Immune Thrombocytopenia (ITP)Phase 2NCT04669600
A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
A Phase 2 study testing SAR445088 (BIVV020) in people with Immune Thrombocytopenia (ITP), run by Bioverativ, a Sanofi company at 9 sites in 6 countries.
Trial summary
Primary Objective: To evaluate the effect of BIVV020 on the durability of platelet response in participants with persistent/chronic immune thrombocytopenia (ITP) Secondary Objectives: To assess the safety and tolerability of BIVV020 To assess the pharmacokinetics of BIVV020 To assess the response rate of treatment… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia (ITP)
- What is being tested?
- SAR445088 (BIVV020)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 4 Feb 2021to 7 Feb 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 participants ≥18 years of age at the time of signing the informed consent
- Confirmed diagnosis of primary ITP; for participants who previously received sutimlimab in study TDR16218 (NCT03275454), a response to sutimlimab must have been obtained, as defined by platelet count ≥30 × 10^9/L on 2 visits at least 7 days apart
- For participants who have not previously received sutimlimab: persistent/chronic ITP (ITP lasting for ≥6 months) and all the following conditions:
- Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during the Screening Period;
- Lack of an adequate platelet count response (as defined by maintenance of sustained platelet count ≥30 × 109/L in the absence of bleeding) to at least 2 ITP treatments, 1 of which was a thrombopoietin receptor agonist. Other ITP treatments include: IVIg, anti-D immunoglobulin, corticosteroids, splenectomy, rituximab, cyclophosphamide, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or fostamatinib.
- If receiving weekly thrombopoietin receptor agonist dosing, the last dose must have been administered ≥7 days before the first dose of BIVV020. If receiving daily thrombopoietin receptor agonist dosing, the last dose must have been administered ≥24 hours before the first dose of BIVV020
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- If applicable, concurrent administration of ITP medications (eg. corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or thrombopoietin receptor agonists) is acceptable provided the participant has been on a stable dose for at least 1 month.
- If previously dosed with rituximab, the last dose of rituximab must have been administered at least 12 weeks before the first dose of BIVV020
- Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B where available, Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years of enrollment
- Contraceptive use for women of childbearing potential and men who were sexually active with a female partner of childbearing potential
Who cannot join
- Participants were excluded from the study if any of the following criteria apply:
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his/her participation in the study
- Clinical diagnosis of SLE
- Clinically relevant infection within the month prior to enrollment
- History of venous or arterial thrombosis within the year prior to enrollment
- Secondary ITP from any cause including lymphoma, chronic lymphocytic leukemia, and drug-induced thrombocytopenia
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- Positive hepatitis B surface antigen (HBsAg) or active HCV infection
- HIV infection
- Pregnant or lactating women
- Hemoglobin level <10 g/dL
- The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
9 sites in 6 countries.
9 sites in 6 countries
Spain3 sites
Investigational Site Number :7240002
A Coruña, A Coruña [La Coruña], 15006
Investigational Site Number :7240001
Palma de Mallorca, 07120
Investigational Site Number :7240003
Seville, 41013
United States2 sites
Investigational Site Number :8400002
Tamarac, Florida, 33321
Investigational Site Number :8400001
Washington D.C., District of Columbia, 20007
United Kingdom1 site
Investigational Site Number :8260002
London, London, City of, W12 0HS
About this study
Official title: A Multicenter, Phase 2a, Open-label, Non-randomized Study Evaluating the Efficacy, Safety, and Tolerability of BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Primary Objective:
- To evaluate the effect of BIVV020 on the durability of platelet response in participants with persistent/chronic immune thrombocytopenia (ITP)
Secondary Objectives:
- To assess the safety and tolerability of BIVV020
- To assess the pharmacokinetics of BIVV020
- To assess the response rate of treatment with BIVV020
- To assess the time to response
- To assess the effect of treatment with BIVV020 on the requirement for rescue ITP therapy
- To assess the immunogenicity of BIVV020
Read the detailed description
Study duration:
- Screening period: up to 56 days
- Transition period between last sutimlimab dose and first dose of BIVV020 (for participants who were previously receiving sutimlimab): 14 days, included as part of the 56-day Screening period. Treatment duration: Minimum 52 weeks.
Visit frequency:
- Day 1
- Day 4
- Weeks 1 to 6: Weekly
- Weeks 7 to 12: Every other week
- Weeks 13 to 24: Every 4 weeks
- Weeks 25+: At least every 8 weeks
- End of Study visit: 22 weeks after the last dose of BIVV020
- Conditions
- Immune Thrombocytopenia (ITP)
- Being tested
- SAR445088 (BIVV020)
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR445088 (BIVV020)Drug
Pharmaceutical form:solution for injection
| Study group | Role | What they receive |
|---|---|---|
| SAR445088 | Experimental | Participants received SAR445088 (BIVV020). Receives: Drug: SAR445088 (BIVV020) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With a Durable Platelet Response
A naive participant was a participant who did not use sutimlimab prior to enrollment. A switcher was a participant who used sutimlimab prior to enrollment. A naive participant was a responder if the platelet count was >=50 × 10^9/liter (L) at >=50 percent (%) of scheduled visits, or for participants with baseline platelet count <15 × 10^9/L, a >=20 × 10^9/L increase in platelet count from baseline at >=50% of scheduled visits, without receiving rescue immune thrombocytopenia (ITP) therapy. A switcher was a responder if the maintenance platelet count was >=30 × 10^9/L at >=50% of scheduled visits, without receiving rescue ITP therapy.
Measured: From Week 3 to Week 24
Secondary outcomes (10)
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAE)
An AE was defined as any untoward medical occurrence in a participant temporally associated with the use of study treatment, whether or not considered related to the study treatment. SAEs were any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs were AEs that developed after the first study treatment administration in the safety analysis period which was defined as the period from the first study intervention administration to the end of study (EOS) visit (up to Week 103).
Measured: From first study treatment administration (Day 1) up to Week 103
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Criteria for potentially clinically significant laboratory abnormalities (PCSA): White blood cells: less than (<)3.0 Giga/L (Non-Black [NB]) or <2.0 Giga/L (Black [B]), greater than or equal to (>=)16.0 Giga/L; Lymphocytes: greater than (>)4.0 Giga/L; Neutrophils: <1.5 Giga/L (NB) or <1.0 Giga/L (B); Monocytes: >0.7 Giga/L; Basophils: >0.1 Giga/L; Eosinophils: >0.5 Giga/L or >upper limit of normal (ULN) (if ULN >=0.5 Giga/L); Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L) (Male[M]) or <=95 g/L (Female[F]), >=185 g/L (M) or >=165 g/L (F), Decrease from baseline (DFB) >=20 g/L; Hematocrit: <=0.37 volume/volume (v/v) (M) or <=0.32 v/v (F); Red blood cells (RBC): >=6 Tera/L; Platelets:<100 Giga/L, >=700 Giga/L. Only the worst case during the treatment-emergent (TE) period for each participant with worsening from baseline is presented.
Measured: On Days 1, 15, 29, at Weeks 8, 12, 24, and then every 8 weeks until the end of study (EOS) (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Criteria for PCSA: Blood Urea Nitrogen: >=17 millimole (mmol)/L; Creatinine: >=150 micromole (mcmol)/L (Adults), >=30% and <100% change from baseline, >=100% change from baseline; Potassium: <3 mmol/L, >=5.5 mmol/L; Sodium: <=129 mmol/L, >=160 mmol/L; Aspartate Aminotransferase (AST): >3 ULN, >5 ULN, >10 ULN, >20 ULN; Alanine Aminotransferase (ALT): >3 ULN, >5 ULN, >10 ULN, >20 ULN; Alkaline Phosphatase (ALP): >1.5 ULN; Bilirubin: >1.5 ULN, >2 ULN; ALT and Total Bilirubin: ALT >3 ULN and TBILI >2 ULN. Only the worst case during the TE period for each participant with worsening from baseline is presented.
