Clinical trial NCT05856331: Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Alpha 1-Antitrypsin Deficiency
Emphysema
Keywords: AATD
Alpha 1-Antitrypsin Deficiency
Emphysema
SAR447537
INBRX-101
A1PI
AAT
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2023-05-12
Enrollment: 99
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Double-blind, randomized, active-control, parallel group interventional study
Primary purpose: TREATMENT

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

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.

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% and ≤ 80% predicted at screening
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 (INBRX-101), A1PI or human IgG
3. Known selective or severe Immunoglobulin A (IgA) deficiency
4. Known or suspected diagnosis of type 1 diabetes or diagnosed with uncontrolled type 2 diabetes
5. Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days
6. On waiting list for lung or liver transplant
7. Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening
8. Evidence of decompensated cirrhosis
9. Active cancers or has a history of malignancy within 5 years prior to screening
10. History of unstable cor pulmonale
11. Clinically significant congestive heart failure

Primary outcomes:
measure: Serum functional AAT (fAAT) levels at steady-state
timeFrame: 32 Weeks
Secondary outcomes:
measure: fAAT Concentration changes
timeFrame: 32 Weeks
measure: Days with fAAT above the lower limit of the normal range
timeFrame: 32 weeks
measure: Incidence of TEAEs
timeFrame: 32 Weeks
measure: Anti-drug antibodies
timeFrame: 32 Weeks
measure: Population Pharmacokinetics: Clearance
timeFrame: 32 Weeks
measure: Population Pharmacokinetics: Volume of Distribution
timeFrame: 32 Weeks
measure: Covariate Analysis: Biometric Values: Weight
timeFrame: 32 Weeks
measure: Covariate Analysis: Biometric Values: Height
timeFrame: 32 Weeks
measure: Covariate Analysis: Biometric Values: Age
timeFrame: 32 Weeks
measure: Covariate Analysis: Biometric Values: Sex
timeFrame: 32 Weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham- Site Number : 105
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: St Joseph's Hospital and Medical Center- Site Number : 126
state: Arizona
city: Phoenix
country: United States
facility: St Joseph's Hospital and Medical Center
state: Arizona
city: Los Angeles
country: United States
facility: David Geffen School of Medicine- Site Number : 124
state: California
city: Los Angeles
country: United States
facility: David Geffen School of Medicine
state: California
city: Sacramento
country: United States
facility: UC Davis Comprehensive Cancer Center- Site Number : 110
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Denver
country: United States
facility: National Jewish Medical and Research Center- Site Number : 123
state: Colorado
city: Denver
country: United States
facility: National Jewish Medical and Research Center
state: Colorado
city: Danbury
country: United States
facility: Western Connecticut Medical Group- Site Number : 119
state: Connecticut
city: Danbury
country: United States
facility: Western Connecticut Medical Group
state: Connecticut
city: Washington
country: United States
facility: GW Medical Faculty Associates - GW Cancer& Blood Disorders- Site Number : 107
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida College of Medicine- Site Number : 101
state: Florida
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: Bruce W. Carter Miami VA Medical Center - NAVREF - PPDS- Site Number : 114
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Tampa
country: United States
facility: Pulmonary and Sleep of Tampa Bay- Site Number : 115
state: Florida
city: Tampa
country: United States
facility: Pulmonary and Sleep of Tampa Bay
state: Florida
city: Weston
country: United States
facility: Cleveland Clinic Florida
state: Florida
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center- Site Number : 112
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
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 - Lung Center
state: Massachusetts
city: Minneapolis
country: United States
facility: M Health Fairview Clinics and Surgery Center - Minneapolis- Site Number : 125
state: Minnesota
city: Minneapolis
country: United States
facility: M Health Fairview Clinics and Surgery Center - Minneapolis
state: Minnesota
city: Hannibal
country: United States
facility: Hannibal Regional Healthcare System-HRMG-Hannibal- Site Number : 111
state: Missouri
city: Hannibal
country: United States
facility: Hannibal Clinic
state: Missouri
city: New York
country: United States
facility: Columbia University Irving Medical Center- Site Number : 104
state: New York
city: New York
country: United States
facility: Columbia University
state: New York
city: Syracuse
country: United States
facility: Pulmonary Health Physicians
state: New York
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 117
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: DuBois
country: United States
facility: Clinical Research Associates Of Central PA , LLC- Site Number : 128
state: Pennsylvania
city: DuBois
country: United States
facility: Clinical Research Associates Of Central PA , LLC
state: Pennsylvania
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center- Site Number : 122
state: Pennsylvania
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University Hospital- Site Number : 130
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Spartanburg
country: United States
facility: Velocity Clinical Research - Spartanburg - PPDS- Site Number : 120
state: South Carolina
city: Spartanburg
country: United States
facility: Velocity Clinical Research - Spartanburg - PPDS
state: South Carolina
city: Houston
country: United States
facility: Houston Methodist Hospital- Site Number : 113
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health
state: Utah
city: Salt Lake City
country: United States
facility: University of Utah Health Care- Site Number : 106
state: Utah
city: Brisbane
country: Australia
facility: Donna McIntyre
state: Queensland
city: Chermside
country: Australia
facility: Queensland Centre for Pulmonary Transplantation
state: Queensland
city: South Brisbane
country: Australia
facility: Investigational Site Number : 204
state: Queensland
city: North Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Box Hill
country: Australia
facility: Eastern Health Clinical School
state: Victoria
city: Box Hill
country: Australia
facility: Investigational Site Number : 207
state: Victoria
city: Fitzroy
country: Australia
facility: Investigational Site Number : 203
state: Victoria
city: Fitzroy
country: Australia
facility: St Vincent Hospital Melbourne
state: Victoria
city: Frankston
country: Australia
facility: Frankston Hospital
state: Victoria
city: Nedlands
country: Australia
facility: Institute for Respiratory Health
state: Western Australia
city: Nedlands
country: Australia
facility: Investigational Site Number : 206
state: Western Australia
city: Adelaide
country: Australia
facility: Investigational Site Number : 201
city: Chermside
country: Australia
facility: Investigational Site Number : 202
city: Frankston
country: Australia
facility: Investigational Site Number : 205
city: Hellerup
country: Denmark
facility: Investigational Site Number : 701
city: Vejle
country: Denmark
facility: Investigational Site Number : 702
city: Greenlane
country: New Zealand
facility: NZRSI
state: Auckland
city: Auckland
country: New Zealand
facility: Investigational Site Number : 403
city: Wellington
country: New Zealand
facility: Investigational Site Number : 404
city: Wellington
country: New Zealand
facility: P3 Research
city: Warszawa
country: Poland
facility: Investigational Site Number : 801
state: Mazowieckie
city: Kraków
country: Poland
facility: Investigational Site Number : 802
city: Santander
country: Spain
facility: Investigational Site Number : 901
state: Cantabria
city: Santiago de Compostela
country: Spain
facility: Investigational Site Number : 903
city: Göteborg
country: Sweden
facility: Investigational Site Number : 601
city: Dundee
country: United Kingdom
facility: Ninewells Hospital - PPDS
state: Angus
city: Wythenshawe
country: United Kingdom
facility: Investigational Site Number : 306
state: Cheshire West And Chester
city: Manchester
country: United Kingdom
facility: Medicines Evaluation Unit
state: Cheshire
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter Hospital (Wonford) - Barrack Rd
state: Devon
city: Southampton
country: United Kingdom
facility: Investigational Site Number : 304
state: Hampshire
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
state: Hampshire
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 302
state: Warwickshire
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham
state: Warwickshire
city: Exeter
country: United Kingdom
facility: Investigational Site Number : 307
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: