Autoimmune Haemolytic AnaemiaPhase 1NCT04269551
A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease
A Phase 1 study testing BIVV020 in people with Autoimmune Haemolytic Anaemia, run by Bioverativ, a Sanofi company at 10 sites in 6 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Haemolytic Anaemia
- What is being tested?
- BIVV020
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 10 sitesin 6 countries
- Study dates
- 15 Jun 2020to 6 Jan 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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+.
Show all 10Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
10 sites in 6 countries.
10 sites in 6 countries
United States5 sites
Investigational Site Number :8400002
Fayetteville, Georgia, 30214
Investigational Site Number :8400008
Pittsburgh, Pennsylvania, 15232
Investigational Site Number :8400004
Seattle, Washington, 98108
Investigational Site Number :8400009
St Louis, Missouri, 63110
Investigational Site Number :8400006
The Bronx, New York, 10467
United Kingdom1 site
Investigational Site Number :8260001
London, London, City of, NW1 2PJ
About this study
Official title: A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease
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
Read the detailed description
Up to 23 weeks (screening period up to 8 weeks, treatment period 15 weeks)
- Conditions
- Autoimmune Haemolytic Anaemia
- Being tested
- BIVV020
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BIVV020Drug
Pharmaceutical form:solution for injection Route of administration: intravenous
| Study group | Role | What they receive |
|---|---|---|
| BIVV020 IV | Experimental | Single administration dose 1, plus two optional doses of BIVV020 administered intravenously. Receives: Drug: BIVV020 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of adverse events (AEs)
Number of participants with adverse events (AEs)
Measured: Screening to Day 106
Secondary outcomes (11)
Mean change from baseline in bilirubin over time
Assessment of total bilirubin
Measured: Day 1 to Day 106
Mean change from baseline in hemoglobin over time
Assessment of hemoglobin
Measured: Day 1 to Day 106
Complement System Classical Pathway Levels as Measured by WIESLAB Assay
Inhibition by BIVV020 of the complement system classical pathway measured by the WIESLAB assay
Measured: Day 1 to Day 106
Complement System Alternative Pathway Levels as Measured by WIESLAB Assay
Effect of BIVV0020 on the complement system alternative pathway measured by the WIESLAB assay
Measured: Day 1 to Day 106
Total Complement (CH50) Levels
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.
Measured: Day 1 to Day 106
Total Complement Factor C4 Levels
Total C4 Levels will be assessed in plasma using complement assays
Measured: Day 1 to Day 106
PK parameter: Cmax
Observed maximum plasma concentration
Measured: Day 1 to Day 106
PK parameter: tmax
Observed first time to reach Cmax
Measured: Day 1 to Day 106
PK parameter: AUClast
Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast
Measured: Day 1 to Day 106
PK parameter: AUC0-∞
Calculated area under the plasma concentration versus time curve extrapolated to infinity
Measured: Day 1 to Day 106
Number of participants with anti-BIVV antibodies
Observed number of participants with BIVV020 antibodies
Measured: Day 1 to Day 106
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start15 Jun 2020
- Primary completion6 Jan 2022
- Study completion6 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Haemolytic AnaemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioverativ, a Sanofi company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04269551
- Sponsor's protocol ID
- PDY16370
- Other IDs
- 2019-001844-22, U1111-1233-0831, 2019
- Status last verified
- Sep 2025
- First submitted
- 29 Jan 2020
- Last updated
- 22 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Chow T, Wardecki M, Storek M, Wong N. Confirmation of Fixed Quarterly Riliprubart Regimen in Patients with Cold Agglutinin Disease Using Population PK/PD and Exposure-Response Analyses. Clin Pharmacol Ther. 2025 Aug;118(2):449-458. doi: 10.1002/cpt.3692. Epub 2025 Apr 30.
PubMed 40308078D'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.
PubMed 38085846
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease
- ClinicalTrials.gov ID
- NCT04269551
- Acronym / sponsor ID
- PDY16370
- Conditions
- Autoimmune Haemolytic Anaemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2020 (actual)
- Primary completion
- 6 Jan 2022 (actual)
- Study completion
- 6 Jan 2022 (actual)
- First posted
- 17 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BIVV020 IV | Experimental | Drug: BIVV020Single administration dose 1, plus two optional doses of BIVV020 administered intravenously. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male and/or female patients, ≥ 18 years of age with cold agglutinin disease as defined by:
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BIVV020Pharmaceutical form:solution for injection Route of administration: intravenous | Drug | BIVV020 IV |
Primary outcome
Assessment of adverse events (AEs)
Time frame: Screening to Day 106
Number of participants with adverse events (AEs)
11 secondary outcomes
Mean change from baseline in bilirubin over time
Time frame: Day 1 to Day 106
Mean change from baseline in hemoglobin over time
Time frame: Day 1 to Day 106
Complement System Classical Pathway Levels as Measured by WIESLAB Assay
Time frame: Day 1 to Day 106
Complement System Alternative Pathway Levels as Measured by WIESLAB Assay
Time frame: Day 1 to Day 106
Total Complement (CH50) Levels
Time frame: Day 1 to Day 106
Total Complement Factor C4 Levels
Time frame: Day 1 to Day 106
PK parameter: Cmax
Time frame: Day 1 to Day 106
PK parameter: tmax
Time frame: Day 1 to Day 106
PK parameter: AUClast
Time frame: Day 1 to Day 106
PK parameter: AUC0-∞
Time frame: Day 1 to Day 106
Number of participants with anti-BIVV antibodies
Time frame: Day 1 to Day 106
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2025.
- Derived from this study: PubMed 40308078Chow T, Wardecki M, Storek M, Wong N. Confirmation of Fixed Quarterly Riliprubart Regimen in Patients with Cold Agglutinin Disease Using Population PK/PD and Exposure-Response Analyses. Clin Pharmacol Ther. 2025 Aug;118(2):449-458. doi: 10.1002/cpt.3692. Epub 2025 Apr 30.
- Derived from this study: PubMed 38085846D'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.
- PDY16370 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
10 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BIVV020 in people with Autoimmune Haemolytic Anaemia, run by Bioverativ, a Sanofi company at 10 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Germany, Italy, Netherlands, Norway and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of adverse events (AEs) (time frame: Screening to Day 106). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04269551, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2025.