Zelenirstat for Acute Myeloid Leukemia

Overseen By Pacylex Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Pacylex Pharmaceuticals

Must not be taking: Strong CYP3A4 inducers

Disqualifiers: Cardiac disease, Uncontrolled hypertension, HIV, others

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

Compensation: Varies

Number of Visits: Unknown

35 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a dose-finding study of oral zelenirstat (PCLX-001) in patients with R/R AML. There are two parts to the study: Dose Escalation and Dose Expansion.

Research Team

Naveen Pemmaraju

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for adults with relapsed or refractory Acute Myeloid Leukemia (R/R AML) who have tried at least one other therapy and are not eligible for treatments expected to help them. They must be able to understand the study, sign consent, have certain liver function test levels, and an ECOG performance status of 0-2.

Inclusion Criteria

My heart is functioning well.

My liver is working well.

Agreement to use adequate contraception during and after the study period

Exclusion Criteria

I have an active hepatitis B or C infection.

I am currently receiving radiation or cancer treatment for another cancer.

Known hypersensitivity to study drugs or excipients

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Determine the minimum safe and biologically-effective dose of daily oral PCLX-001 in patients with R/R AML using a Bayesian optimal interval design

28-day cycles, up to 18 months

Continuous daily dosing

Dose Expansion

Treat 20 evaluable patients with the minimum safe and biologically-effective dose to determine safety and preliminary clinical activity

28-day cycles, up to 18 months

Continuous daily dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

• PCLX-001

Trial Overview The study tests different doses of a new oral drug called zelenirstat (PCLX-001) in two parts: finding the right dose and then seeing how well it works at that dose. It's focused on patients whose AML has come back or hasn't responded to treatment.

Participant Groups

Group I: zelenirstat intervention in R/R AML at 70mg daily Experimental Treatment 1 Intervention

Three patients will be treated at 70 mg daily dose level. If 0/3 patients experience DLT, 3 patients will be treated at the next dose level.

Group II: zelenirstat intervention in R/R AML at 40mg daily Experimental Treatment 1 Intervention

Three patients will be treated at 40 mg daily dose level. If 0/3 patients experience DLT, 3 patients will be treated at the next dose level.

Group III: zelenirstat intervention in R/R AML at 280mg daily (if needed) Experimental Treatment 1 Intervention

Three patients will be treated at 280 mg daily dose level. If 0/3 patients experience DLT, 3 additional patients will be treated at the same dose and the study will be concluded.

Group IV: zelenirstat intervention in R/R AML at 210mg daily (if needed) Experimental Treatment 1 Intervention

Three patients will be treated at 210 mg daily dose level. If 0/3 patients experience DLT, 3 patients will be treated at the next dose level.

Group V: zelenirstat intervention in R/R AML at 140mg daily Experimental Treatment 1 Intervention

Three patients will be treated at 140 mg each dose level. If 0/3 patients experience DLT, 3 patients will be treated at the next dose level.

Group VI: zelenirstat intervention in R/R AML at 100mg daily Experimental Treatment 1 Intervention

Three patients will be treated at 100 mg daily dose level. If 0/3 patients experience DLT, 3 patients will be treated at the next dose level.

Who Is Running the Clinical Trial?

Pacylex Pharmaceuticals

Lead Sponsor

Trials

2

Recruited

100+

Ozmosis Research Inc.

Industry Sponsor

Trials

25

Recruited

5,200+

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+