NCT05856331

Brief Summary

Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
99

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Oct 2023

Geographic Reach
8 countries

65 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 28, 2023

Completed
2 months until next milestone

First Posted

Study publicly available on registry

May 12, 2023

Completed
5 months until next milestone

Study Start

First participant enrolled

October 12, 2023

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 6, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 6, 2025

Completed
Last Updated

August 11, 2025

Status Verified

August 1, 2025

Enrollment Period

1.8 years

First QC Date

March 28, 2023

Last Update Submit

August 8, 2025

Conditions

Keywords

AATDAlpha 1-Antitrypsin DeficiencyEmphysemaSAR447537INBRX-101A1PIAAT

Outcome Measures

Primary Outcomes (1)

  • Serum functional AAT (fAAT) levels at steady-state

    To assess the 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

    32 Weeks

Secondary Outcomes (10)

  • fAAT Concentration changes

    32 Weeks

  • Days with fAAT above the lower limit of the normal range

    32 weeks

  • Incidence of TEAEs

    32 Weeks

  • Anti-drug antibodies

    32 Weeks

  • Population Pharmacokinetics: Clearance

    32 Weeks

  • +5 more secondary outcomes

Study Arms (3)

SAR447537 (INBRX-101) Q3W

EXPERIMENTAL

IV every 3-weeks (Q3W) and placebo (normal saline)

Drug: SAR447537

SAR447537 (INBRX-101) Q4W

EXPERIMENTAL

IV every 4-weeks (Q4W) and placebo (normal saline)

Drug: SAR447537

Zemaira (A1PI)

ACTIVE COMPARATOR

60 mg/kg IV once weekly (QW) and placebo (normal saline)

Drug: Zemaira

Interventions

A1PI, Recombinant, Bivalent Fc Fusion Protein

SAR447537 (INBRX-101) Q3WSAR447537 (INBRX-101) Q4W

Alpha1-Proteinase Inhibitor (Human)

Also known as: Respreeza
Zemaira (A1PI)

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (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% and ≤ 80% predicted at screening
  • Current non-smoking status.

You may not qualify if:

  • Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug
  • Known or suspected allergy to components of SAR447537 (INBRX-101), A1PI or human IgG
  • Known selective or severe Immunoglobulin A (IgA) deficiency
  • Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes
  • 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
  • Clinically significant congestive heart failure

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (65)

University of Alabama at Birmingham- Site Number : 105

Birmingham, Alabama, 35233-2110, United States

Location

University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States

Location

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

Phoenix, Arizona, 85013-4220, United States

Location

St Joseph's Hospital and Medical Center

Phoenix, Arizona, 85013-4220, United States

Location

David Geffen School of Medicine- Site Number : 124

Los Angeles, California, 90095-3075, United States

Location

David Geffen School of Medicine

Los Angeles, California, 90095, United States

Location

UC Davis Comprehensive Cancer Center- Site Number : 110

Sacramento, California, 95817, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

National Jewish Medical and Research Center- Site Number : 123

Denver, Colorado, 80206-2761, United States

Location

National Jewish Medical and Research Center

Denver, Colorado, 80206, United States

Location

Western Connecticut Medical Group

Danbury, Connecticut, 06810, United States

Location

University of Florida College of Medicine- Site Number : 101

Gainesville, Florida, 32610, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Bruce W. Carter Miami VA Medical Center - NAVREF - PPDS- Site Number : 114

Miami, Florida, 33125-1624, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Pulmonary and Sleep of Tampa Bay- Site Number : 115

Tampa, Florida, 33607-6316, United States

Location

Pulmonary and Sleep of Tampa Bay

Tampa, Florida, 33607, United States

Location

Loyola University Medical Center- Site Number : 112

Maywood, Illinois, 60153, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Indiana University

