Autoimmune Haemolytic AnaemiaPhase 1NCT04802057
Safety and Tolerability Study in Adults With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088
A Phase 1 study testing SAR445088 in people with Autoimmune Haemolytic Anaemia, run by Bioverativ, a Sanofi company at 5 sites in 5 countries.
Trial 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… 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?
- SAR445088
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 22 Mar 2021to 4 Mar 2025
- 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 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:
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- 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.
- 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 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.
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- 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.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
Netherlands1 site
Investigational Site Number : 5280001
Amsterdam, 1081 HV
United Kingdom1 site
Investigational Site Number : 8260001
London, London, City of, NW1 2PG
About this study
Official title: An Open-label, Long-term, Safety and Tolerability Study of SAR445088 in Participants With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088
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
Read the detailed description
The screening period for this study is up to 6 weeks. The treatment period for this study will continue for 2 years after last participant entered Part 2 (either having switched from Part 1 or as a SAR445088-naïve participant), or until SAR445088 development is discontinued, whichever comes first.
- Conditions
- Autoimmune Haemolytic Anaemia
- Being tested
- SAR445088
- 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.
SAR445088Drug
Pharmaceutical form: solution for injection
Route of administration: IV and SC (Part 1) IV (Part 2)
| Study group | Role | What they receive |
|---|---|---|
| SAR445088 | Experimental | Repeat dose of SAR445088 Receives: Drug: SAR445088 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events (TEAE)
The number of participants experiencing TEAEs will be reported for the overall safety population.
Measured: 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
Assessment of total bilirubin
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in hemoglobin over time
Assessment of hemoglobin
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in lactate dehydrogenase over time
Assessment of lactate dehydrogenase
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in reticulocyte count over time
Assessment of reticulocyte count
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
Complement System Classical Pathway Levels as Measured by WIESLAB Assay
Inhibition by SAR445088 of the complement system classical pathway measured by the WIESLAB assay.
Measured: Day 1 or Day 1-IV to Week 48-IV
Complement System Alternative Pathway Levels as Measured by WIESLAB Assay
Part 1: Effect of SAR445088 on the complement system alternative pathway measured by the WIESLAB assay.
Measured: Day 1 to Week 24
Mean change in CH50 over time
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 or Day 1-IV to end of study, approximately 6 years
Total Complement Factor C4 Levels
Total C4 Levels will be assessed in plasma using complement assays
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
PK parameter: Cmax
Maximum observed concentration (Cmax) of SAR445088 in plasma will be assessed
Measured: Day 1 or Day 1-IV to Week 48-IV
PK Parameter: AUC
Area under the plasma concentration versus time curve calculated using the trapezoidal method at steady state
Measured: Day 1 or Day 1-IV to Week 48-IV
Number of participants with anti-SAR445088 antibodies
The immunogenicity for SAR445088 will be assessed by summarizing the number of participants with anti-SAR445088 antibodies (ADA)
Measured: Day 1 or Day 1-IV to end of study, approximately 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2021
- Study start22 Mar 2021
- Primary completion4 Mar 2025
- Study completion4 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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
- 9 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
- NCT04802057
- Sponsor's protocol ID
- LTS16637
- Other IDs
- U1111-1244-0808, 2023-510210-68, 2019-004423-21, 2019, 2023
- Status last verified
- Sep 2025
- First submitted
- 15 Mar 2021
- Last updated
- 11 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 40308078
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Long-term, Safety and Tolerability Study of SAR445088 in Participants With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088
- ClinicalTrials.gov ID
- NCT04802057
- Acronym / sponsor ID
- LTS16637
- Conditions
- Autoimmune Haemolytic Anaemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 22 Mar 2021 (actual)
- Primary completion
- 4 Mar 2025 (actual)
- Study completion
- 4 Mar 2025 (actual)
- First posted
- 17 Mar 2021 (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 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
Detailed description
The screening period for this study is up to 6 weeks. The treatment period for this study will continue for 2 years after last participant entered Part 2 (either having switched from Part 1 or as a SAR445088-naïve participant), or until SAR445088 development is discontinued, whichever comes first.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SAR445088 | Experimental | Drug: SAR445088Repeat dose of SAR445088 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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:
- OR were never treated with SAR445088 before entering Part 2, and met the below criteria to establish CAD diagnosis:
- 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.
- Having given written informed consent prior to undertaking any study-related procedure.
Key exclusion criteria (9 listed)
- 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.
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 |
|---|---|---|
| SAR445088Pharmaceutical form: solution for injection Route of administration: IV and SC (Part 1) IV (Part 2) | Drug | SAR445088 |
Primary outcome
Number of participants with treatment-emergent adverse events (TEAE)
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
The number of participants experiencing TEAEs will be reported for the overall safety population.
11 secondary outcomes
Mean change from baseline in total bilirubin over time
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in hemoglobin over time
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in lactate dehydrogenase over time
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Mean change from baseline in reticulocyte count over time
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Complement System Classical Pathway Levels as Measured by WIESLAB Assay
Time frame: Day 1 or Day 1-IV to Week 48-IV
Complement System Alternative Pathway Levels as Measured by WIESLAB Assay
Time frame: Day 1 to Week 24
Mean change in CH50 over time
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Total Complement Factor C4 Levels
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
PK parameter: Cmax
Time frame: Day 1 or Day 1-IV to Week 48-IV
PK Parameter: AUC
Time frame: Day 1 or Day 1-IV to Week 48-IV
Number of participants with anti-SAR445088 antibodies
Time frame: Day 1 or Day 1-IV to end of study, approximately 6 years
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 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.
- LTS16637 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
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SAR445088 in people with Autoimmune Haemolytic Anaemia, run by Bioverativ, a Sanofi company at 5 sites in 5 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 16 inclusion and 9 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: stopped early, as of the sponsor's last update on 11 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 5 sites in Germany, Italy, Netherlands, Norway, United Kingdom. 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 was stopped early. Results are not available in the source record.
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?
Number of participants with treatment-emergent adverse events (TEAE) (time frame: Day 1 or Day 1-IV to end of study, approximately 6 years). 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 NCT04802057, which also lists 1 linked publication.
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 11 Sep 2025.