Safety and Tolerability Study in Adults With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088

NCT04802057 | Terminated Phase 1 FDA Drug

• 4 other identifiers: LTS16637U1111-1244-08082023-510210-682019-004423-21
• Study Type: interventional
• Target: 9
• Locations: 5 countries
• Sites: 5

Brief Summary

Primary Objective: To assess the long-term safety and tolerability in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088 Secondary Objective: To assess, in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088:

• The long-term effect of SAR445088 on complement mediated hemolysis
• The long-term pharmacodynamics (PD) effect of SAR445088 relating to complement inhibition
• The long-term pharmacokinetic (PK) profile of SAR445088
• The long-term immunogenicity of SAR445088

Conditions

Autoimmune Haemolytic Anaemia

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-emergent adverse events (TEAE)

  The number of participants experiencing TEAEs will be reported for the overall safety population.

  Day 1 or Day 1-IV to end of study, approximately 6 years

Secondary Outcomes (11)

• Mean change from baseline in total bilirubin over time

  Day 1 or Day 1-IV to end of study, approximately 6 years

• Mean change from baseline in hemoglobin over time

  Day 1 or Day 1-IV to end of study, approximately 6 years

• Mean change from baseline in lactate dehydrogenase over time

  Day 1 or Day 1-IV to end of study, approximately 6 years

• Mean change from baseline in reticulocyte count over time

  Day 1 or Day 1-IV to end of study, approximately 6 years

• Complement System Classical Pathway Levels as Measured by WIESLAB Assay

  Day 1 or Day 1-IV to Week 48-IV

Study Arms (1)

SAR445088

EXPERIMENTAL

Repeat dose of SAR445088

Drug: SAR445088

Interventions

SAR445088 DRUG

Pharmaceutical form: solution for injection Route of administration: IV and SC (Part 1) IV (Part 2)

SAR445088

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female adult patients ≥18 years of age with CAD who were previously treated with SAR445088 and met criteria the below criteria for entry into Part 1:
• met the eligibility criteria of a previous study evaluating SAR445088;
• successfully enrolled and completed dosing in a previous study evaluating SAR445088;
• successfully completed end of study procedures in a previous study evaluating SAR445088; and
• per Investigator judgement, had a favorable benefit-to-risk profile after receiving SAR445088.
• OR were never treated with SAR445088 before entering Part 2, and met the below criteria to establish CAD diagnosis:
• chronic hemolysis;
• polyspecific direct antiglobulin test (DAT) positive status;
• monospecific DAT strongly positive for C3d;
• cold agglutinin \[CAg\] titer ≥64 at 4°C;
• IgG DAT ≤1+;
• hemoglobin level ≤10 g/dL;
• elevated bilirubin not attributable to liver disease;
• Documented vaccinations against encapsulated bacterial pathogens given within five years of enrollment and at least 14 days prior to dosing (vaccinations have to be initiated at least 14 days prior to dosing and completed before Week 5 of Part 2).
• Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.

You may not qualify if:

• Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high-grade hematologic malignancy, or known solid organ tumor.
• Clinically relevant infection within 1 month of enrollment.
• Clinical diagnosis of systemic lupus erythematosus (SLE).
• Treatment with anti-CD20 monotherapy within 3 months or anti-CD20 combination therapies within 6 months prior to screening.
• Concurrent treatment with systemic immunosuppressive agents targeting B- or T-cell function and/or cytotoxic agents within 3 months prior to screening. Concurrent treatment with other systemic immunosuppressants within 5.5 half-lives of the drug prior to screening.
• Any specific complement system inhibitor other than SAR445088 (eg, eculizumab) within 3 months prior to screening.
• Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within 3 months prior to screening.
• History of hypersensitivity to SAR445088 or any of its components.
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Bioverativ, a Sanofi company: lead

Study Sites (5)

Investigational Site Number : 2760001

Essen, 45147, Germany

Location

Investigational Site Number : 3800001

Milan, Milano, 20122, Italy

Location

Investigational Site Number : 5280001

Amsterdam, 1081 HV, Netherlands

Location

Investigational Site Number : 5780001

Bergen, 5021, Norway

Location

Investigational Site Number : 8260001

London, London, City of, NW1 2PG, United Kingdom

Location

Related Links

• LTS16637 Plain Language Results Summary

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2021
• First Posted: March 17, 2021
• Study Start: March 22, 2021
• Primary Completion: March 4, 2025
• Study Completion: March 4, 2025
• Last Updated: September 11, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

DE (1); NL (1); GB (1); IT (1); NO (1)