PCLX-001 for Lymphoma and Cancer

No longer recruiting at 5 trial locations

Overseen By Pacylex Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Pacylex Pharmaceuticals

Must not be taking: Steroids, CYP3A4 inhibitors

Disqualifiers: Cardiac disease, HIV, Hepatitis, others

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

Compensation: Varies

Number of Visits: 2

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial investigates PCLX-001, a new oral medication, to determine the optimal dose for treating certain types of cancer and B-cell lymphomas. Researchers aim to understand how the body processes the drug and its effects on cancer cells. They test different doses to identify which is most effective with the fewest side effects. Individuals with advanced solid tumors or B-cell lymphomas who have not responded to other treatments may be suitable for this study. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I have to stop taking my current medications for the trial?

The trial requires you to stop using strong CYP3A4 inhibitors and inducers at least 14 days before starting the study drug and throughout the study. If you're taking these types of medications, you'll need to stop them to participate.

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

Research has shown that PCLX-001 has been tested in humans. In earlier studies with patients who had relapsed or hard-to-treat lymphoma, the drug showed promise. Researchers primarily assessed patient tolerance to the drug. The results indicated that PCLX-001 was generally well-tolerated at various dose levels. Some side effects were reported, but they were mostly mild, meaning they were not severe for most participants.

This is an early-stage study, which often focuses on determining the right dose and identifying possible side effects. Since PCLX-001 remains in early testing, further research is necessary to fully understand its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about PCLX-001 for lymphoma and cancer because it offers a novel approach compared to existing treatments like chemotherapy and radiation. Unlike these traditional therapies that often target rapidly dividing cells non-specifically, PCLX-001 works by specifically inhibiting a key enzyme involved in the cancer cells' metabolism, potentially reducing collateral damage to healthy cells. Additionally, PCLX-001 is administered orally, making it more convenient than many current treatments that require hospital visits for intravenous administration. This unique mechanism and delivery method could lead to more targeted therapies with fewer side effects, and that’s why it’s generating buzz in the medical community.

What evidence suggests that PCLX-001 might be an effective treatment for lymphoma and cancer?

Research shows that PCLX-001 effectively treats lymphoma. Earlier studies found PCLX-001 to be up to 10 times more powerful at killing lymphoma cells than some leading cancer drugs. Lab research also demonstrated that PCLX-001 can completely halt cancer cell growth. Additionally, early human trials found PCLX-001 safe and generally well-tolerated at doses up to 200 mg per day. These findings suggest that PCLX-001 has strong potential to effectively treat lymphoma and similar cancers. ^{1 3 4 6 7}

Who Is on the Research Team?

Randeep Sangha

Principal Investigator

Cross Cancer Institute

Are You a Good Fit for This Trial?

Adults with certain advanced cancers (like B-cell lymphomas and solid tumors) who've tried at least one other treatment without success are eligible for this trial. They should have a life expectancy of over 12 weeks, be able to perform daily activities with minimal assistance (ECOG status 0-1), and have organs functioning well enough to meet specific medical tests. Pregnant women can't join, and participants must agree to use contraception.

Inclusion Criteria

Ability to understand and willingness to sign a written informed consent

Women of childbearing potential must have a negative serum beta human chorionic gonadotropin (β-HCG) pregnancy test obtained within 7 (±3) days before the start of administration of study drug

My heart functions well, confirmed by a heart scan.

Exclusion Criteria

Previous assignment to treatment during this study

Clinically relevant findings in the ECG such as a second- or third-degree atrioventricular block, prolongation of the QRS complex > 120 ms (except for bundle branch block pattern), or prolongation of the of the QTc interval (Fridericia) over 450 ms unless agreed otherwise between the investigator and the sponsor's medically responsible person

I do not have serious infections or those not responding to treatment.

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 PCLX-001 to determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D)

28 days per cycle, multiple cycles

Visits at the end of each cycle for safety review

Dose Expansion

Participants receive PCLX-001 at the RP2D to evaluate preliminary clinical activity

28 days per cycle, multiple cycles

Tumor measurements at initiation and end of every 2nd cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PCLX-001

Trial Overview The trial is testing PCLX-001, an oral drug, in two parts: dose escalation to find the safe amount that can be given, followed by expansion cohorts where more patients receive this determined dose. It's open-label meaning everyone knows what treatment they're getting; it's not randomized or controlled against another treatment.

How Is the Trial Designed?

Group I: PCLX-001 intervention 70mg Experimental Treatment 1 Intervention

Cohort 3: Participants were administered 70mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group II: PCLX-001 intervention 40mg Experimental Treatment 1 Intervention

Cohort 2: Participants were administered 40mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group III: PCLX-001 intervention 280mg Experimental Treatment 1 Intervention

Cohort 7: Participants were administered 280mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group IV: PCLX-001 intervention 210mg Experimental Treatment 1 Intervention

Cohort 6: Participants were administered 210mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group V: PCLX-001 intervention 20mg Experimental Treatment 1 Intervention

Cohort 1: Participants were administered 20mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group VI: PCLX-001 intervention 140mg Experimental Treatment 1 Intervention

Cohort 5: Participants were administered 140mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Group VII: PCLX-001 intervention 100mg Experimental Treatment 1 Intervention

Cohort 4: Participants were administered 100mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.

Who Is Running the Clinical Trial?

Pacylex Pharmaceuticals

Lead Sponsor

Trials

2

Recruited

100+

Ozmosis Research Inc.

Industry Sponsor

Trials

25

Recruited

5,200+

Citations

1. pacylex.reportablenews.com pacylex.reportablenews.com/pr/pacylex-targeting-cancer-with-novel-mechanism-and-precision-approach

PCLX-001 has robust efficacy in leukemia and lymphoma. Pacylex data (on file) shows that PCLX-001 may be up to 10x more potent at killing lymphoma cells than two blockbuster cancer drugs, Imbruvia® (ibrutinib) and ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.e15094

A first-in-human, open-label, phase I trial of daily oral PCLX ... Conclusions: PCLX-001 is safe and well tolerated up to the 200 mg daily dose. Our results support the ongoing development of PCLX-001 as an oral ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8947478/

Novel, First-in-Human, Oral PCLX-001 Treatment in a ... Preclinical studies have shown that PCLX-001 markedly inhibits hematologic and lymphoma cell lines in tissue culture, and achieves complete ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04836195

NCT04836195 | Study of PCLX-001 in R/R Advanced Solid ... This is a phase I dose-escalation study of oral PCLX-001, conducted in a multicenter, non-randomized, open-label, non-controlled design.

5. pacylex.com pacylex.com/post/safety-and-efficacy-results-phase-1

Pacylex Pharmaceuticals Reports Safety and Efficacy ... The Phase 1 dose escalation safety and tolerability study was conducted in 29 lymphoma and solid tumor patients, who averaged 4 prior lines of ...

6. pacylex.reportablenews.com pacylex.reportablenews.com/pr/pacylex-pharmaceuticals-reports-zelenirstat-phase-1-safety-and-efficacy-data-at-asco-2024

Pacylex Pharmaceuticals Reports Zelenirstat Phase 1 ... The Phase 1 dose escalation safety and tolerability study was conducted in 29 patients with refractory/relapsed (r/r) lymphoma and refractory ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S0006497118730036

Nonclinical Efficacy and Toxicity and Selection of a Safe ... In mice, PCLX-001 produced complete remission of most xenografts at ≥35 mg/kg/day but also produced some deaths associated with bacterial ...