A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)

Study Overview
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Brief Summary
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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
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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

Official Title
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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)

Conditions
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Immune Thrombocytopenia (ITP)

Intervention / Treatment
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* Drug: BIVV020


Participation Criteria
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Eligibility Criteria
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Inclusion criteria : 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 If applicable, concurrent administration of ITP medications (eg. corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or thrombopoietin receptor agonists) is acceptable provided the patient 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 are sexually active with a female partner of childbearing potential Exclusion criteria: Participants are 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 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 patient's potential participation in a clinical trial.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BIVV0020<br>All eligible participants will receive BIVV020 for at least 52 weeks. | Drug: BIVV020<br>* Pharmaceutical form:solution for injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants with a durable platelet response | Durable platelet response is defined for naïve participants as the proportion of participants with a platelet count ≥50 × 109/L at ≥50% of scheduled visits, or for participants with baseline platelet count <15 × 109/L, a ≥20 × 109/L increase in platelet count from baseline at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24. Durable platelet response is defined for participants who previously received sutimlimab as maintenance of platelet count ≥30 × 109/L at ≥50% of scheduled visits, without receiving rescue ITP therapy, as assessed from Week 3 to Week 24. | Week 3 to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with treatment emergent adverse events |  | Week 52 | 
| Plasma concentrations of BIVV020 |  | Week 52 | 
| Response rate of treatment with BIVV020 | Response rate at Weeks 24 and 52, defined as a platelet count ≥50 × 109/L and a greater than 2-fold increase from baseline, measured on 2 occasions at least 7 days apart, with the absence of bleeding (bleeding is defined as bleeding with a score ≥2 on the WHO bleeding scale), and the lack of combination ITP therapy during this period. | Weeks 24 and 52 | 
| Time to first platelet response | Time from baseline to first platelet response, defined as greater than or equal to each of the following values: 50 × 109/L and 100 × 109/L (confirmed by 2 measurements at least 7 days apart) | Baseline to Week 52 | 
| Proportion of patients who did not require rescue therapy for an acute episode of thrombocytopenia after Week 3 |  | From Week 3 to Week 52 | 
| Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies |  | Week 24, Week 52 |