Safety and Tolerability Study in Adults With Cold Agglutinin Disease Previously Treated With BIVV020

• Status: Recruiting
• Trial end date: 2023-05-01
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objective:

To assess the long-term safety and tolerability in patients with cold agglutinin disease (CAD), after multiple doses of BIVV020

Secondary Objective:

To assess, in patients with cold agglutinin disease (CAD), after multiple doses of BIVV020:

• The long-term effect of BIVV020 on complement mediated hemolysis

• The long-term pharmacodynamics (PD) effect of BIVV020 relating to complement inhibition

• The pharmacokinetic profile (PK) after repeated administration of BIVV020

• The long-term immunogenicity of BIVV020

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioverativ, a Sanofi company; Sanofi

Criteria

Inclusion criteria :

Male and female adult patients ≥18 years of age with CAD who:

• met the eligibility criteria of a previous study evaluating BIVV020;

• successfully enrolled and completed dosing in a previous study evaluating BIVV020;

• successfully completed end of study procedures in a previous study evaluating BIVV020; and

• per Investigator judgement, had a favorable benefit to risk profile after receiving BIVV020.

Documented vaccinations against encapsulated bacterial pathogens given within five years of enrollment and at least 14 days prior to dosing.

Having given written informed consent prior to undertaking any study-related procedure.

Exclusion criteria:

Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high grade hematologic malignancy, or known solid organ tumor.

Clinically relevant infection within one month of enrollment.

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 other than BIVV020 (eg, eculizumab) 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.

History of hypersensitivity to BIVV020 or any of its components.

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.