# 228042
Bratislava, 821 06

Other trials in Slovakia

Hungary1 site

Orszagos Koranyi Pulmonologiai Intezet /ID# 228810
Budapest, 1121

Other trials in Hungary

About this study

Official 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

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.

Read the detailed description

Participants in Cohorts 1 and 3 will receive Open-label therapy. Participants in Cohorts 2 will receive Double-blinded therapy.

Conditions: Cystic Fibrosis (CF)
Being tested: ABBV-576, Galicaftor, Placebo, Navocaftor, ABBV-119
Keywords: Cystic Fibrosis (CF), Galicaftor, Navocaftor, ABBV-119, ABBV-576
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ABBV-576Drug
Oral capsules
GalicaftorDrug
Also called ABBV-2222
Oral capsules
PlaceboDrug
Oral capsules
NavocaftorDrug
Also called ABBV-3067
Oral capsules
ABBV-119Drug
Oral capsules

Study groups in NCT04853368
Study group	Role	What they receive
F508del Homozygous Cystic Fibrosis (CF) Participants	Experimental	F508del homozygous cystic fibrosis (CF) participants receive galicaftor/navocaftor dual combination (28 days) followed by galicaftor/navocaftor/ABBV-119 triple combination therapy (28 days). Receives: Drug: Galicaftor; Drug: Navocaftor; Drug: ABBV-119
F508del Heterozygous CF Participants (Active Drug Group)	Experimental	F508del heterozygous CF participants receive galicaftor/navocaftor/ABBV-119 combination therapy (28 days). Receives: Drug: Galicaftor; Drug: Navocaftor; Drug: ABBV-119
F508del Heterozygous CF Participants (Placebo Group)	Placebo comparator	F508del heterozygous CF participants receive placebo (28 days). Receives: Drug: Placebo
F508del Homozygous and Heterozygous CF Participants	Experimental	F508del homozygous and heterozygous CF participants receive galicaftor/navocaftor/ABBV-576 triple combination therapy for 28 days. Receives: Drug: ABBV-576; Drug: Galicaftor; Drug: Navocaftor

What is being measured

The primary outcome is the main question the study is designed to answer.

Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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.
Measured: Day 1 (Baseline) through Day 29
Cohort 3: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
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.
Measured: Day 1 (Baseline) through Day 29

Secondary outcomes (8)

Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
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.
Measured: Day 1 (Baseline) through Day 29
Absolute Change From Baseline Through Day 29 in Forced Vital Capacity (FVC)
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.
Measured: Day 1 (Baseline) through Day 29
Absolute Change From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
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.
Measured: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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.
Measured: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Forced Vital Capacity (FVC)
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.
Measured: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
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.
Measured: Day 1 (Baseline) through Day 29
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline.
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.
Measured: Day 1 (Baseline) through Day 29
Cohort 3: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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.
Measured: Day 1 (Baseline) through Day 29

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

20 Sep 20215 Jun 2023

First posted21 Apr 2021
Study start20 Sep 2021
Primary completion5 Jun 2023
Study completion5 Jun 2023

Why it stopped: Strategic considerations

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
More trials run by AbbVieThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 48 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04853368
Sponsor's protocol ID: M19-771
Other IDs: 2020-005805-25
Status last verified: Jul 2024
First submitted: 19 Apr 2021
Last updated: 16 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: 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.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official 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
ClinicalTrials.gov ID: NCT04853368
Acronym / sponsor ID: M19-771
Conditions: Cystic Fibrosis (CF)
Keywords: Cystic Fibrosis (CF), Galicaftor, Navocaftor, ABBV-119, ABBV-576
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Strategic considerations
Enrolment: 48 participants (actual)
Lead sponsor: AbbVieIndustry (pharma, biotech, devices)
Study start: 20 Sep 2021 (actual)
Primary completion: 5 Jun 2023 (actual)
Study completion: 5 Jun 2023 (actual)
First posted: 21 Apr 2021 (actual)
Results: Posted 16 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Participants in Cohorts 1 and 3 will receive Open-label therapy. Participants in Cohorts 2 will receive Double-blinded therapy.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 4

