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

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

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

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:

  1. Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during the Screening Period;
  2. 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.
  3. 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
  4. 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.
  5. 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

Exclusion criteria:

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
• 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.