Phase 2 study comparing SAR447537 to plasma-derived A1PI therapy for AATD emphysema
- Conditions
- Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
- Registration Number
- 2023-508084-76-00
- Lead Sponsor
- Sanofi AATD Inc.
- Brief Summary
To evaluate the pharmacodynamic effect on serum trough functional AAT (fAAT) levels at steady-state, following a treatment period of up to 32-weeks, in participants with AATD emphysema treated with SAR447537 compared to A1PI
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Not specified
- Target Recruitment
- 34
- 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.
- 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, 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 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
- Secondary Outcome Measures
Name Time Method 1. Mean change in serum fAAT concentration from baseline to fAAT average concentration at steady state (Cavg,ss) in participants treated with INBRX-101 compared to A1PI 2. Percentage of days with fAAT above the lower limit of the normal range during steady-state dosing in participants treated with INBRX-101 compared to A1PI 1. Mean change in serum fAAT concentration from baseline to fAAT average concentration at steady state (Cavg,ss) in participants treated with INBRX-101 compared to A1PI 2. Percentage of days with fAAT above the lower limit of the normal range during steady-state dosing in participants treated with INBRX-101 compared to A1PI
3. 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) 3. 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)
4. Frequency of anti-drug antibodies (ADA) against SAR447537 and endogenous AAT, as well as neutralizing ADA (NAb) against SAR447537 and endogenous AAT 5. Population PK modeling to assess impact of physiologically relevant patient participants characteristics (eg, covariates including, but not limited to, age, sex, body size, ethnicity) and disease on PK 4. Frequency of anti-drug antibodies (ADA) against SAR447537 and endogenous AAT, as well as neutralizing ADA (NAb) against SAR447537 and endogenous AAT 5. Population PK modeling to assess impact of physiologically relevant patient participants characteristics (eg, covariates including, but not limited to, age, sex, body size, ethnicity) and disease on PK
Trial Locations
- Locations (65)
University of Alabama at Birmingham- Site Number : 105
🇺🇸Birmingham, Alabama, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
St Joseph's Hospital and Medical Center- Site Number : 126
🇺🇸Phoenix, Arizona, United States
St Joseph's Hospital and Medical Center
🇺🇸Phoenix, Arizona, United States
David Geffen School of Medicine- Site Number : 124
🇺🇸Los Angeles, California, United States
David Geffen School of Medicine
🇺🇸Los Angeles, California, United States
UC Davis Comprehensive Cancer Center- Site Number : 110
🇺🇸Sacramento, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
National Jewish Medical and Research Center- Site Number : 123
🇺🇸Denver, Colorado, United States
National Jewish Medical and Research Center
🇺🇸Denver, Colorado, United States
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