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New protein replacement therapy aims to slow lung damage in rare disease

NCT ID NCT05897424

Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a new drug called SAR447537 (INBRX-101) in 185 adults with alpha-1 antitrypsin deficiency (AATD) emphysema, a genetic condition that causes lung damage. The drug is a lab-made protein designed to replace the missing enzyme and slow disease progression. The main goal is to check long-term safety, with a secondary focus on whether it can reduce the rate of lung density loss over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SAR447537 (INBRX-101), a lab-made protein designed to replace the missing alpha-1 antitrypsin enzyme
What this could lead to
If successful, this could provide a more effective and convenient long-term treatment option to slow lung damage in people with AATD emphysema.
What could go wrong
This is an early-phase, open-label study with no placebo group, so results may not prove the drug works better than existing therapies. Long-term safety is still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 185 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Sep 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency pulmonary emphysema

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alpha 1 antitrypsin deficiency Emphysema

Contacts and locations

Locations

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

    Boston, Massachusetts, 02115-6105, United States

  • Columbia University Irving Medical Center- Site Number : 104

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

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

    Los Angeles, California, 90095-3075, United States

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

    Hannibal, Missouri, 63401-6890, United States

  • Houston Methodist Hospital- Site Number : 113

    Houston, Texas, 77030-2703, United States

  • Indiana University- Site Number : 127

    Indianapolis, Indiana, 46202-5128, United States

  • Investigational Site Number : 201

    North Adelaide, South Australia, 5000, Australia

  • Investigational Site Number : 202

    Chermside, Queensland, 4032, Australia

  • Investigational Site Number : 203

    Fitzroy, Victoria, 3065, Australia

  • Investigational Site Number : 204

    South Brisbane, Queensland, 4101, Australia

  • Investigational Site Number : 205

    Frankston, Victoria, 3199, Australia

  • Investigational Site Number : 207

    Box Hill, Victoria, 3128, Australia

  • Investigational Site Number : 304

    Southampton, Hampshire, SO16 6YD, United Kingdom

  • Investigational Site Number : 306

    Wythenshawe, Cheshire, M23 9QZ, United Kingdom

  • Investigational Site Number : 307

    Exeter, Devon, EX2 5DW, United Kingdom

  • Investigational Site Number : 308

    Dundee, Angus, DD1 9SY, United Kingdom

  • Investigational Site Number : 309

    Coventry, CV2 2DX, United Kingdom

  • Investigational Site Number : 403

    Auckland, 1051, New Zealand

  • Investigational Site Number : 404

    Wellington, 6021, New Zealand

  • Investigational Site Number : 501

    Dublin, Dublin, D09 XR63, Ireland

  • Investigational Site Number : 601

    Gothenburg, 413 46, Sweden

  • Investigational Site Number : 701

    Hellerup, 2900, Denmark

  • Investigational Site Number : 702

    Vejle, 7100, Denmark

  • Investigational Site Number : 801

    Warsaw, Masovian Voivodeship, 01-138, Poland

  • Investigational Site Number : 802

    Krakow, 30-688, Poland

  • Investigational Site Number : 901

    Santander, Cantabria, 39008, Spain

  • Investigational Site Number : 902

    Madrid, 28040, Spain

  • Investigational Site Number : 903

    Santiago de Compostela, 15706, Spain

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

    Denver, Colorado, 80206-2761, United States

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

    Danbury, Connecticut, 06810-5038, United States

  • Oregon Health and Science University- Site Number : 117

    Portland, Oregon, 97239-3011, United States

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

    Hershey, Pennsylvania, 17033-2360, United States

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

    Phoenix, Arizona, 85013-4220, United States

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

    Philadelphia, Pennsylvania, 19140-5103, United States

  • UAB Lung Health Center- Site Number : 105

    Birmingham, Alabama, 35205-2610, United States

  • University of California Davis Medical Center- Site Number : 110

    Sacramento, California, 95817-2201, United States

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

    Gainesville, Florida, 32610-3003, United States

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

    Minneapolis, Minnesota, 55455-0341, United States

  • University of Utah Health Care- Site Number : 106

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

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

    Spartanburg, South Carolina, 29303-4225, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.