Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

Phase 2

Recruiting

• Conditions: Alpha 1-Antitrypsin Deficiency; Emphysema
• Interventions: Drug: SAR447537

• Registration Number: NCT05897424
• Lead Sponsor: Sanofi

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.

Study Timeline

• Study First Submitted: 2023-05-31
• Study First Submitted QC: 2023-05-31
• Study First Posted: 2023-06-09
• Study Start: 2024-06-20
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-31
• Last Update Posted: 2025-08-03
• Primary Completion: 2028-06-30
• Study Completion: 2028-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 185

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SAR447537 (INBRX-101)	SAR447537	A1PI, Recombinant, Bivalent Fc Fusion Protein, in a solution for intravenous injection

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (41)

University of Alabama at Birmingham- Site Number : 105; 🇺🇸 Birmingham, Alabama, United States

St. Joseph's Hospital and Medical Center- Site Number : 126; 🇺🇸 Phoenix, Arizona, United States

David Geffen School of Medicine at UCLA- Site Number : 124; 🇺🇸 Los Angeles, California, United States

University of California Davis Health- Site Number : 110; 🇺🇸 Sacramento, California, United States

National Jewish Health Medical Center- Site Number : 123; 🇺🇸 Denver, Colorado, United States

Nuvance Health Medical Practices, Pulmonary & Sleep Specialists- Site Number : 119; 🇺🇸 Danbury, Connecticut, United States

Meris Clinical Research-310 Oakfield Dr- Site Number : 115; 🇺🇸 Brandon, Florida, United States

University of Florida - Gainesville - 1600 SW Archer Rd- Site Number : 101; 🇺🇸 Gainesville, Florida, United States

Indiana University Health University Hospital- Site Number : 127; 🇺🇸 Indianapolis, Indiana, United States

Brigham and Women's Hospital -75 Francis Street- Site Number : 131; 🇺🇸 Boston, Massachusetts, United States

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