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B-cell Non Hodgkin LymphomaPhase 1NCT04836195

Study of PCLX-001 in R/R Advanced Solid Malignancies and B-cell Lymphoma

A Phase 1 study testing PCLX-001 - 20mg, PCLX-001 - 40mg and PCLX-001 - 70mg in people with B-cell Non Hodgkin Lymphoma and Advanced Solid Tumor, run by Pacylex Pharmaceuticals at 4 sites in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a phase I dose-escalation study of oral PCLX-001, conducted in a multicenter, non-randomized, open-label, non-controlled design. The study is comprised of two parts: Part A (single-agent dose escalation) and Part B (single-agent expansion cohorts). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
PCLX-001 - 20mg, PCLX-001 - 40mg and PCLX-001 - 70mg
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
4 sitesCanada
Study dates
14 Sep 2021to 28 Oct 2024
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Ability to understand and the willingness to sign a written informed consent. A signed informed consent must be obtained before any study-specific procedures are performed.
  • Male or female patients aged ≥ 18 years
  • Dose Escalation
  • Participants with histologically-confirmed advanced solid tumor who have failed at least one prior therapy and/or are not eligible for therapies expected to provide clinical benefit.
  • Histologically-confirmed B-cell lymphomas that are expected to express CD20 including DLBCL, HGBL, FL (grades 1 to 3b), MCL, and Burkitt lymphoma who have failed at least two prior therapies and/or are not eligible for therapies expected to provide clinical benefit (including autologous stem cell transplantation). Transformed large B-cell lymphoma patients are eligible. FL patients should meet criteria for requiring treatment.
  • Dose Expansion Cohort A: Participants with histologically-confirmed advanced breast, NSCLC, SCLC, colorectal, and bladder cancers who have failed at least one prior therapy and/or are not eligible for therapies expected to provide clinical benefit.
Show all 14Show fewer
  • Cohort B: Participants with histologically-confirmed R/R B-cell lymphomas that are expected to express CD20 including DLBCL, HGBL, FL (grades 1-3a), FL (grade 3b), MCL, and Burkitt lymphoma who have failed at least two prior therapies and/or are not eligible for therapies expected to provide clinical benefit. Transformed large B-cell lymphoma patients are eligible. FL patients should meet criteria for requiring treatment.
  • Patients must have evaluable or measurable disease.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
  • Life expectancy of at least 12 weeks
  • Patients must have adequate bone marrow, liver, kidney and cardiac function.
  • Patients must have adequate coagulation.
  • Women of childbearing potential must have a negative pregnancy test.
  • Women of childbearing potential and fertile men must agree to use adequate contraception when sexually active from signing of the informed consent form for the full study until at least 6 months after the last study drug administration.

Who cannot join

  • Known hypersensitivity to the study drugs or excipients of the preparations or any agent given in association with this study.
  • History of cardiac disease: congestive heart failure New York Heart Association (NYHA) class > II, unstable angina (angina symptoms at rest), new-onset angina (within the past 6 months before study entry), myocardial infarction within the past 6 months before study entry, or uncontrolled cardiac arrhythmias.
  • Uncontrolled arterial hypertension despite optimal medical management.
  • Moderate or severe hepatic impairment.
  • Patients with known human immunodeficiency virus (HIV) infection.
  • Patients who have an active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection requiring treatment.
Show all 23Show fewer
  • Infections not responding to therapy or active clinically serious infections.
  • Symptomatic metastatic brain or meningeal tumors unless the patient is > 3 months from definitive therapy, has a stable imaging study and is clinically stable. Patients with asymptomatic brain metastases must not be on steroid therapy.
  • Current or past history of central nervous system (CNS) lymphoma.
  • Uncontrolled seizure disorder requiring therapy.
  • History of organ allograft transplantation or autologous stem cell transplantation ≤ 3 months prior to the first dose of study drug. Patients who received prior CAR-T or other T-cell targeting treatment (approved or investigational) ≤ 4 weeks prior to study drug administration.
  • Evidence or history of bleeding disorder within 4 weeks before the first dose of study drug.
  • Serious, non-healing wound, ulcer, or bone fracture.
  • Any malabsorption condition.
  • Breastfeeding. Female patients must not breastfeed during treatment and until 4 months after last study drug administration.
  • Treatment with systemic steroids (prednisone dose ≥10 mg/day or equivalent dose).
  • Acute toxic effects of previous anticancer chemotherapy or immunotherapy that have not yet stabilized or if significant post-treatment toxicities have been observed.
  • Radiotherapy for target lesions during study or within 3 weeks before the first dose of study drug.
  • Major surgery or significant trauma within 4 weeks before the first dose of study drug.
  • Concomitant participation in another clinical study with investigational medicinal product(s).
  • Substance abuse, medical, psychological, or social conditions that may interfere with the patient's participation in the study or evaluation of the study results.
  • Use of strong CYP3A4 inhibitors and inducers from 14 days prior to first administration of study drug. Strong CYP3A4 inhibitors and inducers are prohibited during the study and until the active follow up visit.
  • Clinically relevant findings in the ECG.

