Autoimmune Hemolytic Anemia, Chronic Immune Thrombocytopenia
Conditions
Brief summary
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).
Interventions
BIOLOGICALBMS-986353
Specified dose of specified days
DRUGFludarabine Phosphate
Specified dose of specified days
DRUGCyclophosphamide
Specified dose on specified days
Sponsors
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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/
Design outcomes
Primary
| 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
| 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 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 |
Countries
Denmark, Germany, United Kingdom, United States
Contacts
CONTACTBMS Clinical Trials Contact Center www.BMSClinicalTrials.com
CONTACTFirst line of the email MUST contain NCT # and Site #.
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed