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

Investigation of an Investigational Medication for 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

Updated on 04 Apr 2025. Study ID: NCT05856331

This study investigates a treatment for adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema. AATD is a genetic condition that can lead to lung disease, like emphysema, due to a lack of a protein that protects the lungs. The purpose of this study is to compare the effects of an investigational medication with a plasma-derived therapy in people with this condition.

Participants will be randomly assigned to receive either the investigational medication or the plasma-derived therapy. The study will assess how the body processes and responds to the medication, as well as its safety. The study will also check for any immune responses to the treatments.

  • Who can participate: Adults aged 18 to 80 with a diagnosis of AATD and evidence of emphysema can participate. Participants must be non-smokers and have a specific lung function range.
  • Study details: Participants will receive either the investigational medication or a plasma-derived therapy. The study will measure how the body processes the treatments and monitor for safety and immune responses.