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Alpha 1-Antitrypsin DeficiencyPhase 2ELEVAATENCT05856331

Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

A Phase 2 study testing SAR447537 and Zemaira in people with Alpha 1-Antitrypsin Deficiency and Emphysema, run by Sanofi at 65 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Alpha 1-Antitrypsin Deficiency, Emphysema
What is being tested?
SAR447537 and Zemaira
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
99 participants
Where
65 sitesin 8 countries
Study dates
12 Oct 2023to 6 Aug 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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
Show all 11Show fewer
  • 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

Trial locations and contacts

65 sites in 8 countries.

65 sites in 8 countries

United States39 sites

  • University of Alabama at Birmingham- Site Number : 105

    Birmingham, Alabama, 35233-2110

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35249

  • Western Connecticut Medical Group

    Danbury, Connecticut, 06810

  • National Jewish Medical and Research Center- Site Number : 123

    Denver, Colorado, 80206-2761

  • National Jewish Medical and Research Center

    Denver, Colorado, 80206

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

    DuBois, Pennsylvania, 15801-2277

  • Clinical Research Associates Of Central PA , LLC

    DuBois, Pennsylvania, 15801-2277

  • University of Florida College of Medicine- Site Number : 101

    Gainesville, Florida, 32610

  • University of Florida

    Gainesville, Florida, 32610

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

    Hannibal, Missouri, 63401-6890

  • Hannibal Clinic

    Hannibal, Missouri, 63401

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

    Hershey, Pennsylvania, 17033-2360

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Houston Methodist Hospital- Site Number : 113

    Houston, Texas, 77030

  • Houston Methodist Hospital

    Houston, Texas, 77030

  • Indiana University

    Indianapolis, Indiana, 46202-5128

  • David Geffen School of Medicine- Site Number : 124

    Los Angeles, California, 90095-3075

  • David Geffen School of Medicine

    Los Angeles, California, 90095

  • Loyola University Medical Center- Site Number : 112

    Maywood, Illinois, 60153

  • Loyola University Medical Center

    Maywood, Illinois, 60153

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

    Miami, Florida, 33125-1624

  • University of Miami

    Miami, Florida, 33136

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

    Minneapolis, Minnesota, 55455-4800

  • M Health Fairview Clinics and Surgery Center - Minneapolis

    Minneapolis, Minnesota, 55455-4800

  • Columbia University Irving Medical Center- Site Number : 104

    New York, New York, 10032

  • Temple University Hospital- Site Number : 130

    Philadelphia, Pennsylvania, 19140

  • Temple University Hospital

    Philadelphia, Pennsylvania, 19140

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

    Phoenix, Arizona, 85013-4220

  • St Joseph's Hospital and Medical Center

    Phoenix, Arizona, 85013-4220

  • Oregon Health and Science University- Site Number : 117

    Portland, Oregon, 97239

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • UC Davis Comprehensive Cancer Center- Site Number : 110

