Alpha 1-Antitrypsin Deficiency, Emphysema
Conditions
Keywords
AATD, Alpha 1-Antitrypsin Deficiency, Emphysema, INBRX-101, AAT, SAR447537
Brief summary
Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema
Detailed description
This is a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.
Interventions
DRUGSAR447537
A1PI, Recombinant, Bivalent Fc Fusion Protein
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Multicenter, single arm, open-label extension study
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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% predicted at screening and post-bronchodilator FEV1/FVC\<0.7 5. Current non-smoking status
Exclusion criteria
For newly identified participants 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, A1PI or human IgG 3. Uncontrolled diabetes mellitus despite adequate antidiabetic pharmacologic treatment with a screening HbA1c value ≥9% 4. Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days 5. On waiting list for lung or liver transplant 6. Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening 7. Evidence of decompensated cirrhosis 8. Active cancers or has a history of malignancy within 5 years prior to screening 9. History of unstable cor pulmonale The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Long-term safety and tolerability | 3 years | Incidence of all treatment emergent adverse events (TEAEs), TEAEs ≥ Grade 3, serious adverse events (SAEs), TEAEs leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in lung density by quantitative computerized tomography (CT) | 3 years | Assess the annual rate of change in lung density assessed by serial quantitative CT (15th percentile point, PD15) at total lung capacity (TLC), centrally read, from baseline to end of treatment (EoT). |
| Trough SAR447537 concentration changes | 3 years | Change in SAR447537 concentration levels from baseline to end of treatment |
| Trough serum functional AAT (fAAT) concentration changes | 3 years | Change in fAAT concentration levels from baseline to end of treatment |
| Covariate Analysis: Biometric Values: Weight | 3 years | Assessment of the impact of participant's weight \[in kg\] on the pharmacokinetic profile of SAR447537 |
| Covariate Analysis: Biometric Values: Height | 3 years | Assessment of the impact of participant's height \[in cm\] on the pharmacokinetic profile of SAR447537 |
| Covariate Analysis: Biometric Values: Age | 3 years | Assessment of the impact of participant's age \[in years\] on the pharmacokinetic profile of SAR447537 |
| Covariate Analysis: Biometric Values: Sex | 3 years | Assessment of the impact of participant's sex \[male or female\] on the pharmacokinetic profile of SAR447537 |
| Anti-drug antibodies | 3 years | Frequency of anti-drug antibodies (ADA) against SAR447537 as well as neutralizing ADA (NAb) against SAR447537 |
Countries
Australia, Denmark, Ireland, New Zealand, Poland, Spain, Sweden, United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Sciences & Operations
Sanofi
Outcome results
None listed