Indianapolis, Indiana, 46202-5128, United States

Location

M Health Fairview Clinics and Surgery Center - Minneapolis- Site Number : 125

Minneapolis, Minnesota, 55455-4800, United States

Location

M Health Fairview Clinics and Surgery Center - Minneapolis

Minneapolis, Minnesota, 55455-4800, United States

Location

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

Hannibal, Missouri, 63401-6890, United States

Location

Hannibal Clinic

Hannibal, Missouri, 63401, United States

Location

Columbia University Irving Medical Center- Site Number : 104

New York, New York, 10032, United States

Location

Oregon Health and Science University- Site Number : 117

Portland, Oregon, 97239, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Clinical Research Associates Of Central PA , LLC- Site Number : 128

DuBois, Pennsylvania, 15801-2277, United States

Location

Clinical Research Associates Of Central PA , LLC

DuBois, Pennsylvania, 15801-2277, United States

Location

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

Hershey, Pennsylvania, 17033-2360, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Temple University Hospital- Site Number : 130

Philadelphia, Pennsylvania, 19140, United States

Location

Temple University Hospital

Philadelphia, Pennsylvania, 19140, United States

Location

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

Spartanburg, South Carolina, 29303-4225, United States

Location

Velocity Clinical Research - Spartanburg - PPDS

Spartanburg, South Carolina, 29303, United States

Location

Houston Methodist Hospital- Site Number : 113

Houston, Texas, 77030, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

University of Utah Health

Salt Lake City, Utah, 84108, United States

Location

University of Utah Health Care- Site Number : 106

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

Location

Donna McIntyre

Brisbane, Queensland, 2650, Australia

Location

Queensland Centre for Pulmonary Transplantation

Chermside, Queensland, Australia

Location

Royal Adelaide Hospital

North Adelaide, South Australia, 5065, Australia

Location

Eastern Health Clinical School

Box Hill, Victoria, 3128, Australia

Location

Investigational Site Number : 203

Fitzroy, Victoria, 3065, Australia

Location

St Vincent Hospital Melbourne

Fitzroy, Victoria, Australia

Location

Frankston Hospital

Frankston, Victoria, 3199, Australia

Location

Institute for Respiratory Health

Nedlands, Western Australia, 6009, Australia

Location

Investigational Site Number : 206

Nedlands, Western Australia, 6009, Australia

Location

Investigational Site Number : 201

Adelaide, 5000, Australia

Location

Investigational Site Number : 202

Chermside, 4032, Australia

Location

Investigational Site Number : 701

Hellerup, 2900, Denmark

Location

Investigational Site Number : 702

Vejle, 7100, Denmark

Location

NZRSI

Greenlane, Auckland, 1051, New Zealand

Location

Investigational Site Number : 403

Auckland, 1051, New Zealand

Location

Investigational Site Number : 404

Wellington, 6021, New Zealand

Location

P3 Research

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 : 903

Santiago de Compostela, La Coruña, 15706, Spain

Location

Investigational Site Number : 601

Gothenburg, 413 46, Sweden

Location

Ninewells Hospital - PPDS

Dundee, Angus, DD1 9SY, United Kingdom

Location

Investigational Site Number : 306

Wythenshawe, Cheshire West And Chester, M23 9QZ, United Kingdom

Location

Medicines Evaluation Unit

Manchester, Cheshire, M23 9QZ, United Kingdom

Location

Investigational Site Number : 302

Birmingham, Warwickshire, B15 2WB, United Kingdom

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin DeficiencyEmphysema

Interventions

alpha 1-AntitrypsinRespreeza

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesSerpinsPeptidesAmino Acids, Peptides, and ProteinsAcute-Phase ProteinsBlood ProteinsProteinsAlpha-GlobulinsSerum GlobulinsGlobulins

Study Officials

  • Clinical Sciences & Operations

    Sanofi

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Double-blind, randomized, active-control, parallel group interventional study
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 28, 2023

First Posted

May 12, 2023

Study Start

October 12, 2023

Primary Completion

August 6, 2025

Study Completion

August 6, 2025

Last Updated

August 11, 2025

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will share

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Locations