Study of Zola-cel (BMS-986353), in Participants With Autoimmune Cytopenia (Breakfree-AiCE)

Juno Therapeutics

Status and phase: Begins enrollment this month; Phase 2

Conditions: Chronic Immune Thrombocytopenia; Autoimmune Hemolytic Anemia

Treatments: Drug: Fludarabine Phosphate; Biological: BMS-986353; Drug: Cyclophosphamide

Study type: Interventional

Funder types: Industry

Identifiers

NCT07603557

CA061-1040

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and efficacy of Zola-cel (BMS-986353), in participants with chronic immune thrombocytopenia (cITP) and autoimmune hemolytic anemia (AIHA).

Enrollment: 52 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

Inclusion Criteria for ITP

• Documented clinical diagnosis of chronic ITP (cITP) without other clinical manifestations of systemic autoimmune disease.
• Has relapsed after or is intolerant to corticosteroids (with or without intravenous immunoglobulin (IVIG) or anti-Rh0(D) Ig) AND has failed, relapsed after, or is intolerant to therapies with ≥ 2 mechanisms of action, with at least one being immunosuppressive or immunomodulatory.

Platelet count < 30 × 109/L. For participants on thrombopoietin receptor agonist (TPO-RA): platelet count < 50 × 109/L.

Inclusion Criteria for AIHA

• Documented clinical diagnosis of AIHA (including warm autoimmune hemolytic anemia (wAIHA), cold agglutinin disease (CAD), or mixed AIHA) without other clinical manifestations of systemic autoimmune disease.

  o wAIHA and mixed warm and cold AIHA: Failed, relapsed after, or is intolerant to at least 2 prior lines of treatment with 2 mechanisms of action (not including corticosteroids or IVIG), one of which is an anti-CD20 monoclonal antibody unless there is a documented contraindication.

  o CAD (all of the following must apply): Failed, relapsed after, or is intolerant to at least 2 prior lines of treatment with 2 mechanisms of action, one of which is an anti-CD20 monoclonal antibody with or without chemotherapy unless there is a documented contraindication.

• Hb <10 g/dL without red blood cell transfusion, or transfusion dependent

• Documented hemolysis

Exclusion Criteria

Medical Conditions

• ITP or AIHA associated with: Evans syndrome, other systemic autoimmune disease or single organ autoimmune disease requiring systemic immunosuppressive therapy, hepatitis C virus, HIV, drug induced (eg, non-steroidal anti-inflammatory drug (NSAIDS), trimethoprim/sulfamethoxazole (TMP-SMX), anticonvulsants), surgical procedures, or hematologic malignancies.
• COVID-19 Vaccine-induced immune thrombotic thrombocytopenia
• Prior history of solid organ malignancies, unless the participant has been free of the disease for ≥ 2 years.

Laboratory Test Findings

• Peripheral blood ANC < 1.5 × 109/L or requiring G-CSF or GM-CSF support o ALT/AST: ITP: ALT/AST: > 3 × ULN AIHA: ALT > 3 ULN. AST up to 5 × ULN may be permitted. o Bilirubin: ITP: total bilirubin > 1.5 × ULN AIHA: direct bilirubin > 1.5 × ULN o International normalized ratio (INR) > 1.5 × ULN

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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52 participants in 4 patient groups

Cohort 1 Part A ITP

Experimental group

Treatment:

Drug: Cyclophosphamide

Biological: BMS-986353

Drug: Fludarabine Phosphate

Cohort 1 Part A AIHA

Experimental group

Treatment:

Drug: Cyclophosphamide

Biological: BMS-986353

Drug: Fludarabine Phosphate

Cohort 1 Part B

Experimental group

Treatment:

Drug: Cyclophosphamide

Biological: BMS-986353

Drug: Fludarabine Phosphate

Cohort 2

Experimental group

Treatment:

Drug: Cyclophosphamide

Biological: BMS-986353

Drug: Fludarabine Phosphate