CAR T Cell Therapy for Leukemia

Not currently recruiting at 39 trial locations

Overseen By Autolus Limited

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Autolus Limited

Disqualifiers: CNS pathology, Infections, Hepatitis, HIV, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 2 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AUTO1, a type of CAR T cell therapy, for adults with B cell acute lymphoblastic leukemia (ALL) that hasn't responded to treatment or has returned. The goal is to determine if AUTO1 is safe and effective by using specially modified T cells to target leukemia cells. Suitable candidates have B cell ALL that has relapsed or not responded to treatment and a confirmed presence of a specific marker called CD19. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that AUTO1 is likely to be safe for humans?

Research has shown that CAR T-cell treatments, like AUTO1, offer potential benefits but also present safety challenges. Studies often report significant side effects, such as Cytokine Release Syndrome (CRS), occurring in about 81.8% of patients. CRS involves a large release of immune proteins, leading to symptoms like fever and tiredness. Additionally, neurological issues, such as confusion or headaches, occur in about 30% of patients, with severe cases in around 10%.

Although these side effects are common with CAR T-cell therapies, ongoing research aims to balance safety and effectiveness. Researchers continue to study these treatments to better understand and manage these side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

AUTO1 is unique because it uses CAR T cell therapy, which is a groundbreaking approach for treating leukemia. Unlike traditional treatments like chemotherapy or stem cell transplants, AUTO1 involves reprogramming a patient's own immune cells to specifically target and destroy cancer cells. Researchers are excited about AUTO1 because it offers a targeted attack on leukemia cells, potentially reducing the risk of harming healthy cells and leading to fewer side effects. Additionally, this personalized treatment has the promise of long-lasting remission, making it a hopeful option for patients who haven't responded well to other therapies.

What evidence suggests that AUTO1 might be an effective treatment for leukemia?

Research shows that CAR T-cell therapy, such as AUTO1, holds promise for treating B cell acute lymphoblastic leukemia (ALL) that has returned or is unresponsive to other treatments. Studies have found that many patients achieve a state where almost no cancer cells are detectable after treatment, with a success rate of 70%. This indicates that a significant number of patients have very few cancer cells remaining post-treatment. The therapy precisely targets cancer cells, potentially leading to fewer side effects compared to other treatments. Overall, these findings suggest that AUTO1 could be an effective option for patients facing this challenging type of leukemia. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Adults over 18 with B cell acute lymphoblastic leukemia that's come back or hasn't responded to treatment. They must have a certain level of cancer cells in their bone marrow, be relatively healthy (ECOG status 0 or 1), and not have severe infections or other specific blood diseases. People with heart, liver, kidney, or lung problems can't join.

Inclusion Criteria

My kidney, liver, lung, and heart functions are all within normal ranges.

I am in my 2nd or later complete remission, with minimal disease detected and less than 5% cancer cells in my bone marrow.

Phase Ib: Primary Cohort IA: Presence of ≥5% blasts in BM at screening

Exclusion Criteria

I am currently fighting an infection that needs strong medication.

I have had CD19 therapy but not blinatumomab, or I had severe side effects from blinatumomab.

I have been diagnosed with Burkitt's leukemia/lymphoma or CML in blast crisis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Leukapheresis

Collection of white blood cells from participants to engineer CAR T cells

1 week

Pre-conditioning

Participants undergo pre-conditioning chemotherapy to prepare for CAR T cell infusion

1 week

Treatment

Participants receive a total target dose of 410E+6 CAR T cells as a split dose on Day 1 and Day 10

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AUTO1

Trial Overview The trial is testing AUTO1, a CAR T cell therapy designed to target CD19 on cancer cells in adults with relapsed/refractory B-ALL. It aims to see how safe it is and how well it works at different stages of the disease.

How Is the Trial Designed?

Group I: AUTO1 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Autolus Limited

Lead Sponsor

Trials

9

Recruited

1,000+

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.7000

Safety and efficacy of obecabtagene autoleucel (obe-cel, ... At screening, patients had a median of 55% BM blasts (range 6-96%) and 26% had EMD. The geometric mean of peak CAR expansion was 126147.6 copies ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/5877/527643/Long-Term-Outcomes-and-Adverse-Events-of-CAR-T-19

Long-Term Outcomes and Adverse Events of CAR T-19 Cell ... The pooled analysis demonstrated a high rate of minimal residual disease-negative complete remission (MRD-CR), with an overall event rate of 70% ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11095278/

Comprehensive analysis of the efficacy and safety of CAR ... The principal advantage of CAR T-cell therapy is the high specificity, leading to reduced occurrence of side effects which are beneficial for ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S1567576924008324

CAR-T cell therapy: Efficacy in management of cancers ... CD22 CAR T-cells demonstrated a favorable complete response rate of 68 % in ALL and 64 % in NHL, even in cases where patients had previously ...

5. nature.com nature.com/articles/s41392-025-02269-w

CAR-T cell therapy for cancer: current challenges and ... 2-targeted CAR has proven effective in treating gastrointestinal tumors in a study including 37 patients (NCT04196413), with an overall response ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10505056/

CAR-T Cell Therapy: the Efficacy and Toxicity Balance - PMC Neurological events occurred in 30% of patients, with grade 3 or higher symptoms in 10%. Overall, both trials assessing tisagenlecleucel and ...

7. nature.com nature.com/articles/s41409-025-02691-2

Safety and efficacy of autologous humanized CD19 CAR-T ... Despite the remarkable clinical outcomes achieved by CAR-T cell therapy, the high rate of relapse and severe adverse events like cytokine ...