Arm	Type	Receives
F508del Homozygous Cystic Fibrosis (CF) Participants	Experimental	Drug: Galicaftor, Drug: Navocaftor, Drug: ABBV-119F508del homozygous cystic fibrosis (CF) participants receive galicaftor/navocaftor dual combination (28 days) followed by galicaftor/navocaftor/ABBV-119 triple combination therapy (28 days).
F508del Heterozygous CF Participants (Active Drug Group)	Experimental	Drug: Galicaftor, Drug: Navocaftor, Drug: ABBV-119F508del heterozygous CF participants receive galicaftor/navocaftor/ABBV-119 combination therapy (28 days).
F508del Heterozygous CF Participants (Placebo Group)	Placebo comparator	Drug: PlaceboF508del heterozygous CF participants receive placebo (28 days).
F508del Homozygous and Heterozygous CF Participants	Experimental	Drug: ABBV-576, Drug: Galicaftor, Drug: NavocaftorF508del homozygous and heterozygous CF participants receive galicaftor/navocaftor/ABBV-576 triple combination therapy for 28 days.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

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.

Key exclusion criteria (1 listed)

Clinically significant laboratory values at screening that would pose undue risk for the participant or interfere with safety assessments (per the investigator).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ABBV-576Oral capsules	Drug	F508del Homozygous and Heterozygous CF Participants
GalicaftorAlso: ABBV-2222Oral capsules	Drug	F508del Heterozygous CF Participants (Active Drug Group), F508del Homozygous Cystic Fibrosis (CF) Participants, F508del Homozygous and Heterozygous CF Participants
PlaceboOral capsules	Drug	F508del Heterozygous CF Participants (Placebo Group)
NavocaftorAlso: ABBV-3067Oral capsules	Drug	F508del Heterozygous CF Participants (Active Drug Group), F508del Homozygous Cystic Fibrosis (CF) Participants, F508del Homozygous and Heterozygous CF Participants
ABBV-119Oral capsules	Drug	F508del Heterozygous CF Participants (Active Drug Group), F508del Homozygous Cystic Fibrosis (CF) Participants

Primary outcome

Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: Day 1 (Baseline) through Day 29
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.
Cohort 3: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
Time frame: Day 1 (Baseline) through Day 29
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.

8 secondary outcomes

Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L
Time frame: Day 1 (Baseline) through Day 29
Absolute Change From Baseline Through Day 29 in Forced Vital Capacity (FVC)
Time frame: Day 1 (Baseline) through Day 29
Absolute Change From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
Time frame: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Forced Vital Capacity (FVC)
Time frame: Day 1 (Baseline) through Day 29
Relative Changes From Baseline Through Day 29 in Forced Expiratory Flow at Mid-Lung Capacity (FEF25-75)
Time frame: Day 1 (Baseline) through Day 29
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline.
Time frame: Day 1 (Baseline) through Day 29
Cohort 3: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time frame: Day 1 (Baseline) through Day 29

Results and safety

Results posted to the registry on 16 Jul 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
C1(d-29 to -1) Dual Combo G + N	24	24
C1(d1 - 29) Triple Combo G + N + ABBV-119	0	0
C2(d1 - 29) Triple Combo G + N + ABBV-119	0	0
C2(d1 - 29) Placebo	0	0
C3 (d1 - 29) Triple Combo G + N + ABBV-576 for F508del Homo	0	0
C3 (d1 - 29) Triple Combo G + N + ABBV-576 for F508del Hetero	0	0

Primary outcome: Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

Time frame: Day 1 (Baseline) through Day 29. Mean (Standard Deviation). Unit: percent predicted FEV1 (%ppFEV1)

