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

Phase 1

Completed

• Conditions: Autoimmune Haemolytic Anaemia
• Interventions: Drug: BIVV020

• Registration Number: NCT04269551
• Lead Sponsor: Bioverativ, a Sanofi company

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

Detailed Description

Up to 23 weeks (screening period up to 8 weeks, treatment period 15 weeks)

Study Timeline

• Study First Submitted: 2020-01-29
• Study First Submitted QC: 2020-02-12
• Study First Posted: 2020-02-17
• Study Start: 2020-06-15
• Primary Completion: 2022-01-06
• Study Completion: 2022-01-06
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-25
• Last Update Posted: 2022-05-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BIVV020 IV	BIVV020	Single administration dose 1, plus two optional doses of BIVV020 administered intravenously.

Primary Outcome Measures

Name	Time	Method
Assessment of adverse events (AEs)	Screening to Day 106	Number of participants with adverse events (AEs)

Secondary Outcome Measures

Name	Time	Method
Complement System Classical Pathway Levels as Measured by WIESLAB Assay	Day 1 to Day 106	Inhibition by BIVV020 of the complement system classical pathway measured by the WIESLAB assay
Complement System Alternative Pathway Levels as Measured by WIESLAB Assay	Day 1 to Day 106	Effect of BIVV0020 on the complement system alternative pathway measured by the WIESLAB assay
Total Complement (CH50) Levels	Day 1 to Day 106	Complement CH50 is a blood test that helps us determine whether protein abnormalities and deficiencies in the complement system are responsible for any increase in autoimmune activity. It will be assessed using complement assays.
PK parameter: Cmax	Day 1 to Day 106	Observed maximum plasma concentration
Mean change from baseline in hemoglobin over time	Day 1 to Day 106	Assessment of hemoglobin
Total Complement Factor C4 Levels	Day 1 to Day 106	Total C4 Levels will be assessed in plasma using complement assays
PK parameter: tmax	Day 1 to Day 106	Observed first time to reach Cmax
Mean change from baseline in bilirubin over time	Day 1 to Day 106	Assessment of total bilirubin
PK parameter: AUClast	Day 1 to Day 106	Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast
PK parameter: AUC0-∞	Day 1 to Day 106	Calculated area under the plasma concentration versus time curve extrapolated to infinity
Number of participants with anti-BIVV antibodies	Day 1 to Day 106	Observed number of participants with BIVV020 antibodies

Trial Locations

Locations (10)

Investigational Site Number :5280001; 🇳🇱 Amsterdam, Netherlands

Investigational Site Number :5780001; 🇳🇴 Bergen, Norway

Investigational Site Number :8400008; 🇺🇸 Pittsburgh, Pennsylvania, United States

Investigational Site Number :8400004; 🇺🇸 Seattle, Washington, United States

Investigational Site Number :8400002; 🇺🇸 Fayetteville, Georgia, United States

Investigational Site Number :8400009; 🇺🇸 Saint Louis, Missouri, United States

Investigational Site Number :3800001; 🇮🇹 Milano, Italy

Investigational Site Number :8400006; 🇺🇸 Bronx, New York, United States

Investigational Site Number :2760001; 🇩🇪 Essen, Germany

Investigational Site Number :8260001; 🇬🇧 London, London, City Of, United Kingdom