Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

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Overview

Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema

This is a Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of SAR447537 (INBRX-101) Compared to Plasma-Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency Emphysema

Interventions

SAR447537DRUG

A1PI, Recombinant, Bivalent Fc Fusion Protein

ZemairaDRUG

Alpha1-Proteinase Inhibitor (Human)

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Males or females 18-80 years of age, inclusive, at the time of screening 2. Diagnosis of AATD 3. Evidence of emphysema secondary to AATD 4. FEV1 of ≥ 30% and ≤ 80% predicted at screening 5. Current non-smoking status. Exclusion Criteria: 1. Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug 2. Known or suspected allergy to components of SAR447537 (INBRX-101), A1PI or human IgG 3. Known selective or severe Immunoglobulin A (IgA) deficiency 4. Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes 5. Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days 6. On waiting list for lung or liver transplant 7. Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening 8. Evidence of decompensated cirrhosis 9. Active cancers or has a history of malignancy within 5 years prior to screening 10. History of unstable cor pulmonale 11. Clinically significant congestive heart failure

Locations (65)

Outcomes

Primary Outcomes

Serum functional AAT (fAAT) levels at steady-state

To assess the mean change in average fAAT concentration as measured by anti-neutrophil elastase capacity \[ANEC\] from baseline to average serum trough fAAT concentration at steady-state (Ctrough,ss) in participants treated with SAR447537 compared to A1PI

Time frame: 32 Weeks

Secondary Outcomes

fAAT Concentration changes

Mean change in serum fAAT concentration from baseline to fAAT average concentration at steady-state (Cavg, ss) in participants treated with SAR447537 compared to A1PI.

Time frame: 32 Weeks

Days with fAAT above the lower limit of the normal range

Percentage of days with fAAT above the lower limit of the normal range during steady-state dosing in participants treated with SAR447537 compared to A1PI.

Time frame: 32 weeks

Incidence of TEAEs

Incidence of all treatment-emergent adverse events (TEAEs), TEAEs ≥ Grade 3, serious adverse events (SAEs), TEAEs leading to IMP discontinuation, adverse events of special interest (AESI) (including infusion- related reactions).

Time frame: 32 Weeks

Anti-drug antibodies

Frequency of anti-drug antibodies (ADA) against SAR447537 and endogenous AAT, as well as neutralizing ADA (NAb) against SAR447537 and endogenous AAT.

Time frame: 32 Weeks

Population Pharmacokinetics: Clearance

Modeling by means of appropriate software to characterize the pharmacokinetic profile of SAR447537 via estimation of the parameter clearance

Time frame: 32 Weeks

Data from ClinicalTrials.gov

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...and 55 more locations