T Cell Therapy for AML or MDS

Not currently recruiting at 4 trial locations

Overseen By Gloria Hoyah

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: NexImmune Inc.

Must not be taking: Steroids, Immunosuppressants

Disqualifiers: GvHD, Cardiovascular disease, Autoimmune, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new experimental therapy called NEXI-001, which uses special T cells (a type of immune cell) to target cancer in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). It focuses on those who have relapsed after receiving a stem cell transplant from a matching donor. The goal is to determine if this treatment is safe and effective in fighting cancer. Individuals with AML or MDS who have experienced a relapse after a matching stem cell transplant might be suitable candidates for this trial. 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 have to stop taking my current medications for the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you will undergo bridging therapy before the main treatment, and the choice of this therapy is up to the investigator, limited to certain acceptable agents. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that the NEXI-001 T cell product is likely to be safe for humans?

Research has shown that the NEXI-001 T cell product is being tested for safety and patient tolerance in those with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). Early results indicate that most participants have tolerated it well. Some have experienced mild to moderate side effects, which are common with many treatments. Serious side effects have not been a major issue so far, but ongoing studies aim to better understand its safety. As NEXI-001 remains under investigation, researchers continue to monitor for any new safety concerns. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), which often involve chemotherapy or stem cell transplants, the NEXI-001 T cell therapy is derived from the T cells of the original HLA-matched hematopoietic cell transplant donor. This approach is unique because it leverages the patient's immune system to specifically target tumor cells. Researchers are excited about NEXI-001 because it has the potential to offer a more targeted and personalized treatment with potentially fewer side effects compared to conventional therapies. By directly harnessing the power of the immune system, this therapy might improve survival rates and quality of life for patients with AML and MDS.

What evidence suggests that the NEXI-001 T cell product might be an effective treatment for AML or MDS?

Research has shown that the NEXI-001 T cell treatment could be promising for people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). Participants in this trial will receive the NEXI-001 T cell product, which early results suggest can effectively find and fight leukemia cells. Importantly, these T cells seem to enhance the immune system's natural ability to combat cancer without causing severe side effects like graft-versus-host disease (GVHD), which can occur with other treatments. Initial studies found that the treatment is generally well-tolerated and manageable for patients. Overall, these findings offer hope that NEXI-001 could be an effective option for those with relapsed AML or MDS after a stem cell transplant. ^{1 2 4 6 7}

Who Is on the Research Team?

Juan Varela, MD, PhD

Principal Investigator

Principal Investigator

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) who have relapsed after a matching bone marrow transplant. They must be in good physical condition, able to follow the study plan, and have specific genetic markers. Those with mismatched transplants or certain levels of disease are not eligible.

Inclusion Criteria

My tests show mutations in my blood cell genes.

You understand and agree to take part in the study, and you are able to follow the study's requirements and provide any needed samples for research.

I am fully active or restricted in physically strenuous activity but can do light work.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Bridging Therapy

Participants undergo bridging therapy for disease control while the NEXI-001 T cell product is being manufactured

Varies

Lymphodepleting Therapy

Participants receive lymphodepleting therapy prior to the infusion of NEXI-001 T cell product

2 weeks

Treatment

Participants receive a single IV infusion of the NEXI-001 T cell product

1 day

Post-Treatment Follow-Up

Participants are monitored for adverse events and anti-leukemia response until the end of study visit is complete

Up to 1 year

Overall Survival Follow-Up

Participants are followed for overall survival from time of disease progression until the last visit of the last patient

12 week intervals

What Are the Treatments Tested in This Trial?

Interventions

• NEXI-001 T cell product

Trial Overview The trial tests NEXI-001 T cells, an experimental therapy targeting leukemia antigens in AML/MDS patients post-transplant relapse. It involves bridging therapy for disease control while preparing the T cell product, followed by lymphodepleting therapy and then a single infusion of NEXI-001.

How Is the Trial Designed?

Group I: Safety Evaluation Phase Experimental Treatment 1 Intervention

Treatment with NEXI-001 T cells, derived from PBMCs of original HLA- matched HCT donor.

Group II: Dose Expansion Phase Experimental Treatment 1 Intervention

Dose Expansion Phase to further define the safety, tolerability and initial anti-tumor efficacy of the NEXI-001 T cell product at the dose established from the Safety Evaluation Phase.

Who Is Running the Clinical Trial?

NexImmune Inc.

Lead Sponsor

Trials

3

Recruited

70+

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.7043

An analysis of a first-in-human study of NEXI-001 donor- ... Early results indicate that the antigen-specific NEXI-001 T cells have the potential to enhance GvL effects and is tolerated with easily manageable side ...

2. mycancergenome.org mycancergenome.org/content/clinical_trials/NCT04284228/

Clinical Trial: NCT04284228 This Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product (a ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04284228?tab=history&a=1

Record History | ver. 1: 2020-02-21 | NCT04284228 This Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product.

4. biospace.com biospace.com/neximmune-presents-initial-positive-data-from-the-nexi-001-phase-1-trial-for-relapsed-refractory-post-allo-hsct-aml-at-the-american-society-of-clinical-oncology-2023-annual-meeting

NexImmune Presents Initial Positive Data from the NEXI ... The goals of the NEXI-001 Phase 1 clinical trial are to evaluate the safety, tolerability, immune response, and clinical activity of the antigen ...

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.2021.39.15_suppl.2538

Preliminary analysis of a phase 1/2 study of NEXI-001 ... NEXI-001 has the potential to enhance GvL effect without the associated toxicities of GVHD, cytokine release syndrome, and neurotoxicity.

6. clin.larvol.com clin.larvol.com/trial-detail/NCT04284228

Antigen-specific T Cell Therapy for AML or MDS Patients ... Entitled 'Preliminary Results of the First-in-Human Study of NEXI-001, a Multi-Antigen Specific CD8+ T Cell Product, in Acute Myeloid Leukemia (AML) Patients ...

7. cancer.gov cancer.gov/clinicaltrials/NCI-2020-01658

Antigen-specific T Cell Therapy for AML or MDS Patients ... This Research study is being done to characterize the safety, tolerability, and preliminary antitumor activity of the NEXI-001 T cell product (a new ...