A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222

A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation

This study will evaluate the safety, tolerability, and efficacy of ABBV-3067 given alone and in combination with various doses of ABBV-2222 in adults with Cystic Fibrosis who are homozygous for the F508del mutation.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: ABBV-3067; Drug: Placebo ABBV-2222; Drug: ABBV-2222; Drug: Placebo ABBV-3067

• Study Type: Interventional
• Enrollment (Actual): 78
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Antwerpen
    • Edegem, Antwerpen, Belgium, 2650
      • Uza /Id# 213412
  • Bruxelles-Capitale
    • Brussels, Bruxelles-Capitale, Belgium, 1070
      • Cliniques Universitaires de Bruxelles Hopital Erasme /ID# 213413
    • Jette, Bruxelles-Capitale, Belgium, 1090
      • UZ Brussel /ID# 212812
  • Oost-Vlaanderen
    • Gent, Oost-Vlaanderen, Belgium, 9000
      • UZ Gent /ID# 213411
  • Vlaams-Brabant
    • Leuven, Vlaams-Brabant, Belgium, 3000
      • Universitair Ziekenhuis Leuven /ID# 213050
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4Z6
      • University of Calgary /ID# 212555
  • British Columbia
    • Vancouver, British Columbia, Canada, V6Z 1Y6
      • St. Paul's Hospital /ID# 212554
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 2Y9
      • QEII - Health Sciences Centre /ID# 212656
  • Ontario
    • Toronto, Ontario, Canada, M5B 1W8
      • Unity Health Toronto - St. Michael's Hospital /ID# 212552
  • Quebec
    • Montreal, Quebec, Canada, H2X 3E4
      • CHUM - Centre hospitalier de l'Universite de Montréal /ID# 227815
    • Montreal, Quebec, Canada, H4A 3J1
      • McGill University Health Center Research Institute /ID# 212553
    • Québec, Quebec, Canada, G1V 4G5
      • Institut universitaire de cardiologie et de pneumologie de Québec - Université L /ID# 212655
• Czechia
  • Brno, Czechia, 625 00
    • Fakultni Nemocnice Brno /ID# 213437
  • Praha, Czechia, 150 06
    • Fakultni Nemocnice v Motole /ID# 212966
• France
  • Paris, France, 75014
    • AP-HP - Hopital Cochin /ID# 212864
  • Reims, France, 51092
    • CHU de Reims - Hôpital Maison Blanche /ID# 212910
  • Roscoff, France, 29684
    • Fondation ILDYS /ID# 212857
  • Saint-Herblain, France, 44800
    • CHU Nantes - Hopital Laennec /ID# 212897
  • Alpes-Maritimes
    • Nice, Alpes-Maritimes, France, 06200
      • Chu de Nice-Hopital L'Archet Ii /Id# 212862
  • Auvergne-Rhone-Alpes
    • Pierre Benite CEDEX, Auvergne-Rhone-Alpes, France, 69495
      • HCL - Hopital Lyon Sud /ID# 212899
  • Gironde
    • Pessac, Gironde, France, 33604
      • CHU Bordeaux - Hopital Haut Leveque /ID# 212898
  • Herault
    • Montpellier Cedex 5, Herault, France, 34295
      • CHU Montpellier - Hôpital Saint Eloi /ID# 212856
• Hungary
  • Budapest, Hungary, 1121
    • Orszagos Koranyi Pulmonologiai Intezet /ID# 213494
• Netherlands
  • Den Haag, Netherlands, 2545 AA
    • HagaZiekenhuis /ID# 212926
  • Utrecht, Netherlands, 3584 CX
    • Universitair Medisch Centrum Utrecht /ID# 212935
• New Zealand
  • Otago, New Zealand, 9016
    • Dunedin Hospital /ID# 221104
  • Auckland
    • Epsom, Auckland, New Zealand, 1051
      • Greenlane Clinical Centre /ID# 221103
  • Canterbury
    • Christchurch, Canterbury, New Zealand, 8011
      • Christchurch Hospital /ID# 221105
  • Waikato
    • Hamilton, Waikato, New Zealand, 3240
      • Waikato Hospital /ID# 221102
• Poland
  • Pomorskie
    • Gdansk, Pomorskie, Poland, 80-308
      • Szpital Dzieciecy Polanki /ID# 221330
• Serbia
  • Beograd, Serbia, 11000
    • Institut za zdravstvenu zastitu majke i deteta Srbije Dr Vukan Cupic /ID# 212820
• Slovakia
  • Bratislava, Slovakia, 821 01
    • Univerzitna nemocnica Bratislava Nemocnica Ruzinov /ID# 213146
  • Bratislava, Slovakia, 821 06
    • Univerzitna nemocnica Bratislava Nemocnica Ruzinov /ID# 213596
• United Kingdom
  • Cambridge, United Kingdom, CB2 0AY
    • Royal Papworth Hospital NHS Foundation Trust /ID# 212507
  • Leeds, United Kingdom, LS9 7TF
    • Leeds Teaching Hospitals NHS Trust /ID# 212491
  • Liverpool, United Kingdom, L14 3PE
    • Liverpool Heart and Chest Hospital NHS Foundation Trust /ID# 212291
  • London, United Kingdom, SW3 6NP
    • Royal Brompton and Harefield Hospitals /ID# 212490
  • Newcastle upon Tyne, United Kingdom, NE7 7DN
    • The Newcastle Upon Tyne Hospitals NHS Foundation Trust /ID# 212665
  • London, City Of
    • London, London, City Of, United Kingdom, E1 2ES
      • Barts Health NHS Trust /ID# 213016
  • Nottinghamshire
    • Nottingham, Nottinghamshire, United Kingdom, NG5 1PB
      • Nottingham University Hospitals NHS Trust /ID# 212531
  • Wales
    • Cardiff, Wales, United Kingdom, CF14 4XN
      • Cardiff & Vale University Health Board /ID# 212504
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas /ID# 212541
  • Florida
    • Tampa, Florida, United States, 33606
      • Tampa General Hospital /ID# 212342
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics /ID# 212351
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Univ Michigan Med Ctr /ID# 212657
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • Cardinal Glennon Children's Hospital /ID# 212349
    • Saint Louis, Missouri, United States, 63110
      • Washington University-School of Medicine /ID# 212352
  • Ohio
    • Columbus, Ohio, United States, 43205-2664
      • Nationwide Children's Hospital /ID# 213158
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina /ID# 212187
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center /ID# 212353
  • Utah
    • Salt Lake City, Utah, United States, 84112-5500
      • University of Utah /ID# 212350