Measured: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
The number of participants with PCSA for coagulation parameters during the TE period without PCSA definition by biological function are presented. The parameters evaluated were prothrombin time, prothrombin international normalized ratio and activated partial thromboplastin time (APTT).
Measured: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
Criteria for PCSA: potential of Hydrogen (pH) <=4.6, >=8. Only the worst case during the TE period for each participant with worsening from baseline is presented.
Measured: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Plasma Concentrations of SAR445088 (BIVV020)
Plasma samples were collected at specified timepoints.
Measured: 1-hour post-dose on Day 1, on Days 8, 15, 29, 43, at Weeks 12, 16, 24, 32, 40, 48, 56, 64, 72, 80 and EOS visit, up to 103 weeks
Number of Responders to SAR445088 (BIVV020)
A participant was a responder if the platelet count was >=50 × 10^9/L and there was a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart with the absence of bleeding [bleeding score >=2 on the World Health Organization (WHO) bleeding scale] while the platelet counts were maintained above the threshold and lack of combination ITP therapy during this period. WHO bleeding scores: 1=Petechiae; 2=Mild blood loss; 3=Gross blood loss; and 4=Debilitating blood loss.
Measured: At Weeks 24 and 56
Time to First Platelet Response
Time to first platelet response was defined as greater than or equal to each of the following values: 50 × 10^9/L or 100 × 10^9/L (confirmed by 2 measurements at least 7 days apart). It was calculated as date of first occurrence of confirmed platelet count response before rescue therapy.
Measured: From Baseline (Day 1) up to Week 56
Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
Data was collected to assess the effect of treatment with SAR445088 (BIVV020) on the requirement for rescue ITP therapy.
Measured: Up to Week 84
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Plasma samples were analyzed for the presence of ADAs for SAR445088 (BIVV020) using validated assays. Treatment-induced ADA was defined as ADAs that developed during the treatment-emergent period and without pre-existing ADA. Pre-existing ADA was defined as ADAs present in samples drawn before first study treatment administration. Treatment-boosted ADA positive was defined as pre-existing ADA (i.e., ADA positive at baseline) that was boosted at least a 9-fold increase of titer values during the treatment-emergent period than the baseline. Treatment-emergent ADA positive was defined as either treatment-induced ADA positive or treatment-boosted ADA positive during the treatment-emergent period. Inconclusive ADA was defined as the one that could not irrefutably be classified as with or without treatment-emergent ADA.
Measured: Up to Week 103
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2020
- Study start4 Feb 2021
- Primary completion15 Feb 2022
- Study completion7 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune Thrombocytopenia (ITP)Studies 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 2
- 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
- NCT04669600
- Sponsor's protocol ID
- PDY16894
- Other IDs
- 2020-004162-18, U1111-1253-2343
- Status last verified
- Sep 2025
- First submitted
- 9 Dec 2020
- Last updated
- 10 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- 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
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 2a, Open-label, Non-randomized Study Evaluating the Efficacy, Safety, and Tolerability of BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT04669600
- Acronym / sponsor ID
- PDY16894
- Conditions
- Immune Thrombocytopenia (ITP)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Bioverativ, a Sanofi companyIndustry (pharma, biotech, devices)
- Study start
- 4 Feb 2021 (actual)
- Primary completion
- 15 Feb 2022 (actual)
- Study completion
- 7 Feb 2023 (actual)
- First posted
- 17 Dec 2020 (actual)
- Results
- Posted 22 Feb 2024
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 evaluate the effect of BIVV020 on the durability of platelet response in participants with persistent/chronic immune thrombocytopenia (ITP)
Secondary Objectives:
- To assess the safety and tolerability of BIVV020
- To assess the pharmacokinetics of BIVV020
- To assess the response rate of treatment with BIVV020
- To assess the time to response
- To assess the effect of treatment with BIVV020 on the requirement for rescue ITP therapy
- To assess the immunogenicity of BIVV020
Detailed description
Study duration:
- Screening period: up to 56 days
- Transition period between last sutimlimab dose and first dose of BIVV020 (for participants who were previously receiving sutimlimab): 14 days, included as part of the 56-day Screening period. Treatment duration: Minimum 52 weeks.
