JBZ-001 for Cancer

Recruiting at 1 trial location

Overseen By Steve Kaesshaefer

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Jabez Bioscience, Inc

Must not be taking: Antiretrovirals, Corticosteroids

Disqualifiers: Cardiovascular disease, CNS metastases, HIV, others

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

Compensation: Varies

Number of Visits: 2

25 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called JBZ-001 for people with hard-to-treat cancers, including some solid tumors and specific blood cancers like certain lymphomas. The study aims to determine the safety and tolerability of JBZ-001 and assess its effectiveness against these cancers. Participants will receive varying doses of the drug to identify the optimal dose. The trial seeks individuals with advanced cancer that hasn't responded to other treatments or who can no longer undergo standard treatment. As a Phase 1 trial, this research focuses on understanding how JBZ-001 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that JBZ-001 is likely to be safe for humans?

Research shows that JBZ-001, a new treatment under investigation, is in the very early stages of study. It has not yet been fully tested for safety in people. So far, studies have focused on the drug's tolerability and potential side effects.

Early research typically involves only a small number of participants. This approach helps doctors determine the safest dose to administer. The treatment is given in varying amounts to assess what participants can tolerate without serious side effects.

JBZ-001 is being studied for use in hard-to-treat cancers, such as certain solid tumors and lymphomas (a type of blood cancer). Since the trial is in its first phase, safety information is still being collected. This phase is crucial for identifying the optimal dose that balances effectiveness and safety.

Although detailed safety information is not yet available, the trial is designed to closely monitor any side effects. Participants in this study will help researchers learn more about the safety profile of JBZ-001 and its potential side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about JBZ-001 for cancer because it represents a potential breakthrough in targeting cancer cells more effectively. Unlike traditional chemotherapy, which attacks both cancerous and healthy cells, JBZ-001 is designed to selectively target only the cancer cells, potentially reducing side effects and improving patient quality of life. Additionally, JBZ-001 is administered orally, which is more convenient compared to the intravenous methods often required for current cancer treatments. This new treatment also explores varying dosages, ranging from 5 mg to 25 mg daily, allowing researchers to determine the most effective dose with the least side effects.

What evidence suggests that JBZ-001 might be an effective treatment for cancer?

Research has shown that JBZ-001, a new drug, shows promise in fighting difficult cancers like lymphoma and leukemia. Early studies found that it blocks a substance cancer cells need to grow. Specifically, 16.7% of patients in these studies had tumors that shrank significantly. Additionally, 44.4% of patients experienced disease control, meaning their cancer stopped growing or spreading temporarily. These initial results suggest JBZ-001 could effectively treat certain stubborn cancers. ^{1 6 7 8 9}

Who Is on the Research Team?

Zuzana Jirakova, MD PhD

Principal Investigator

Jabez Biosciences, Inc

Are You a Good Fit for This Trial?

This trial is for patients with advanced solid tumors or blood cancers (hematological malignancies) who have not responded to standard treatments. Participants must meet certain health criteria to be eligible.

Inclusion Criteria

I can perform daily activities with minimal assistance.

For dose escalation and expansion: - Solid tumors: histologically confirmed relapsed or refractory advanced solid tumor with no standard approved treatment available, or ineligible for, or did not tolerate standard approved treatment - NHL: histologically confirmed relapsed or refractory diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) or Mantle cell lymphoma (MCL) with no standard approved treatment available, or ineligible for, or did not tolerate standard approved treatment

My side effects from previous cancer treatments have mostly gone away.

Exclusion Criteria

Known hypersensitivity to JBZ-001 or any of its excipients

Corrected interval between Q and T wave on ECG (QTc) ≥ 470 msec using Fredericia's formula

I don't have any health issues that could affect the study or make it hard for me to follow the study plan.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive escalating doses of JBZ-001 to evaluate safety and tolerability

28 days

Weekly visits for dose escalation monitoring

Indication Expansion

Participants receive JBZ-001 in selected solid tumor types and NHL to evaluate efficacy

up to 1 year

Monthly visits for efficacy assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• JBZ-001

Trial Overview

The trial is testing JBZ-001, a DHODH inhibitor, in two parts: first, determining safe dosage levels in patients with solid tumors and non-Hodgkin lymphoma; second, assessing its effects on specific cancer types.

How Is the Trial Designed?

Group I: Experimental Cohort 5 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 25 mg orally daily

Group II: Experimental Cohort 4 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 17.5 mg orally daily

Group III: Experimental Cohort 3 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose of 10 mg orally daily

Group IV: Experimental Cohort 2 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose 5 mg orally daily

Group V: Experimental Cohort 1 Experimental Treatment 1 Intervention

1\~6 subjects in this cohort will receive 1 dose 5 mg orally.

Who Is Running the Clinical Trial?

Jabez Bioscience, Inc

Lead Sponsor

Trials

1

Recruited

30+

Published Research Related to This Trial

In a real-world study of 1002 patients with previously treated non-small cell lung cancer (NSCLC), atezolizumab showed an 18-month overall survival rate of 41.1%, confirming its effectiveness outside of clinical trial settings.

