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

Study of Investigational Medication for Genetic Lung Disease (Alpha-1 Antitrypsin Deficiency)

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 16 Jan 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 cause lung disease, such as emphysema, which is a type of chronic lung condition that makes it hard to breathe. The purpose of this study is to compare the effects of an investigational medication to a standard therapy derived from plasma, which is a component of blood.

Participants in the study will receive either the investigational medication or the standard plasma-derived therapy. The study will measure how the body processes the medication, its effects on the body, and any immune responses. Participants will be monitored for safety throughout the study.

  • Who can participate: Adults aged 18 to 80 years with a diagnosis of Alpha-1 Antitrypsin Deficiency and evidence of emphysema can join. Participants must have a specific lung function range and be non-smokers.
  • Study Details: Participants will receive either the investigational medication or a standard therapy. The study will assess how the body responds to the treatment and monitor for any side effects. A placebo, an inactive substance, will not be used in this study.