Human alpha-1-proteinase inhibitor immunoglobulin G fusion protein, recombinant for Alpha-1 antitrypsin deficiency (AATD) | Emphysema

Inclusion criteria

• {"criterion_text":"- Males or females 18-80 years of age, inclusive, at the time of screening."}
• {"criterion_text":"- Diagnosis of AATD."}
• {"criterion_text":"- Evidence of emphysema secondary to AATD."}
• {"criterion_text":"- FEV1 of ≥ 30% predicted at screening and post-bronchodilator FEV1/FVC<0.7."}
• {"criterion_text":"- Current non-smoking status."}

Exclusion criteria

• {"criterion_text":"- For Newly Identified Participants: Receipt of A1PI augmentation therapy within 5 weeks prior to the first dose of study drug."}
• {"criterion_text":"- Known or suspected allergy to components of SAR447537, A1PI or human IgG."}
• {"criterion_text":"- Received IV immunoglobulins, monoclonal antibodies and/or other biologic therapies within 30 days."}
• {"criterion_text":"- On waiting list for lung or liver transplant."}
• {"criterion_text":"- Acute respiratory tract infection or COPD exacerbation within 4 weeks prior to or during screening."}
• {"criterion_text":"- Evidence of decompensated cirrhosis."}
• {"criterion_text":"- Active cancers or has a history of malignancy within 5 years prior to screening."}
• {"criterion_text":"- History of unstable cor pulmonale."}
• {"criterion_text":"- Uncontrolled diabetes mellitus despite adequate antidiabetic pharmacologic treatment with a screening HbA1c value ≥9%"}

Primary endpoints

• {"endpoint_text":"- Incidences of all treatment-emergent adverse events (TEAEs), TEAEs ≥Grade 3, serious adverse events (SAEs), TEAEs requiring withdrawal from investigational product (IP) treatment leading to discontinuation from SAR447537, and adverse events of special interest (AESIs) (including infusion-related reactions) leading to discontinuation from and adverse events of special interest (AESIs) (including infusion-related reactions).","definition_or_measurement_approach":"Incidences will be recorded as treatment-emergent adverse events (TEAEs), graded by severity (including Grade ≥3), serious adverse events (SAEs), TEAEs requiring withdrawal/discontinuation, and adverse events of special interest (AESIs) including infusion-related reactions."}

Secondary endpoints

• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Measured by serial quantitative CT centrally read, using PD15 at TLC from baseline to End of Treatment (EoT)."}
• {"endpoint_text":"- Serum PK profile of SAR447537 and fAAT.","definition_or_measurement_approach":"Serum pharmacokinetic (PK) profiling for SAR447537 and functional AAT (fAAT) measured in serum samples."}
• {"endpoint_text":"- Population PK modeling to assess impact of physiologically relevant participant characteristics (eg, covariates including, but not limited to, age, sex, body size, ethnicity) and disease on PK of SAR447537 through Population PK modeling.","definition_or_measurement_approach":"Population PK modelling to assess covariate effects (age, sex, body size, ethnicity, disease) on SAR447537 PK."}
• {"endpoint_text":"- Incidence of anti-drug antibodies (ADAs) against SAR447537 as well as neutralizing anti-drug antibodies (NAbs) against SAR447537.","definition_or_measurement_approach":"Measurement of incidence of anti-drug antibodies (ADAs) and neutralizing antibodies (NAbs) to SAR447537 using immunogenicity assays."}

Methods

• Clinical study leaflets (country-specific clinical-study-leaflet documents present for PL, ES, DK, SE, IE).
• Email templates for participant outreach (country-specific email templates present: PL, ES, IE, DK).
• Phone-and-email scripts for recruitment outreach (country-specific phone-and-email-script documents).
• Doctor-to-doctor referral letters and GP letters (documents present for several countries, e.g., Spain, Ireland, Poland).
• Local advertisements (document 'Local-ad' present for Sweden).
• Scout platform materials (Scout-Pass, Reloadable-Scout-Pass, Scout brochures and user guides) and study brochures to engage participants.
• Country-specific recruitment informed consent procedure documents (e.g., Recruitment-Informed-Consent-Procedure_PL, DK versions).

Poland

Earliest CTIS Part Ii Submission Date

26-08-2024

Latest Decision Or Authorization Date

12-09-2025

Processing Time Days

382

Number Of Sites

2

Number Of Participants

11

Denmark

Earliest CTIS Part Ii Submission Date

26-08-2024

Latest Decision Or Authorization Date

12-09-2025

Processing Time Days

382

Number Of Sites

2

Number Of Participants

11

Spain

Earliest CTIS Part Ii Submission Date

28-08-2024

Latest Decision Or Authorization Date

24-09-2025

Processing Time Days

392

Number Of Sites

3

Number Of Participants

11

Ireland

Earliest CTIS Part Ii Submission Date

16-08-2024

Latest Decision Or Authorization Date

30-10-2025

Processing Time Days

440

Number Of Sites

1

Number Of Participants

7

Sweden

Earliest CTIS Part Ii Submission Date

22-08-2024

Latest Decision Or Authorization Date

03-03-2026

Processing Time Days

558

Number Of Sites

1

Number Of Participants

8

Primary sponsor

Full Name

Sanofi AATD Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Pharmaceutical Product Development LLC (PPD Development LP / PPD International Holdings LLC)

Responsibilities

Multiple operational responsibilities including Medical Monitoring, Clinical Supplies, sample management for EU/UK and other clinical trial support tasks (codes and values provided in sponsor duties).

Name

Suvoda LLC

Responsibilities

eClinical trial systems (code:3 indicated in sponsor duties).

Name

Medidata Solutions Inc.

Responsibilities

Data management/eClinical services (code:7 indicated in sponsor duties).

Third parties

• {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"antigenic AAT sample analysis","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Canada","full_name":"Cellcarta Biosciences Inc.","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code:7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code:3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Professional Case Management Clinical Trials LLC","duties_or_roles":"Home Health","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"University Of Dundee(The)","duties_or_roles":"code:4","organisation_type":"Educational Institution"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Medical Monitoring, Clinical Supplies","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Vida Diagnostics Inc.","duties_or_roles":"CT Scan Imaging Central Read","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Participant Compensation and Participant Travel Services","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Sample Manager for EU and UK; code:4","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Denmark","full_name":"Clinigma ApS","duties_or_roles":"Conduct Subject phone interviews","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"University Of Florida","duties_or_roles":"code:4","organisation_type":"Educational Institution"}
• {"country":"United Kingdom","full_name":"The University Of Birmingham","duties_or_roles":"code:4","organisation_type":"Educational Institution"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Respiratory/Spirometry","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Vida Diagnostics Inc.","duties_or_roles":"CT Scan Imaging Central Read","organisation_type":"Non-Pharmaceutical company"}