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

NCT05897424 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: INBRX101-01-2022023-508137-14-00
• Study Type: interventional
• Target: 185
• Locations: 9 countries
• Sites: 40

Brief Summary

Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema

Conditions

Alpha 1-Antitrypsin Deficiency; Emphysema

Keywords

AATD; Alpha 1-Antitrypsin Deficiency; Emphysema; INBRX-101; AAT; SAR447537

Outcome Measures

Primary Outcomes (1)

• Long-term safety and tolerability

  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).

  3 years

Secondary Outcomes (8)

• Change in lung density by quantitative computerized tomography (CT)

  3 years

• Trough SAR447537 concentration changes

  3 years

• Trough serum functional AAT (fAAT) concentration changes

  3 years

• Covariate Analysis: Biometric Values: Weight

  3 years

• Covariate Analysis: Biometric Values: Height

  3 years

Study Arms (1)

SAR447537 (INBRX-101)

EXPERIMENTAL

A1PI, Recombinant, Bivalent Fc Fusion Protein, in a solution for intravenous injection

Drug: SAR447537

Interventions

SAR447537 DRUG

A1PI, Recombinant, Bivalent Fc Fusion Protein

SAR447537 (INBRX-101)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females 18-80 years of age, inclusive, at the time of screening
• Diagnosis of AATD
• Evidence of emphysema secondary to AATD
• FEV1 of ≥ 30% predicted at screening and post-bronchodilator FEV1/FVC\<0.7
• Current non-smoking status

You may not qualify if:

• For newly identified participants
• Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug
• Known or suspected allergy to components of SAR447537, A1PI or human IgG
• Uncontrolled diabetes mellitus despite adequate antidiabetic pharmacologic treatment with a screening HbA1c value ≥9%
• Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days
• On waiting list for lung or liver transplant
• Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening
• Evidence of decompensated cirrhosis
• Active cancers or has a history of malignancy within 5 years prior to screening
• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (40)

UAB Lung Health Center- Site Number : 105

Birmingham, Alabama, 35205-2610, United States

Location

St Joseph's Hospital and Medical Center- Site Number : 126

Phoenix, Arizona, 85013-4220, United States

Location

David Geffen School of Medicine at UCLA- Site Number : 124

Los Angeles, California, 90095-3075, United States

Location

University of California Davis Medical Center- Site Number : 110

Sacramento, California, 95817-2201, United States

Location

National Jewish Medical and Research Center- Site Number : 123

Denver, Colorado, 80206-2761, United States

Location

Nuvance Health Medical Practices, Pulmonary & Sleep Specialists- Site Number : 119

Danbury, Connecticut, 06810-5038, United States

Location

University of Florida - Gainesville - 1600 SW Archer Rd- Site Number : 101

Gainesville, Florida, 32610-3003, United States

Location

Indiana University- Site Number : 127

Indianapolis, Indiana, 46202-5128, United States

Location

Brigham and Women's Hospital -75 Francis Street- Site Number : 131

Boston, Massachusetts, 02115-6105, United States

Location

University of Minnesota-420 Delaware Str SE- Site Number : 125

Minneapolis, Minnesota, 55455-0341, United States

Location

Hannibal Regional Healthcare System-HRMG-Hannibal- Site Number : 111

Hannibal, Missouri, 63401-6890, United States

Location

Columbia University Irving Medical Center- Site Number : 104

New York, New York, 10032-3729, United States

Location

Oregon Health and Science University- Site Number : 117

Portland, Oregon, 97239-3011, United States

Location

Penn State Health Milton S. Hershey Medical Center- Site Number : 122

Hershey, Pennsylvania, 17033-2360, United States

Location

Temple University Hospital - 3401 N Broad St- Site Number : 130

Philadelphia, Pennsylvania, 19140-5103, United States

Location

Velocity Clinical Research - Spartanburg - PPDS- Site Number : 120

Spartanburg, South Carolina, 29303-4225, United States

Location

Houston Methodist Hospital- Site Number : 113

Houston, Texas, 77030-2703, United States

Location

University of Utah Health Care- Site Number : 106

Salt Lake City, Utah, 84132-0001, United States

Location

Investigational Site Number : 202

Chermside, Queensland, 4032, Australia

Location

Investigational Site Number : 204

South Brisbane, Queensland, 4101, Australia

Location

Investigational Site Number : 201

North Adelaide, South Australia, 5000, Australia

Location

Investigational Site Number : 207

Box Hill, Victoria, 3128, Australia

Location

Investigational Site Number : 203

Fitzroy, Victoria, 3065, Australia

Location

Investigational Site Number : 205

Frankston, Victoria, 3199, Australia

Location

Investigational Site Number : 701

Hellerup, 2900, Denmark

Location

Investigational Site Number : 702

Vejle, 7100, Denmark

Location

Investigational Site Number : 501

Dublin, Dublin, D09 XR63, Ireland

Location

Investigational Site Number : 403

Auckland, 1051, New Zealand

Location

Investigational Site Number : 404

Wellington, 6021, New Zealand

Location

Investigational Site Number : 801

Warsaw, Masovian Voivodeship, 01-138, Poland

Location

Investigational Site Number : 802

Krakow, 30-688, Poland

Location

Investigational Site Number : 901

Santander, Cantabria, 39008, Spain

Location

Investigational Site Number : 902

Madrid, 28040, Spain

Location

Investigational Site Number : 903

Santiago de Compostela, 15706, Spain

Location

Investigational Site Number : 601

Gothenburg, 413 46, Sweden

Location

Investigational Site Number : 308

Dundee, Angus, DD1 9SY, United Kingdom

Location

Investigational Site Number : 306

Wythenshawe, Cheshire, M23 9QZ, United Kingdom

Location

Investigational Site Number : 307

Exeter, Devon, EX2 5DW, United Kingdom

Location

Investigational Site Number : 304

Southampton, Hampshire, SO16 6YD, United Kingdom

Location

Investigational Site Number : 309

Coventry, CV2 2DX, United Kingdom

Location

Related Links

• INBRX101-01-202 Plain Language Results Summary

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency; Emphysema

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multicenter, single arm, open-label extension study

Study Record Dates

• First Submitted: May 31, 2023
• First Posted: June 9, 2023
• Study Start: June 20, 2024
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): September 30, 2028
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

SE (1); PL (2); US (18); IE (1); NZ (2); DK (2); AU (6); ES (3); GB (5)