Clinical trial NCT05897424: Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Official title: A Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Alpha 1-Antitrypsin Deficiency
Emphysema
Keywords: AATD
Alpha 1-Antitrypsin Deficiency
Emphysema
INBRX-101
AAT
SAR447537
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-06-09
Enrollment: 130
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Multicenter, single arm, open-label extension study
Primary purpose: TREATMENT

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

Detailed description: This is a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.

Eligibility criteria: 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:

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. Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes
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
10. Clinically significant congestive heart failure

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Primary outcomes:
measure: Long-term safety and tolerability
timeFrame: 3 years
Secondary outcomes:
measure: Change in lung density by quantitative computerized tomography (CT)
timeFrame: 3 years
measure: Trough SAR447537 concentration changes
timeFrame: 3 years
measure: Trough serum functional AAT (fAAT) concentration changes
timeFrame: 3 years
measure: Covariate Analysis: Biometric Values: Weight
timeFrame: 3 years
measure: Covariate Analysis: Biometric Values: Height
timeFrame: 3 years
measure: Covariate Analysis: Biometric Values: Age
timeFrame: 3 years
measure: Covariate Analysis: Biometric Values: Sex
timeFrame: 3 years
measure: Anti-drug antibodies
timeFrame: 3 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham- Site Number : 105
state: Alabama
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center- Site Number : 126
state: Arizona
city: Los Angeles
country: United States
facility: David Geffen School of Medicine at UCLA- Site Number : 124
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Denver
country: United States
facility: National Jewish Health Medical Center- Site Number : 123
state: Colorado
city: Danbury
country: United States
facility: Nuvance Health Medical Practices, Pulmonary & Sleep Specialists- Site Number : 119
state: Connecticut
city: Brandon
country: United States
facility: Meris Clinical Research-310 Oakfield Dr- Site Number : 115
state: Florida
city: Gainesville
country: United States
facility: University of Florida - Gainesville - 1600 SW Archer Rd- Site Number : 101
state: Florida
city: Indianapolis
country: United States
facility: Indiana University Health University Hospital- Site Number : 127
state: Indiana
city: Boston
country: United States
facility: Brigham and Women's Hospital -75 Francis Street- Site Number : 131
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota-420 Delaware Str SE- Site Number : 125
state: Minnesota
city: Hannibal
country: United States
facility: Hannibal Regional Healthcare System-HRMG-Hannibal- Site Number : 111
state: Missouri
city: New York
country: United States
facility: Columbia University Irving Medical Center- Site Number : 104
state: New York
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 117
state: Oregon
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center- Site Number : 122
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University Hospital - 3401 N Broad St- Site Number : 130
state: Pennsylvania
city: Spartanburg
country: United States
facility: Velocity Clinical Research - Spartanburg - PPDS- Site Number : 120
state: South Carolina
city: Houston
country: United States
facility: Houston Methodist Hospital- Site Number : 113
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Care- Site Number : 106
state: Utah
city: Brisbane
country: Australia
facility: Investigational Site Number : 202
state: Queensland
city: South Brisbane
country: Australia
facility: Investigational Site Number : 204
state: Queensland
city: Adelaide
country: Australia
facility: Investigational Site Number : 201
state: South Australia
city: Frankston
country: Australia
facility: Investigational Site Number : 205
state: Victoria
city: Melbourne
country: Australia
facility: Investigational Site Number : 203
state: Victoria
city: Melbourne
country: Australia
facility: Investigational Site Number : 207
state: Victoria
city: Hellerup
country: Denmark
facility: Investigational Site Number : 701
city: Vejle
country: Denmark
facility: Investigational Site Number : 702
city: Dublin 9
country: Ireland
facility: Investigational Site Number : 501
state: Dublin
city: Auckland
country: New Zealand
facility: Investigational Site Number : 403
city: Wellington
country: New Zealand
facility: Investigational Site Number : 404
city: Krakow
country: Poland
facility: Investigational Site Number : 802
state: Malopolskie
city: Santiago de Compostela
country: Spain
facility: Investigational Site Number : 903
state: A Coruña [La Coruña]
city: Santander
country: Spain
facility: Investigational Site Number : 901
state: Cantabria
city: Madrid
country: Spain
facility: Investigational Site Number : 902
city: Gothenburg
country: Sweden
facility: Investigational Site Number : 601
city: Dundee
country: United Kingdom
facility: Investigational Site Number : 308
state: Angus
city: Exeter
country: United Kingdom
facility: Investigational Site Number : 307
state: Devon
city: Southampton
country: United Kingdom
facility: Investigational Site Number : 304
state: Hampshire
city: Coventry
country: United Kingdom
facility: Investigational Site Number : 309
city: Manchester
country: United Kingdom
facility: Investigational Site Number: 306
city: Wythenshawe
country: United Kingdom
facility: Investigational Site Number : 306
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: