Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
Conditions
Brief summary
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
Detailed description
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), 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
Interventions
DRUGSodium Chloride 0.9%
DRUGRespreeza 1
Sponsors
Sanofi AATD Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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), 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 | — |
Countries
Denmark, Ireland, Poland, Spain, Sweden
Outcome results
None listed