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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: ABBV-3067
* Drug: Placebo ABBV-3067
* Drug: ABBV-2222
* Drug: Placebo ABBV-2222


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

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

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, ABBV-3067, ABBV-2222, F508del Mutation