Zola-cel for Autoimmune Hemolytic Anemia

Not yet recruiting at 7 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

Must be taking: Immunosuppressives, Immunomodulatory, Anti-CD20

Must not be taking: NSAIDs, TMP-SMX, Anticonvulsants

Disqualifiers: Evans syndrome, Hepatitis C, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new treatment called Zola-cel (BMS-986353) for individuals with certain blood disorders. It focuses on assisting those with chronic immune thrombocytopenia (cITP) and autoimmune hemolytic anemia (AIHA), conditions where the immune system attacks blood cells. Participants suitable for this trial have AIHA or cITP and have not responded well to at least two previous treatments. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants should have failed or been intolerant to certain treatments, which might imply changes to your current medication regimen. It's best to discuss this with the trial coordinators.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that Zola-cel (BMS-986353) is likely safe for individuals with autoimmune conditions. Studies have examined patients with diseases like Systemic Sclerosis, which affects connective tissues, and found that the treatment is generally safe with manageable side effects. For instance, one study involved 9 patients with Systemic Sclerosis who received Zola-cel and tolerated it well.

This treatment is a type of CAR-T cell therapy, where cells are modified to better combat diseases. Various autoimmune studies have used this method, and it appears safe so far. Although the effects are still being studied, current evidence suggests that Zola-cel could be a promising option for those exploring new treatments for autoimmune hemolytic anemia. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autoimmune hemolytic anemia?

Unlike the standard treatments for autoimmune hemolytic anemia, which often include corticosteroids and immunosuppressants, Zola-cel (BMS-986353) offers a novel approach. Zola-cel is a cell-based therapy designed to precisely target and modulate the immune system, potentially reducing the need for broad immunosuppression. Researchers are excited about Zola-cel because it promises a more targeted mechanism of action, which could lead to fewer side effects and improved outcomes for patients who do not respond well to existing treatments.

What evidence suggests that Zola-cel might be an effective treatment for autoimmune hemolytic anemia?

Research has shown that Zola-cel, also known as BMS-986353, could aid in treating autoimmune diseases. In past studies, similar treatments targeting CD19 cells have proven effective in conditions like systemic lupus erythematosus. These treatments modify T-cells, a type of immune cell, to locate and destroy harmful cells in the body. Early results suggest this method might improve symptoms in autoimmune hemolytic anemia (AIHA), where the immune system attacks red blood cells. This trial will evaluate Zola-cel in different cohorts, including those specifically for AIHA. Although more research is needed, Zola-cel's mechanism offers hope for effective treatment options. ^{1 2 3 5 6}

Who Is on the Research Team?

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

This trial is for people with chronic immune thrombocytopenia (cITP) or autoimmune hemolytic anemia (AIHA), who have not responded to, cannot tolerate, or have relapsed after at least two previous treatments. Participants must not have other systemic autoimmune diseases.

Inclusion Criteria

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

I have been diagnosed with hemolysis.

I have chronic ITP and no other autoimmune diseases.

Exclusion Criteria

ITP: ALT/AST > 3 × ULN; AIHA: ALT > 3 × ULN, AST up to 5 × ULN may be permitted

ITP: total bilirubin > 1.5 × ULN; AIHA: direct bilirubin > 1.5 × ULN

International normalized ratio (INR) > 1.5 × ULN

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Zola-cel (BMS-986353) for the treatment of chronic immune thrombocytopenia (cITP) and autoimmune hemolytic anemia (AIHA)

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Cyclophosphamide
• Fludarabine Phosphate
• Zola-cel (BMS-986353)

Trial Overview

The study tests Zola-cel (BMS-986353), a new cell therapy, in combination with fludarabine and cyclophosphamide. It aims to see if this treatment is safe and effective for cITP and AIHA patients who haven't improved with standard therapies.

How Is the Trial Designed?

Group I: Cohort 2 Experimental Treatment 3 Interventions

Group II: Cohort 1 Part B Experimental Treatment 3 Interventions

Group III: Cohort 1 Part A ITP Experimental Treatment 3 Interventions

Group IV: Cohort 1 Part A AIHA Experimental Treatment 3 Interventions

Who Is Running the Clinical Trial?

Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

Lead Sponsor

Trials

19

Recruited

3,100+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07603557?lastUpdPost=2020-10-10_

Study of Zola-cel (BMS-986353), in Participants With ...

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

2.

withpower.com

withpower.com/trial/phase-2-anemia-hemolytic-autoimmune-5-2026-f4f1d

Zola-cel for Autoimmune Hemolytic Anemia

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

3.

hipa.ai

hipa.ai/study/NCT07603557

Study of Zola-cel (BMS-986353), in Participants With ...

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 ...

4.

ctv.veeva.com

ctv.veeva.com/study/study-of-zola-cel-bms-986353-in-participants-with-autoimmune-cytopenia-breakfree-aice

Study of Zola-cel (BMS-986353), in Participants With ...

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

5.

medcommsvdp.web.bms.com

medcommsvdp.web.bms.com/f5f8ca35-de1c-4635-8ba4-c127a1709f00/6d003fa0-b4c0-416f-bb55-ddf4c86940bd/6d003fa0-b4c0-416f-bb55-ddf4c86940bd_source__v.pdf

Tolerability and efficacy of BMS-986353 (CC-97540), a ...

• Here, we report updated data on the tolerability and efficacy of. BMS-986353 in the SSc cohort of Breakfree-1. Methods. • Inclusion criteria for the ...

6.

bmsstudyconnect.com

bmsstudyconnect.com/pl/en/clinical-trials/NCT07335562.html

Autoleucel / Zola-cel), CD19-CAR T Cells, Versus Standard of ...

The purpose of this study is to compare the efficacy and safety of BMS-986353 versus standard of care in participants with active Systemic Sclerosis.