Open Label Extension Study of INBRX-101 in Adults With AATD Emphysema

Study Overview
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Brief Summary
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Phase 2 open label extension study to evaluate INBRX-101 in adults with AATD emphysema

Detailed Description
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This is a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.

Official Title
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This is a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.

Conditions
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Alpha 1-Antitrypsin Deficiency, Emphysema

Intervention / Treatment
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* Drug: INBRX-101


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males or females 18-80 years of age, inclusive, at the time of screening Diagnosis of AATD Evidence of emphysema secondary to AATD FEV1 of ≥ 30% predicted at screening and post-bronchodilator FEV1/FVC<0.7 (Cohort 1 new patients only) Current non-smoking status Exclusion Criteria: Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug Known or suspected allergy to components of INBRX-101, A1PI or human IgG Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days On waiting list for lung or liver transplant Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening Evidence of decompensated cirrhosis Active cancers or has a history of malignancy within 5 years prior to screening History of unstable cor pulmonale Clinically significant congestive heart failure

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 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
Intervention Model: Single Group Assignment
Interventional Model Description: Multicenter, single arm, open-label extension study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: INBRX-101 Q3W<br>IV every 3-weeks (Q3W) | Drug: INBRX-101<br>* A1PI, Recombinant, Bivalent Fc Fusion Protein<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Long-term safety and tolerability | Incidence of all treatment emergent adverse events (TEAEs), TEAEs ≥ Grade 3, serious adverse events (SAEs), TEAEs requiring withdrawal from investigational product (IP) treatment, and adverse events of special interest (AESIs). | 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in lung density by quantitative computerized tomography (CT) | Assess the annual rate of change in lung density assessed by serial quantitative CT (15th percentile point, PD15) at total lung capacity (TLC), centrally read, from baseline to end of treatment. | 3 years | 
| Trough INBRX-101 concentration changes | Change in INBRX-101 concentration levels from baseline to end of treatment | 3 years | 
| Trough serum functional AAT (fAAT) concentration changes | Change in fAAT concentration levels from baseline to end of treatment | 3 years | 
| Covariate Analysis: Biometric Values: Weight | Assessment of the impact of patient's weight [in kg] on the pharmacokinetic profile of INBRX-101 | 3 years | 
| Covariate Analysis: Biometric Values: Height | Assessment of the impact of patient's height [in cm] on the pharmacokinetic profile of INBRX-101 | 3 years | 
| Covariate Analysis: Biometric Values: Age | Assessment of the impact of patient's age [in years] on the pharmacokinetic profile of INBRX-101 | 3 years | 
| Covariate Analysis: Biometric Values: Sex | Assessment of the impact of patient's sex [male or female] on the pharmacokinetic profile of INBRX-101 | 3 years | 
| Anti-drug antibodies | Frequency of anti-drug antibodies (ADA) against INBRX-101 as well as neutralizing ADA (NAb) against INBRX-101 | 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AAT, AATD, Alpha 1-Antitrypsin Deficiency, Emphysema, INBRX-101