    Sacramento, California, 95817

  • UC Davis Medical Center

    Sacramento, California, 95817

  • University of Utah Health

    Salt Lake City, Utah, 84108

  • University of Utah Health Care- Site Number : 106

    Salt Lake City, Utah, 84132-0001

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

    Spartanburg, South Carolina, 29303-4225

  • Velocity Clinical Research - Spartanburg - PPDS

    Spartanburg, South Carolina, 29303

  • Pulmonary and Sleep of Tampa Bay- Site Number : 115

    Tampa, Florida, 33607-6316

  • Pulmonary and Sleep of Tampa Bay

    Tampa, Florida, 33607

Other trials in United States

Australia11 sites

  • Investigational Site Number : 201

    Adelaide, 5000

  • Eastern Health Clinical School

    Box Hill, Victoria, 3128

  • Donna McIntyre

    Brisbane, Queensland, 2650

  • Queensland Centre for Pulmonary Transplantation

    Chermside, Queensland

  • Investigational Site Number : 202

    Chermside, 4032

  • Investigational Site Number : 203

    Fitzroy, Victoria, 3065

  • St Vincent Hospital Melbourne

    Fitzroy, Victoria

  • Frankston Hospital

    Frankston, Victoria, 3199

  • Institute for Respiratory Health

    Nedlands, Western Australia, 6009

  • Investigational Site Number : 206

    Nedlands, Western Australia, 6009

  • Royal Adelaide Hospital

    North Adelaide, South Australia, 5065

Other trials in Australia

New Zealand4 sites

  • Investigational Site Number : 403

    Auckland, 1051

  • NZRSI

    Greenlane, Auckland, 1051

  • Investigational Site Number : 404

    Wellington, 6021

  • P3 Research

    Wellington, 6021

Other trials in New Zealand

United Kingdom4 sites

  • Investigational Site Number : 302

    Birmingham, Warwickshire, B15 2WB

  • Ninewells Hospital - PPDS

    Dundee, Angus, DD1 9SY

  • Medicines Evaluation Unit

    Manchester, Cheshire, M23 9QZ

  • Investigational Site Number : 306

    Wythenshawe, Cheshire West And Chester, M23 9QZ

Other trials in United Kingdom

Denmark2 sites

  • Investigational Site Number : 701

    Hellerup, 2900

  • Investigational Site Number : 702

    Vejle, 7100

Other trials in Denmark

Poland2 sites

  • Investigational Site Number : 802

    Krakow, 30-688

  • Investigational Site Number : 801

    Warsaw, Masovian Voivodeship, 01-138

Other trials in Poland

Spain2 sites

  • Investigational Site Number : 901

    Santander, Cantabria, 39008

  • Investigational Site Number : 903

    Santiago de Compostela, La Coruña, 15706

Other trials in Spain

Sweden1 site

  • Investigational Site Number : 601

    Gothenburg, 413 46

Other trials in Sweden

About this study

Official title: Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of SAR447537 (INBRX-101) Compared to Plasma-Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema

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

Read the detailed description

This is a Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of SAR447537 (INBRX-101) Compared to Plasma-Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.

Being tested
SAR447537, Zemaira
Keywords
AATD, Alpha 1-Antitrypsin Deficiency, Emphysema, SAR447537, INBRX-101, A1PI, AAT
Medical subject headings
alpha 1-Antitrypsin Deficiency, Emphysema

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SAR447537Drug

    A1PI, Recombinant, Bivalent Fc Fusion Protein

  • ZemairaDrug

    Also called Respreeza

    Alpha1-Proteinase Inhibitor (Human)

Study groups in NCT05856331
Study groupRoleWhat they receive
SAR447537 (INBRX-101) Q3WExperimentalIV every 3-weeks (Q3W) and placebo (normal saline)
Receives: Drug: SAR447537
SAR447537 (INBRX-101) Q4WExperimentalIV every 4-weeks (Q4W) and placebo (normal saline)
Receives: Drug: SAR447537
Zemaira (A1PI)Active comparator60 mg/kg IV once weekly (QW) and placebo (normal saline)
Receives: Drug: Zemaira

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 32 Weeks

Secondary outcomes (10)

  1. fAAT Concentration changes

    Mean change in serum fAAT concentration from baseline to fAAT average concentration at steady-state (Cavg, ss) in participants treated with SAR447537 compared to A1PI.

    Measured: 32 Weeks

  2. Days with fAAT above the lower limit of the normal range

    Percentage of days with fAAT above the lower limit of the normal range during steady-state dosing in participants treated with SAR447537 compared to A1PI.

    Measured: 32 weeks

  3. Incidence of TEAEs

    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).

    Measured: 32 Weeks

  4. Anti-drug antibodies

    Frequency of anti-drug antibodies (ADA) against SAR447537 and endogenous AAT, as well as neutralizing ADA (NAb) against SAR447537 and endogenous AAT.

    Measured: 32 Weeks

  5. Population Pharmacokinetics: Clearance

    Modeling by means of appropriate software to characterize the pharmacokinetic profile of SAR447537 via estimation of the parameter clearance

    Measured: 32 Weeks

  6. Population Pharmacokinetics: Volume of Distribution

    Modeling by means of appropriate software to characterize the pharmacokinetic profile of SAR447537 via estimation of the parameter volume of distribution

    Measured: 32 Weeks

  7. Covariate Analysis: Biometric Values: Weight

    Assessment of the impact of participant's weight [in kg] on the pharmacokinetic profile of SAR447537

    Measured: 32 Weeks

  8. Covariate Analysis: Biometric Values: Height

    Assessment of the impact of participant's height [in cm] on the pharmacokinetic profile of SAR447537

    Measured: 32 Weeks

  9. Covariate Analysis: Biometric Values: Age

    Assessment of the impact of participant's age [in years] on the pharmacokinetic profile of SAR447537

    Measured: 32 Weeks

  10. Covariate Analysis: Biometric Values: Sex

    Assessment of the impact of participant's sex [male or female] on the pharmacokinetic profile of SAR447537

    Measured: 32 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 May 2023
  2. Study start12 Oct 2023
  3. Primary completion6 Aug 2025
  4. Study completion6 Aug 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Double-blind, randomized, active-control, parallel group interventional study
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
99 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05856331
Sponsor's protocol ID
INBRX101-01-201
Other IDs
2023-508084-76-00, 2023
Acronym
ELEVAATE
Status last verified
Aug 2025
First submitted
28 Mar 2023
Last updated
11 Aug 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SAR447537 and Zemaira in people with Alpha 1-Antitrypsin Deficiency and Emphysema, run by Sanofi at 65 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 65 sites in United States, Australia, New Zealand, United Kingdom, Denmark and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Serum functional AAT (fAAT) levels at steady-state (time frame: 32 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05856331.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2025.

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