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

Study of investigational medication compared to plasma-derived therapy in adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema

Not Recruiting
18 years - 80 years
All
Phase 2
99 participants needed

Study Overview

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

Study Details

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.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Alpha 1-Antitrypsin Deficiency, Emphysema
  • Age: 18 years - 80 years
  • Gender: All

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

The purpose of this study is to investigate the effects of an investigational medication compared to plasma-derived therapy in adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema. This study aims to understand how the investigational medication works in the body and its safety.

Participants in this study will receive either the investigational medication or the plasma-derived therapy. The study is double-blind, meaning neither the participants nor the researchers know who is receiving which treatment. The study will assess various factors such as how the body processes the medication, any immune response, and overall safety. Participants will be monitored throughout the study for any side effects.

  • Who can participate: Adults aged 18 to 80 years with a diagnosis of AATD and evidence of emphysema can participate. Participants must have a specific lung function range and be non-smokers.
  • Study details: Participants will receive either the investigational medication or plasma-derived therapy. The study is double-blind, ensuring unbiased results. Participants will be monitored for safety and any side effects during the study.
Updated on 21 Aug 2025. Study ID: NCT05856331