immune thrombocytopenia ITP
Conditions
Interventions
An intravenous (IV) loading dose of BIVV020 at 50 mg/kg will be administered on
Day 1, and
will be followed by maintenance doses of 600 mg SC weekly starting on Day 8.
Sponsors
Genzyme Europe BV
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
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: a) Platelet count =30 × 10^9/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; c) 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; d) 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; e) 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Naïve participants: Proportion of participants with a platelet count >=50 × 109/L at >=50% of scheduled visits, or for participants with baseline platelet count =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. • Participants who previously received sutimlimab: Proportion of participants with 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Standard clinical and laboratory parameters and adverse events • Plasma concentrations of 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. • 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) • Proportion of participants who did not require rescue therapy for an acute episode of thrombocytopenia after Week 3 • Incidence and titer (if relevant) of anti-BIVV020 antibodies | — |
Countries
Netherlands
Outcome results
None listed