A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease

NCT04269551 | Completed Phase 1 FDA Drug

• 3 other identifiers: PDY163702019-001844-22U1111-1233-0831
• Study Type: interventional
• Target: 12
• Locations: 6 countries
• Sites: 10

Brief Summary

Primary Objective: To assess the safety and tolerability in participants with cold agglutinin disease (CAD), after a single dose of intravenous (IV) BIVV020 Secondary Objectives: To assess, in participants with cold agglutinin disease, after a single dose of intravenous (IV) BIVV020:

• The effect of BIVV020 on complement mediated hemolysis
• The pharmacodynamics (PD) of BIVV020 relating to complement inhibition
• The pharmacokinetics (PK) of BIVV020
• The immunogenicity of BIVV020

Conditions

Autoimmune Haemolytic Anaemia

Outcome Measures

Primary Outcomes (1)

• Assessment of adverse events (AEs)

  Number of participants with adverse events (AEs)

  Screening to Day 106

Secondary Outcomes (11)

• Mean change from baseline in bilirubin over time

  Day 1 to Day 106

• Mean change from baseline in hemoglobin over time

  Day 1 to Day 106

• Complement System Classical Pathway Levels as Measured by WIESLAB Assay

  Day 1 to Day 106

• Complement System Alternative Pathway Levels as Measured by WIESLAB Assay

  Day 1 to Day 106

• Total Complement (CH50) Levels

  Day 1 to Day 106

Study Arms (1)

BIVV020 IV

EXPERIMENTAL

Single administration dose 1, plus two optional doses of BIVV020 administered intravenously.

Drug: BIVV020

Interventions

BIVV020 DRUG

Pharmaceutical form:solution for injection Route of administration: intravenous

BIVV020 IV

Eligibility Criteria

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

You may qualify if:

• Male and/or female patients, ≥ 18 years of age with cold agglutinin disease as defined by:
• Chronic hemolysis per Investigator's judgement,
• Polyspecific direct antiglobulin test (DAT) positive,
• Monospecific DAT strongly positive for C3d,
• Cold agglutinin (CAg) titer ≥ 64 at 4 C; and,
• IgG DAT ≤1+.
• A hemoglobin level ≤11 mg/dL.
• A total bilirubin level above the normal reference range that is thought to be due to hemolysis.
• Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B meningococcus and Streptococcus pneumoniae) within five years of screening or willing to complete protocol specified vaccinations.
• Having given written informed consent prior to undertaking any study-related procedure.

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 of any kind within one month preceding screening.
• Treatment with anti-CD20 monotherapy within three months or anti CD20 combination therapies within six 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 within three months prior to screening.
• Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within three months prior to screening.
• If female, pregnant or lactating.
• 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 (10)

Investigational Site Number :8400002

Fayetteville, Georgia, 30214, United States

Location

Investigational Site Number :8400009

St Louis, Missouri, 63110, United States

Location

Investigational Site Number :8400006

The Bronx, New York, 10467, United States

Location

Investigational Site Number :8400008

Pittsburgh, Pennsylvania, 15232, United States

Location

Investigational Site Number :8400004

Seattle, Washington, 98108, United States

Location

Investigational Site Number :2760001

Essen, 45147, Germany

Location

Investigational Site Number :3800001

Milan, Milano, 20122, Italy

Location

Investigational Site Number :5280001

Amsterdam, 1105AZ, Netherlands

Location

Investigational Site Number :5780001

Bergen, 5021, Norway

Location

Investigational Site Number :8260001

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

Location

Related Publications (1)

• D'Sa S, Vos JMI, Barcellini W, Wardecki M, Perrin L, Barker G, Zilberstein M, Storek M, Chow T, Roth A. Safety, tolerability, and activity of the active C1s antibody riliprubart in cold agglutinin disease: a phase 1b study. Blood. 2024 Feb 22;143(8):713-720. doi: 10.1182/blood.2023022153.

  PMID: 38085846 DERIVED

Related Links

• PDY16370 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: January 29, 2020
• First Posted: February 17, 2020
• Study Start: June 15, 2020
• Primary Completion: January 6, 2022
• Study Completion: January 6, 2022
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

NL (1); DE (1); US (5); NO (1); GB (1); IT (1)