New protein replacement therapy aims to slow lung damage in rare disease
NCT ID NCT05897424
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.
Disclaimer
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the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Brigham and Women's Hospital -75 Francis Street- Site Number : 131
Boston, Massachusetts, 02115-6105, United States
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Columbia University Irving Medical Center- Site Number : 104
New York, New York, 10032-3729, United States
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David Geffen School of Medicine at UCLA- Site Number : 124
Los Angeles, California, 90095-3075, United States
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Hannibal Regional Healthcare System-HRMG-Hannibal- Site Number : 111
Hannibal, Missouri, 63401-6890, United States
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Houston Methodist Hospital- Site Number : 113
Houston, Texas, 77030-2703, United States
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Indiana University- Site Number : 127
Indianapolis, Indiana, 46202-5128, United States
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Investigational Site Number : 201
North Adelaide, South Australia, 5000, Australia
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Investigational Site Number : 202
Chermside, Queensland, 4032, Australia
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Investigational Site Number : 203
Fitzroy, Victoria, 3065, Australia
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Investigational Site Number : 204
South Brisbane, Queensland, 4101, Australia
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Investigational Site Number : 205
Frankston, Victoria, 3199, Australia
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Investigational Site Number : 207
Box Hill, Victoria, 3128, Australia
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Investigational Site Number : 304
Southampton, Hampshire, SO16 6YD, United Kingdom
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Investigational Site Number : 306
Wythenshawe, Cheshire, M23 9QZ, United Kingdom
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Investigational Site Number : 307
Exeter, Devon, EX2 5DW, United Kingdom
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Investigational Site Number : 308
Dundee, Angus, DD1 9SY, United Kingdom
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Investigational Site Number : 309
Coventry, CV2 2DX, United Kingdom
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Investigational Site Number : 403
Auckland, 1051, New Zealand
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Investigational Site Number : 404
Wellington, 6021, New Zealand
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Investigational Site Number : 501
Dublin, Dublin, D09 XR63, Ireland
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Investigational Site Number : 601
Gothenburg, 413 46, Sweden
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Investigational Site Number : 701
Hellerup, 2900, Denmark
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Investigational Site Number : 702
Vejle, 7100, Denmark
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Investigational Site Number : 801
Warsaw, Masovian Voivodeship, 01-138, Poland
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Investigational Site Number : 802
Krakow, 30-688, Poland
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Investigational Site Number : 901
Santander, Cantabria, 39008, Spain
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Investigational Site Number : 902
Madrid, 28040, Spain
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Investigational Site Number : 903
Santiago de Compostela, 15706, Spain
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National Jewish Medical and Research Center- Site Number : 123
Denver, Colorado, 80206-2761, United States
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Nuvance Health Medical Practices, Pulmonary & Sleep Specialists- Site Number : 119
Danbury, Connecticut, 06810-5038, United States
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Oregon Health and Science University- Site Number : 117
Portland, Oregon, 97239-3011, United States
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Penn State Health Milton S. Hershey Medical Center- Site Number : 122
Hershey, Pennsylvania, 17033-2360, United States
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St Joseph's Hospital and Medical Center- Site Number : 126
Phoenix, Arizona, 85013-4220, United States
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Temple University Hospital - 3401 N Broad St- Site Number : 130
Philadelphia, Pennsylvania, 19140-5103, United States
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UAB Lung Health Center- Site Number : 105
Birmingham, Alabama, 35205-2610, United States
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University of California Davis Medical Center- Site Number : 110
Sacramento, California, 95817-2201, United States
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University of Florida - Gainesville - 1600 SW Archer Rd- Site Number : 101
Gainesville, Florida, 32610-3003, United States
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University of Minnesota-420 Delaware Str SE- Site Number : 125
Minneapolis, Minnesota, 55455-0341, United States
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University of Utah Health Care- Site Number : 106
Salt Lake City, Utah, 84132-0001, United States
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Velocity Clinical Research - Spartanburg - PPDS- Site Number : 120
Spartanburg, South Carolina, 29303-4225, United States