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Confirmed clinical diagnosis of Cystic Fibrosis (CF) who are homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
• Stable pulmonary status
• Lung function >= 40 and <= 90% of predicted normal for age, gender and height at Screening

Exclusion Criteria:

• History of solid organ or hematopoietic transplant
• Cirrhosis with portal hypertension
• Use of CFTR modulator therapy within 60 days prior to Screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

8

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ABBV-3067 50 mg + Placebo for ABBV-2222; Participants received ABBV-3067 50 mg tablet orally once daily (QD) plus placebo matching ABBV-2222 capsule, orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: Placebo ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + Placebo for ABBV-2222; Participants received ABBV-3067 150 mg tablet orally QD plus placebo matching ABBV-2222 capsule, orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: Placebo ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + ABBV-2222 10 mg; Participants received ABBV-3067 150 mg tablet orally QD plus ABBV-2222 10 mg capsule orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + ABBV-2222 30 mg; Participants received ABBV-3067 150 mg tablet orally QD plus ABBV-2222 30 mg capsule orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + ABBV-2222 100 mg; Participants received ABBV-3067 150 mg tablet orally QD plus ABBV-2222 100 mg capsule orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + ABBV-2222 200 mg; Participants received ABBV-3067 150 mg tablet orally QD plus ABBV-2222 200 mg capsule, orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: ABBV-2222; Capsule taken orally.
Experimental: ABBV-3067 150 mg + ABBV-2222 300 mg; Participants received ABBV-3067 150 mg tablet orally QD plus ABBV-2222 300 mg capsule, orally QD for 28 days.	Drug: ABBV-3067; Tablet taken orally.; Drug: ABBV-2222; Capsule taken orally.
Placebo Comparator: Placebo for ABBV-3067 + Placebo for ABBV-2222; Participants received placebo matching ABBV-3067 tablet orally QD plus placebo matching ABBV-2222 capsule, orally QD for 28 days.	Drug: Placebo ABBV-2222; Capsule taken orally.; Drug: Placebo ABBV-3067; Tablet taken orally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Day 1 (Baseline) through Day 29

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Absolute Change From Baseline Through Day 29 in Sweat Chloride (SwCl)	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.	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.	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.	Day 1 (Baseline) through Day 29
Relative 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 analyses.	Day 1 (Baseline) through Day 29
Relative 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.	Day 1 (Baseline) through Day 29
Relative Change 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.	Day 1 (Baseline) through Day 29

Collaborators and Investigators

• Sponsor: AbbVie
• Investigators: Study Director: ABBVIE INC., AbbVie

Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2019
• Primary Completion (Actual): June 9, 2022
• Study Completion (Actual): June 9, 2022

Study Registration Dates

• First Submitted: May 30, 2019
• First Submitted That Met QC Criteria: May 30, 2019
• First Posted (Actual): May 31, 2019

Study Record Updates

• Last Update Posted (Actual): June 28, 2023
• Last Update Submitted That Met QC Criteria: June 8, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; ABBV-3067; ABBV-2222, F508del Mutation
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: M19-530; 2019-000750-63 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Time Frame: For details on when studies are available for sharing visit https://vivli.org/ourmember/abbvie/
• IPD Sharing Access Criteria: 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/
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No