Visit frequency:
- Day 1
- Day 4
- Weeks 1 to 6: Weekly
- Weeks 7 to 12: Every other week
- Weeks 13 to 24: Every 4 weeks
- Weeks 25+: At least every 8 weeks
- End of Study visit: 22 weeks after the last dose of BIVV020
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: SAR445088 (BIVV020)Participants received SAR445088 (BIVV020). |
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 female participants ≥18 years of age at the time of signing the informed consent
- Confirmed diagnosis of primary ITP; for participants who previously received sutimlimab in study TDR16218 (NCT03275454), a response to sutimlimab must have been obtained, as defined by platelet count ≥30 × 10^9/L on 2 visits at least 7 days apart
- For participants who have not previously received sutimlimab: persistent/chronic ITP (ITP lasting for ≥6 months) and all the following conditions:
- Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B where available, Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years of enrollment
- Contraceptive use for women of childbearing potential and men who were sexually active with a female partner of childbearing potential
Key exclusion criteria (11 listed)
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his/her participation in the study
- Clinical diagnosis of SLE
- Clinically relevant infection within the month prior to enrollment
- History of venous or arterial thrombosis within the year prior to enrollment
- Secondary ITP from any cause including lymphoma, chronic lymphocytic leukemia, and drug-induced thrombocytopenia
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 |
|---|---|---|
| SAR445088 (BIVV020)Pharmaceutical form:solution for injection | Drug | SAR445088 |
Primary outcome
Percentage of Participants With a Durable Platelet Response
Time frame: From Week 3 to Week 24
A naive participant was a participant who did not use sutimlimab prior to enrollment. A switcher was a participant who used sutimlimab prior to enrollment. A naive participant was a responder if the platelet count was >=50 × 10^9/liter (L) at >=50 percent (%) of scheduled visits, or for participants with baseline platelet count <15 × 10^9/L, a >=20 × 10^9/L increase in platelet count from baseline at >=50% of scheduled visits, without receiving rescue immune thrombocytopenia (ITP) therapy. A switcher was a responder if the maintenance platelet count was >=30 × 10^9/L at >=50% of scheduled visits, without receiving rescue ITP therapy.
10 secondary outcomes
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (SAE)
Time frame: From first study treatment administration (Day 1) up to Week 103
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Time frame: On Days 1, 15, 29, at Weeks 8, 12, 24, and then every 8 weeks until the end of study (EOS) (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Clinical Chemistry
Time frame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Coagulation
Time frame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
Time frame: On Days 1, 15, 29, Weeks 8, 12 and 24, then every 8 weeks until the EOS (Week 103)
Plasma Concentrations of SAR445088 (BIVV020)
Time frame: 1-hour post-dose on Day 1, on Days 8, 15, 29, 43, at Weeks 12, 16, 24, 32, 40, 48, 56, 64, 72, 80 and EOS visit, up to 103 weeks
Number of Responders to SAR445088 (BIVV020)
Time frame: At Weeks 24 and 56
Time to First Platelet Response
Time frame: From Baseline (Day 1) up to Week 56
Percentage of Participants Who Did Not Require Rescue Therapy for an Acute Episode of Thrombocytopenia After Week 3
Time frame: Up to Week 84
Number of Participants With Anti-Drug Antibody (ADAs) Response to SAR445088 (BIVV020)
Time frame: Up to Week 103
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- PDY16894 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
9 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SAR445088 (BIVV020) in people with Immune Thrombocytopenia (ITP), run by Bioverativ, a Sanofi company at 9 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 11 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 10 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 9 sites in Spain, United States, Czechia, Germany, Netherlands 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With a Durable Platelet Response (time frame: From Week 3 to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Sep 2025.