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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05897424




Registration number
NCT05897424
Ethics application status
Date submitted
31/05/2023
Date registered
9/06/2023

Titles & IDs
Public title
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Scientific 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
Secondary ID [1] 0 0
2023-508137-14-00
Secondary ID [2] 0 0
INBRX101-01-202
Universal Trial Number (UTN)
Trial acronym
ELEVAATE OLE
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 - SAR447537

Experimental: SAR447537 (INBRX-101) - A1PI, Recombinant, Bivalent Fc Fusion Protein, in a solution for intravenous injection


Treatment: Drugs: SAR447537
A1PI, Recombinant, Bivalent Fc Fusion Protein
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Long-term safety and tolerability
Assessment method [1] 0 0
Incidence of all treatment emergent adverse events (TEAEs), TEAEs = Grade 3, serious adverse events (SAEs), TEAEs leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions).
Timepoint [1] 0 0
3 years
Secondary outcome [1] 0 0
Change in lung density by quantitative computerized tomography (CT)
Assessment method [1] 0 0
Assess the annual rate of change in lung density assessed by serial quantitative CT (15th percentile point, PD15) at total lung capacity (TLC), centrally read, from baseline to end of treatment (EoT).
Timepoint [1] 0 0
3 years
Secondary outcome [2] 0 0
Trough SAR447537 concentration changes
Assessment method [2] 0 0
Change in SAR447537 concentration levels from baseline to end of treatment
Timepoint [2] 0 0
3 years
Secondary outcome [3] 0 0
Trough serum functional AAT (fAAT) concentration changes
Assessment method [3] 0 0
Change in fAAT concentration levels from baseline to end of treatment
Timepoint [3] 0 0
3 years
Secondary outcome [4] 0 0
Covariate Analysis: Biometric Values: Weight
Assessment method [4] 0 0
Assessment of the impact of participant's weight \[in kg\] on the pharmacokinetic profile of SAR447537
Timepoint [4] 0 0
3 years
Secondary outcome [5] 0 0
Covariate Analysis: Biometric Values: Height
Assessment method [5] 0 0
Assessment of the impact of participant's height \[in cm\] on the pharmacokinetic profile of SAR447537
Timepoint [5] 0 0
3 years
Secondary outcome [6] 0 0
Covariate Analysis: Biometric Values: Age
Assessment method [6] 0 0
Assessment of the impact of participant's age \[in years\] on the pharmacokinetic profile of SAR447537
Timepoint [6] 0 0
3 years
Secondary outcome [7] 0 0
Covariate Analysis: Biometric Values: Sex
Assessment method [7] 0 0
Assessment of the impact of participant's sex \[male or female\] on the pharmacokinetic profile of SAR447537
Timepoint [7] 0 0
3 years
Secondary outcome [8] 0 0
Anti-drug antibodies
Assessment method [8] 0 0
Frequency of anti-drug antibodies (ADA) against SAR447537 as well as neutralizing ADA (NAb) against SAR447537
Timepoint [8] 0 0
3 years

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% predicted at screening and post-bronchodilator FEV1/FVC<0.7
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 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
20/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/09/2028
Actual
Sample size
Target
130
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Lung Research Qld - Chermside
Recruitment postcode(s) [1] 0 0
4032 - Chermside

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 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
Email 0 0
Contact-us@sanofi.com
Contact person for scientific queries

Data sharing statement


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/NCT05897424