The safety profile was acceptable, with an overall adverse event rate of 43.9% and immune-related adverse events at 19.0%, but caution is advised for patients with poorer performance status (ECOG ≥ 2), as they had a significantly lower survival rate of 14.3%.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and Safety of Atezolizumab Monotherapy in Previously Treated Japanese Patients With Unresectable Advanced or Recurrent NSCLC: A Multicenter, Prospective, Observational Study (J-TAIL). Miura, S., Nishio, M., Akamatsu, H., et al.[2023]

The PePS2 trial demonstrated that pembrolizumab can be safely administered to patients with non-small-cell lung cancer (NSCLC) and a performance status of 2, with a toxicity rate of 28% and no severe treatment-related adverse events, indicating a manageable safety profile.

Efficacy outcomes showed that 38% of first-line and 36% of subsequent-line patients experienced durable clinical benefit, with higher response rates correlating with tumor proportion scores, suggesting that pembrolizumab is effective for this underserved patient group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pembrolizumab in patients with non-small-cell lung cancer of performance status 2 (PePS2): a single arm, phase 2 trial. Middleton, G., Brock, K., Savage, J., et al.[2020]

In a phase 2 trial involving 36 patients with untreated brain metastases from melanoma or non-small-cell lung cancer (NSCLC), pembrolizumab demonstrated activity, achieving a brain metastasis response in 22% of melanoma patients and 33% of NSCLC patients, with responses being durable for most.

The treatment had an acceptable safety profile, with serious adverse events occurring in a small percentage of patients, indicating that systemic immunotherapy could be a viable option for those with untreated or progressive brain metastases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pembrolizumab for patients with melanoma or non-small-cell lung cancer and untreated brain metastases: early analysis of a non-randomised, open-label, phase 2 trial. Goldberg, SB., Gettinger, SN., Mahajan, A., et al.[2022]

JTX-4014, a new PD-1 targeting antibody, was well tolerated in a trial with 18 patients and did not show new safety concerns compared to existing PD-1 inhibitors, establishing a recommended dose of 500 mg every 3 weeks or 1000 mg every 6 weeks.

The treatment demonstrated clinical activity with a 16.7% overall response rate and a 44.4% disease control rate, particularly in patients with PD-L1 positive tumors, suggesting potential for further development in cancer therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I, first-in-human trial of programmed cell death receptor-1 (PD-1) inhibitor, JTX-4014, in adult patients with advanced, refractory, solid tumors. Papadopoulos, KP., Lakhani, N., Falchook, GS., et al.[2021]

JS001, a monoclonal antibody targeting the PD-1 receptor, was well tolerated in a phase I study involving 36 patients with advanced melanoma and urologic cancers, with no dose-limiting toxicities observed.

The treatment showed promising anti-tumor activity, achieving a 22.2% objective response rate and a 50.0% disease control rate, particularly in patients with immune-active profiles, leading to further studies focusing on specific melanoma subtypes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and clinical activity with an anti-PD-1 antibody JS001 in advanced melanoma or urologic cancer patients. Tang, B., Yan, X., Sheng, X., et al.[2020]

Citations

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Phase 1 Trial of JBZ-001 Targets Solid Tumors and Non- ...

The inhibition shows promising results against aggressive cancers like lymphoma and leukemia, and we've also seen potential in some relapsed ...

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An open-label phase 1 study to investigate JBZ001 in adults ...

It showed superior preclinical efficacy when compared with other clinical candidates. It also showed a favorable toxicity profile with no ...

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health.osu.edu

health.osu.edu/health/cancer/new-cancer-drug-targets-tumor-growth-enzyme

New cancer drug developed at Ohio State in clinical trial

The phase I trial is testing a drug called JBZ-001 that blocks an enzyme that enables cancer cells to thrive.

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withpower.com

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JBZ-001 for Cancer

The treatment demonstrated clinical activity with a 16.7% overall response rate and a 44.4% disease control rate, particularly in patients with PD-L1 positive ...

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The primary objective is to evaluate the safety and tolerability and establish an OBD of single agent JBZ-001 in patients with solid tumors and NHL. Secondary ...

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Trial to Evaluate the Safety & Tolerability of JBZ-001 in Pts ...

This will be a phase 1, open-label, dose-escalation and expansion, FIH trial to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of ...

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Trial to Evaluate the Safety & Tolerability of JBZ-001 in Pts ...

The Phase 1 study, underway at OSUCCC - James, aims to assess the safety, tolerability, and preliminary efficacy of JBZ-001 in patients with advanced solid ...

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Trial to Evaluate the Safety & Tolerability of JBZ-001 in Pts ...

This will be a phase 1, open-label, dose-escalation and expansion, FIH trial to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of JBZ-001.

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Translational Pharmacokinetic-Pharmacodynamic Modeling of ...

Background: HOSU-53 (JBZ-001), an orally bioavailable new chemical entity, represents a highly potent dihydroorotate dehydrogenase (DHODH) ...