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

Bioverativ, a Sanofi company12 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Immune Thrombocytopenia (ITP)

Interventions

SAR445088 (BIVV020)DRUG

Pharmaceutical form:solution for injection

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (9)

Investigational Site Number :8400001

Washington D.C., District of Columbia, United States

Investigational Site Number :8400002

Tamarac, Florida, United States

Investigational Site Number :2030002

Ostrava - Poruba, Czechia

Outcomes

Primary Outcomes

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.

Time frame: From Week 3 to Week 24

Secondary Outcomes

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).

Time frame: 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.

Data from ClinicalTrials.gov

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