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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05856331




Registration number
NCT05856331
Ethics application status
Date submitted
28/03/2023
Date registered
12/05/2023

Titles & IDs
Public title
Study of INBRX-101 Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Scientific title
Phase 2, Double-Blind, Randomized, Active-Control, Parallel Group Study to Assess the Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Safety of INBRX-101 Compared to Plasma-Derived Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema
Secondary ID [1] 0 0
2023-508084-76-00
Secondary ID [2] 0 0
INBRX101-01-201
Universal Trial Number (UTN)
Trial acronym
ELEVAATE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alpha 1-Antitrypsin Deficiency 0 0
Emphysema 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - INBRX-101
Treatment: Drugs - Zemaira

Experimental: INBRX-101 Q3W - IV every 3-weeks (Q3W) and placebo (normal saline)

Experimental: INBRX-101 Q4W - IV every 4-weeks (Q4W) and placebo (normal saline)

Active comparator: Zemaira (A1PI) - 60 mg/kg IV once weekly (QW) and placebo (normal saline)


Treatment: Drugs: INBRX-101
A1PI, Recombinant, Bivalent Fc Fusion Protein

Treatment: Drugs: Zemaira
Alpha1-Proteinase Inhibitor (Human)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Serum functional AAT (fAAT) levels at steady-state
Timepoint [1] 0 0
32 Weeks
Secondary outcome [1] 0 0
fAAT Concentration changes
Timepoint [1] 0 0
32 Weeks
Secondary outcome [2] 0 0
Days with fAAT above the lower limit of the normal range
Timepoint [2] 0 0
32 weeks
Secondary outcome [3] 0 0
Incidence of TEAEs
Timepoint [3] 0 0
32 Weeks
Secondary outcome [4] 0 0
Anti-drug antibodies
Timepoint [4] 0 0
32 Weeks
Secondary outcome [5] 0 0
Population Pharmacokinetics: Clearance
Timepoint [5] 0 0
32 Weeks
Secondary outcome [6] 0 0
Population Pharmacokinetics: Volume of Distribution
Timepoint [6] 0 0
32 Weeks
Secondary outcome [7] 0 0
Covariate Analysis: Biometric Values: Weight
Timepoint [7] 0 0
32 Weeks
Secondary outcome [8] 0 0
Covariate Analysis: Biometric Values: Height
Timepoint [8] 0 0
32 Weeks
Secondary outcome [9] 0 0
Covariate Analysis: Biometric Values: Age
Timepoint [9] 0 0
32 Weeks
Secondary outcome [10] 0 0
Covariate Analysis: Biometric Values: Sex
Timepoint [10] 0 0
32 Weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2025
Actual
Sample size
Target
90
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Donna McIntyre - Brisbane
Recruitment hospital [2] 0 0
Queensland Centre for Pulmonary Transplantation - Chermside
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - North Adelaide
Recruitment hospital [4] 0 0
Eastern Health Clinical School - Box Hill
Recruitment hospital [5] 0 0
St Vincent Hospital Melbourne - Fitzroy
Recruitment hospital [6] 0 0
Frankston Hospital - Frankston
Recruitment hospital [7] 0 0
Institute for Respiratory Health - Nedlands
Recruitment postcode(s) [1] 0 0
2650 - Brisbane
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
5065 - North Adelaide
Recruitment postcode(s) [4] 0 0
3128 - Box Hill
Recruitment postcode(s) [5] 0 0
- Fitzroy
Recruitment postcode(s) [6] 0 0
3199 - Frankston
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Connecticut
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
South Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Utah
Country [18] 0 0
New Zealand
State/province [18] 0 0
Auckland
Country [19] 0 0
New Zealand
State/province [19] 0 0
Wellington
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Angus
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Cheshire
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Devon
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Hampshire
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Warwickshire

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sanofi
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05856331