CBX-250 for Acute Myeloid Leukemia

(CROSSCHECK-001 Trial)

Recruiting at 6 trial locations

Overseen By Rachel Ghiraldi

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Crossbow Therapeutics, Inc.

Must not be taking: Anti-leukemic therapy

Disqualifiers: HIV, Hepatitis B, Cardiac disease, others

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

Compensation: Varies

Number of Visits: Unknown

72 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests CBX-250, an experimental drug for certain blood cancers, including acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML). The goal is to assess the safety and effectiveness of CBX-250, particularly for those unresponsive to other treatments. Participants will receive injections over a 28-day cycle. The trial seeks individuals with advanced blood cancers that have recurred or resisted previous treatments and who possess a specific genetic marker (HLA-A*02:01). As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires stopping most anti-leukemic therapies at least 14 days before starting CBX-250, except for hydroxyurea, which can be continued with approval. Other medications like steroids and biologics have specific time requirements before starting the trial.

Is there any evidence suggesting that CBX-250 is likely to be safe for humans?

Research has shown that CBX-250 is being tested for safety and tolerance in people with certain blood cancers, such as acute myeloid leukemia. Early studies suggest that most patients tolerate the treatment well. These studies aim to determine the treatment's safety and potential side effects.

Since CBX-250 is in the early testing stages, the primary goal is to observe how the body reacts and identify any side effects. This phase is the first step in assessing the treatment's safety for people. While detailed information on specific side effects is not yet available, this phase is crucial for identifying potential risks.

Prospective trial participants should know that the treatment is still undergoing safety testing. Consulting with a healthcare provider can help clarify what this might mean for individual circumstances. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CBX-250 for acute myeloid leukemia because it offers a novel approach compared to standard chemotherapy treatments. CBX-250 is administered subcutaneously, which can be more convenient and less invasive than traditional intravenous methods. Additionally, it targets specific pathways in cancer cells, potentially leading to fewer side effects and improved effectiveness. This new mechanism of action could provide a promising alternative for patients who do not respond well to existing therapies.

What evidence suggests that CBX-250 might be an effective treatment for acute myeloid leukemia?

Research shows that CBX-250, the investigational treatment in this trial, is designed to help the immune system fight leukemia by using T-cells, a type of immune cell, to attack the cancer. Studies have found that it can destroy leukemia cells even if they lack a specific marker, thanks to a process called bystander killing. This means CBX-250 might work against cancer cells that usually avoid treatment. Early results also suggest it could help treat myeloid cancers, including acute myeloid leukemia (AML). While more research is needed, these initial findings are promising for people with relapsed or hard-to-treat AML. ^{1 2 4 6 7}

Who Is on the Research Team?

Briggs Morrison, MD

Principal Investigator

Crossbow Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for individuals aged ≥12 with relapsed/refractory Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia who have not responded to certain treatments. Participants must have a specific HLA type and white blood cell counts below a set threshold.

Inclusion Criteria

My MDS is high-risk and hasn't responded to 4-6 cycles of specific treatments.

I have been tested positive for the HLA-A*02:01 gene.

My daily activity is not severely limited.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Priming Phase

Participants receive a priming dose of CBX-250 over 7 days

1 week

Target Phase

Participants receive CBX-250 on a fixed step-up dosing schedule over 28 days

4 weeks

Subsequent Treatment Cycles

Participants continue CBX-250 in 28-day cycles until progressive disease or unacceptable toxicity

Until disease progression or unacceptable toxicity

Follow-up

Participants are monitored for safety and effectiveness after treatment

Approximately 3 years

What Are the Treatments Tested in This Trial?

Interventions

• CBX-250

Trial Overview CBX-250 is being tested in this Phase 1 trial. It's given as an under-the-skin injection in cycles of 28 days, starting with a priming phase followed by a target phase. Patients continue until disease progression or unacceptable side effects occur.

How Is the Trial Designed?

Group I: CBX-250 Experimental Treatment 1 Intervention

subcutaneous CBX-250

Who Is Running the Clinical Trial?

Crossbow Therapeutics, Inc.

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06994676

A Study of CBX-250 in Participants With Acute Myeloid ... Study CBX-250-001 is a Phase 1, open-label, dose-escalation study of CBX-250 in participants with relapsed/refractory AML, HR-MDS and CMML.

2. rarediseaseadvisor.com rarediseaseadvisor.com/news/first-patient-dosed-phase-1-trial-cbx250-mds/

First Patient Dosed in Phase 1 Trial of CBX-250 for MDS The CROSSCHECK-001 study is a first-in-human trial of CBX-250, a novel T-cell engager for myeloid malignancies, including MDS.

3. app.trialscreen.org app.trialscreen.org/trials/phase-1-cbx-250-participants-acute-myeloid-leukemia-high-risk-myelodysplastic-trial-nct06994676

A Study of CBX-250 in Participants With Acute Myeloid ... Benefits: Participation in this study provides an opportunity to receive a potentially effective investigational treatment for AML, HR-MDS, or CMML. The study ...

4. ash.confex.com ash.confex.com/ash/2024/webprogram/Paper201699.html

CBX250 Is a Novel Cathepsin G Peptide-HLA-Targeting T ... Our results demonstrated that CBX-250 was able to induce dose-dependent bystander killing of neighboring CG1/pMHC target-negative leukemia cells ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124029550

CBX250 Is a Novel Cathepsin G Peptide-HLA-Targeting T ... Our results demonstrated that CBX-250 was able to induce dose-dependent bystander killing of neighboring CG1/pMHC target-negative leukemia cells ...

6. firstwordpharma.com firstwordpharma.com/story/6092973

Crossbow Therapeutics Doses First Patient in Phase 1 ... The study is evaluating the safety, tolerability, and preliminary clinical activity of CBX-250 in patients aged 12 years and older with relapsed ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12523351/

T-Cell Engagers in Acute Myeloid Leukemia CBX-250 is to be administered via SC administration in 28-day cycles. In Cycle 1, a priming phase will occur over 7 days, with a target phase ...