Measure	Cohort 1(Day 1 - 29) Triple Combination Galicaftor + Navocaftor + ABBV-119 for F508del Homozygous n = 20	Cohort 2(Day 1 - 29) Triple Combination Galicaftor+ Navocaftor + ABBV-119 for F508del Heterozygous n = 7	Cohort 2 (Day 1 - 29) Placebo for F508del Heterozygous n = 4
Value	2.2 (3.68)	2.6 (5.62)	-2.0 (6.63)

Cohort 1(Day 1 - 29) Triple Combination Galicaftor + Navocaftor + ABBV-119 for F508del Homozygous: LS Mean of Difference: 2.6 (90% CI 1.22 to 4.04), p = 0.002, Mixed Models Analysis
Cohort 2(Day 1 - 29) Triple Combination Galicaftor+ Navocaftor + ABBV-119 for F508del Heterozygous: LS Mean of Difference: 1.3 (90% CI -2.07 to 4.67), p = 0.254, Mixed Models Analysis
Cohort 2(Day 1 - 29) Triple Combination Galicaftor+ Navocaftor + ABBV-119 for F508del Heterozygous vs Cohort 2 (Day 1 - 29) Placebo for F508del Heterozygous: LS Mean of Difference: 0.9 (90% CI -5.07 to 6.77), p = 0.402, Mixed Models Analysis

Population analysed: The Per-Protocol (PP) Population includes all enrolled subjects (randomized subjects for Cohort 2) who carry the intended CFTR mutation based on central lab report and received at least one dose of the Triple Therapy or placebo.

Primary outcome: Cohort 3: Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl) in mmol/L

Time frame: Day 1 (Baseline) through Day 29. Mean (Full Range). Unit: Milli-mole/Liter (mmol/L)

Measure	Cohort 3 (Day 1 - 29) Triple Combination Galicaftor + Navocaftor + ABBV-576 for F508del Homozygous n = 1	Cohort 3 (Day 1 - 29)Triple Combination Galicaftor + Navocaftor + ABBV-576 for F508del Heterozygous n = 1
Value	24 (24 to 24)	40 (40 to 40)

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: All-cause mortality is reported from enrollment to the end of study, median time on follow up in Part 1 was 28 days (d) for Cohort 1 (C1). In Part 2, was 28d for C1; 28 and 28d for C2; and 14, 20.5, and 14d for C3. Treatment-emergent AEs and serious AEs were collected from first dose of study drug until 30d after the last dose of study drug; mean duration on study drug in Part 1 was 28.2d for C1. In Part 2, was 25.8d for C1; 28 and 28.3d for C2; and 16.8, 20.5, and 17.5d for C3.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1 (Day -29 to -1) Dual Combination Galicaftor + Navocaftor for F508del Homozygous	0 of 24 (0%)	4 of 24 (16.7%)	0 of 24
Cohort 1(Day 1 - 29) Triple Combination Galicaftor + Navocaftor + ABBV-119 for F508del Homozygous	1 of 24 (4.2%)	10 of 24 (41.7%)	0 of 24
Cohort 2(Day 1 - 29) Triple Combination Galicaftor+ Navocaftor + ABBV-119 for F508del Heterozygous	0 of 9 (0%)	4 of 9 (44.4%)	0 of 9
Cohort 2 (Day 1 - 29) Placebo F508del Heterozygous	0 of 4 (0%)	2 of 4 (50%)	0 of 4
Cohort 3 (Day 1 - 29) Triple Combination Galicaftor + Navocaftor + ABBV-576 for F508del Homozygous	1 of 9 (11.1%)	7 of 9 (77.8%)	0 of 9
Cohort 3(Day 1 - 29)Triple Combination Galicaftor + Navocaftor + ABBV-576 for F508del Heterozygous	0 of 2 (0%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. 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.

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Cystic Fibrosis (CF)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

ABBVIE INC. study director

AbbVie

41 sites in 7 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ABBV-576, Galicaftor and Navocaftor in people with Cystic Fibrosis (CF), run by AbbVie at 41 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 16 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 41 sites in United States, Australia, Belgium, Netherlands, New Zealand and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) (time frame: Day 1 (Baseline) through Day 29). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04853368.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2024.