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

A Phase 2, Multicenter, Open-Label Study of Zolacabtagene Autoleucel (BMS-986353), CD19-Targeted NEX-T CAR T Cells, in Participants With Chronic Immune Thrombocytopenia (cITP) and Autoimmune Hemolytic Anemia (AIHA)

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Autoimmune Hemolytic Anemia; Chronic Immune Thrombocytopenia
• Intervention / Treatment: Drug: Cyclophosphamide; Biological: BMS-986353; Drug: Fludarabine Phosphate

• Study Type: Interventional
• Enrollment (Estimated): 52
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: First line of the email MUST contain NCT # and Site #.
• Study Contact Backup: Name: BMS Clinical Trials Contact Center www.BMSClinicalTrials.com; Phone Number: 855-907-3286; Email: Clinical.Trials@bms.com

Study Locations

• Denmark
  • Odense, Denmark, DK-5000
    • Local Institution - 201
    • Contact: Site 201
• Germany
  • Erlangen, Germany, 91054
    • Local Institution - 302
    • Contact: Site 302
  • Saxony-Anhalt
    • Magdeburg, Saxony-Anhalt, Germany, 39120
      • Local Institution - 301
      • Contact: Site 301
• United Kingdom
  • Sheffield, United Kingdom, S10 2SJ
    • Local Institution - 402
    • Contact: Site 402
  • Greater London
    • London, Greater London, United Kingdom, W12 OHS
      • Local Institution - 401
      • Contact: Site 401
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Local Institution - 101
      • Contact: Site 101
  • Texas
    • Houston, Texas, United States, 77030-2740
      • Local Institution - 103
      • Contact: Site 103
  • Washington
    • Seattle, Washington, United States, 98109
      • Local Institution - 102
      • Contact: Site 102

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 2	Drug: Cyclophosphamide; Specified dose on specified days; Biological: BMS-986353; Specified dose of specified days; Other Names: CC-97540; Zola-cel; Zolacabtagene autoleucel; Drug: Fludarabine Phosphate; Specified dose of specified days
Experimental: Cohort 1 Part A ITP	Drug: Cyclophosphamide; Specified dose on specified days; Biological: BMS-986353; Specified dose of specified days; Other Names: CC-97540; Zola-cel; Zolacabtagene autoleucel; Drug: Fludarabine Phosphate; Specified dose of specified days
Experimental: Cohort 1 Part A AIHA	Drug: Cyclophosphamide; Specified dose on specified days; Biological: BMS-986353; Specified dose of specified days; Other Names: CC-97540; Zola-cel; Zolacabtagene autoleucel; Drug: Fludarabine Phosphate; Specified dose of specified days
Experimental: Cohort 1 Part B	Drug: Cyclophosphamide; Specified dose on specified days; Biological: BMS-986353; Specified dose of specified days; Other Names: CC-97540; Zola-cel; Zolacabtagene autoleucel; Drug: Fludarabine Phosphate; Specified dose of specified days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Cohort 1 Part A: Number of participants with treatment-emergent adverse events (TEAEs)	Up to approximately Month 36
Cohort 1 Part A: Number of participants with serious AEs (SAEs)	Up to approximately Month 36
Cohort 1 Part A: Number of participants with AEs of special interest (AESI)	Up to approximately Month 36
Cohort 1 Part A: Number of participants with clinically significant laboratory abnormalities	Up to approximately Month 36
Cohort 1 Part B: Hematologic Complete Response (CR)	Up to approximately Month 6

Secondary Outcome Measures

Outcome Measure	Time Frame
Cohort 1 PART B: Hematologic Overall Response (OR)	Up to approximately Month 6
Cohort 1 PART A and Cohort 2: Hematologic CR and OR	Up to approximately Month 6
Cohort 1 PART B and Cohort 2: Number of participants with TEAEs	Up to approximately Month 36
Cohort 1 PART B and Cohort 2: Number of participants with SAEs	Up to approximately Month 36
Cohort 1 PART B and Cohort 2: Number of participants with AESIs	Up to approximately Month 36
Cohort 1 PART B and Cohort 2: Number of participants with clinically significant laboratory abnormalities	Up to approximately Month 36
Number of participants with Hematologic PR	Up to approximately Month 6
Number of participants with Hematologic CR	Up to approximately Month 36
Number of participants with Hematologic PR	Up to approximately Month 36
Number of participants with Hematologic OR	Up to approximately Month 36
Number of participants with Best Overall Response (BOR)	Up to approximately Month 36
Number of participants with durable CR, PR and OR	Up to approximately 12 months from Zola-cel infusion
Time to First Response (TTR)	Up to approximately Month 36
Time to First Complete Response (TTCR)	Up to approximately Month 36
Duration of response (DOR)	Up to approximately Month 36
Treatment-free Remission (TFR)	Up to approximately Month 36
Proportion of participants who requires rescue therapy for ITP or AIHA	Up to approximately Month 36
Time to first administration of rescue therapy for ITP or AIHA	Up to approximately Month 36
Proportion of AIHA participants who experience hemolysis features	Up to approximately Month 36
Number of AIHA participants with cold agglutinin disease (CAD) who experience acrocyanosis	Up to approximately Month 36
Change from baseline in hemolysis indicators in AIHA participants	Up to approximately Month 36
Proportion of ITP participants with WHO-classified bleeding events as assessed by WHO bleeding scale	Up to approximately Month 36
Change from baseline in 36-Item Short Form Health Questionnaire version 2 (SF-36 v2)	Up to approximately Month 36
Change from baseline in Patient Global Impression of Severity (PGI-S) Fatigue score	Up to approximately Month 36
Patient Global Impression of Change (PGI-C) Fatigue mean score	Up to approximately Month 36
Change from baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP - PAQ) score	Up to approximately Month 36
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score	Up to approximately Month 36

Collaborators and Investigators

• Sponsor: Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

Study record dates

Study Major Dates

• Study Start (Estimated): June 15, 2026
• Primary Completion (Estimated): May 6, 2030
• Study Completion (Estimated): May 6, 2030

Study Registration Dates

• First Submitted: May 18, 2026
• First Submitted That Met QC Criteria: May 18, 2026
• First Posted (Actual): May 22, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 18, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Autoimmune Diseases; Immune System Diseases; Hematologic Diseases; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Anemia, Hemolytic, Autoimmune; Organic Chemicals; Hydrocarbons; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Cyclophosphamide; fludarabine phosphate
• Other Study ID Numbers: CA061-1040

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
• IPD Sharing Time Frame: See Plan Description
• IPD Sharing Access Criteria: See Plan Description
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No