Evaluating JBZ-001 for patients with advanced solid and blood cancers

A Phase 1, Open-label, Dose-escalation and Expansion, First-in-human Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of JBZ-001, a Dihydroorotate Dehydrogenase (DHODH) Inhibitor, in Patients With Advanced Solid and Hematological Malignancies

This study is testing a new drug called JBZ-001 to see if it can safely help people with advanced solid tumors and blood cancers that haven't responded to other treatments.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Jabez Bioscience, Inc (industry)
• Locations: 2 sites (Columbus, Ohio and 1 other locations)
• Trial ID: NCT06801002 on ClinicalTrials.gov

What this trial studies

This phase 1, open-label trial aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of JBZ-001, a DHODH inhibitor, in patients with refractory solid tumors and hematological malignancies. The study consists of two parts: a dose escalation phase for solid tumors and non-Hodgkin lymphoma (NHL), followed by indication expansions for selected tumor types. Patients will be enrolled using a '3+3' design, with a focus on monitoring dose-limiting toxicities and potential efficacy signals. The trial will also evaluate the optimal biologic dose of JBZ-001 and includes specific eligibility criteria for participants.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with relapsed or refractory advanced solid tumors or specific types of non-Hodgkin lymphoma for which no standard treatment is available.

Not a fit: Patients with early-stage cancers or those who have not yet received standard treatments may not benefit from this trial.

Why it matters

Potential benefit: If successful, this trial could provide a new treatment option for patients with advanced solid tumors and hematological malignancies who have exhausted standard therapies.

How similar studies have performed: Other studies evaluating DHODH inhibitors have shown promise, suggesting potential for success in this novel approach.

Eligibility criteria

Inclusion Criteria:

1. Participant must be ≥18 years of age, at the time of signing the informed consent.
2. Dose escalation and expansion:

   1. Solid tumors: have a histologically confirmed relapsed or refractory advanced solid tumor for which no standard approved treatment is available, or is ineligible for, or did not tolerate standard approved treatment.
   2. NHL: have a histologically confirmed relapsed or refractory diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) or Mantle cell lymphoma (MCL) for which no standard approved treatment is available, or is ineligible for, or did not tolerate standard approved treatment.
3. Measurable/evaluable disease or documented relapse, respectively, relevant for tumor type as follows:

   1. Solid tumors: per Response evaluation criteria in solid tumors (RECIST) 1.1 with at least one target lesion
   2. NHL: Measurable disease defined as ≥1 measurable nodal lesion (long axis \>1.5 cm and short axis \>1.0 cm) or ≥1 measurable extra-nodal lesion (long axis \>1.0 cm) on computed tomography (CT) scan or magnetic resonance imaging (MRI)
4. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
5. All previous anti-cancer therapy-related AEs should have resolved to grade 1 or baseline value with the exception of alopecia and stable, treated endocrine toxicities of immune checkpoint inhibitors (ICIs) Note: Subjects with irreversible toxicity that in the opinion of the treating physician is not reasonably expected to be exacerbated by the investigational product may be included (e.g., hearing loss, hormone deficiency requiring replacement therapy) -

Exclusion Criteria:

1. Known hypersensitivity to JBZ-001 or any of its excipients
2. Corrected interval between Q and T wave on ECG (QTc) ≥ 470 msec using Fredericia's formula.
3. Has significant and symptomatic cardiovascular disease (such as congestive heart failure New York Heart Association class III or higher, myocardial infarction, cerebrovascular disease, unstable angina, unstable arrhythmia) within the 3 months prior to first dose of JBZ-001.
4. Has another malignant disease requiring treatment, with the exception of curatively treated malignancies or malignancies with very low potential for recurrence or progression.
5. For solid tumor subjects:

   1. Symptomatic ascites or pleural effusion. A subject who is clinically stable for 2 weeks following treatment for these conditions (including therapeutic thoraco- or paracentesis) is eligible.
   2. Known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are clinically stable for at least 2 weeks prior to study entry, as long as the dose is ≤ 4 mg of dexamethasone or equivalent per day
6. Known active HIV infection on antiretroviral therapy. Note: Testing is not required for eligibility.
7. Known active infection with hepatitis B or hepatitis C. Note: Testing is not required for eligibility.
8. Any other active infection requiring systemic therapy.
9. Major surgery (excluding procedures to stabilize the vertebrae) within 4 weeks or minor surgery within 2 weeks prior to first dose of JBZ-001.
10. Has a history of GI surgery or other procedures that might interfere with the absorption or swallowing of the study drug.
11. History or clinical evidence of any medical condition which the investigator judges as likely to interfere with the results of the study, poses an additional risk in participating, or makes the subject unlikely to comply with the study-related visits and assessments.
12. Female participants: pregnant or breastfeeding.

Where this trial is running

Columbus, Ohio and 1 other locations

• The Ohio State University — Columbus, Ohio, United States (RECRUITING)
• START Mountain Region — West Valley City, Utah, United States (RECRUITING)

Study contacts

• Study coordinator: Steve Kaesshaefer

Conditions: Solid Tumor, lymphoma, NHL, Non-Hodgkin's lymphoma, DHODH, Small Molecule, DHO