Trial locations and contacts

4 sites in 1 country.

Canada4 sites
  • Cross Cancer Institute

    Edmonton, Alberta, T6G 1Z2

  • CR Centre Hospitalier de l'Université de Montréal - CHUM

    Montreal, Quebec, H2X 0A9

  • Princess Margaret Hospital

    Toronto, Ontario, M5G 2M9

  • BC Cancer - Vancouver

    Vancouver, British Columbia, V5Z4E6

Other trials in Canada

About this study

Official title: Phase I Trial of PCLX-001 in Relapsed/Refractory B-cell Non-Hodgkin Lymphoma and Advanced Solid Malignancies

This is a phase I dose-escalation study of oral PCLX-001, conducted in a multicenter, non-randomized, open-label, non-controlled design. The study is comprised of two parts: Part A (single-agent dose escalation) and Part B (single-agent expansion cohorts).

Read the detailed description

This is a phase I dose-escalation study of oral PCLX-001, conducted in a multicenter, non-randomized, open-label, non-controlled design. The study is comprised of two parts: Part A (single-agent dose escalation) and Part B (single-agent expansion cohorts).

For Part A dose-escalation, patients will be enrolled in cohorts of 3 to 6 patients to each dose level. A new dose level cannot open to accrual until toxicity has been determined in the preceding dose level (i.e. all patients have completed their first cycle of therapy and data for all patients in that dose level have been reviewed at a safety cohort review meeting). Six patients will be treated at the maximum tolerated dose (MTD) and/or recommended phase II dose (RP2D). If required, the MTD cohort may be expanded by an additional 10 patients for further toxicity and response assessment. The MTD cohort expansion may be restricted to B-cell lymphoma or advanced solid tumours to ensure there is proper distribution during dose escalation.

For Part B (single agent expansion cohorts), two expansion cohorts (N=20 each) will be opened to determine the preliminary clinical activity of PCLX-001 at the RP2D:

  • Expansion Cohort A: Participants with advanced solid malignancies showing preclinical sensitivity or molecular markers of sensitivity to PCLX-001. This includes breast, nonsmall cell lung (NSCLC), small-cell lung (SCLC), colorectal (CRC), and bladder cancers
  • Expansion Cohort B: Participants with relapsed/refractory (R/R) B-cell lymphoma: diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma (HGBL), follicular lymphoma (FL), mantle cell lymphoma (MCL), and Burkitt lymphoma. Transformed large B-cell lymphoma will also be included.
Being tested
PCLX-001 - 20mg, PCLX-001 - 40mg, PCLX-001 - 70mg, PCLX-001 - 100mg, PCLX-001 - 140mg
Medical subject headings
Lymphoma, B-Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PCLX-001 - 20mgDrug

    Also called zelenirstat

    20mg daily oral pills

  • PCLX-001 - 40mgDrug

    Also called zelenirstat

    40mg daily oral pills

  • PCLX-001 - 70mgDrug

    Also called zelenirstat

    70mg daily oral pills

  • PCLX-001 - 100mgDrug

    Also called zelenirstat

    100mg daily oral pills

  • PCLX-001 - 140mgDrug

    Also called zelenirstat

    140mg daily oral pills

  • PCLX-001 - 210mgDrug

    Also called zelenirstat

    210mg daily oral pills

  • PCLX-001 - 280mgDrug

    Also called zelenirstat

    280mg daily oral pills

Study groups in NCT04836195
Study groupRoleWhat they receive
PCLX-001 intervention 20mgExperimentalCohort 1: Participants were administered 20mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 20mg
PCLX-001 intervention 40mgExperimentalCohort 2: Participants were administered 40mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 40mg
PCLX-001 intervention 70mgExperimentalCohort 3: Participants were administered 70mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 70mg
PCLX-001 intervention 100mgExperimentalCohort 4: Participants were administered 100mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 100mg
PCLX-001 intervention 140mgExperimentalCohort 5: Participants were administered 140mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 140mg
PCLX-001 intervention 210mgExperimentalCohort 6: Participants were administered 210mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 210mg
PCLX-001 intervention 280mgExperimentalCohort 7: Participants were administered 280mg of PCLX-001 orally as continuous daily dosing on a 28-day cycle.
Receives: Drug: PCLX-001 - 280mg

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To Determine, During the Dose Escalation Phase, the Recommended Dose of PCLX-001 for the Dose Expansion Phase of the Trial.

    The recommended dose will be the dose level below that for the cohort in which maximum tolerated dose (MTD) was reached/exceeded. MTD will have been reached when 2 or more patients in a cohort experience DLT.

    Measured: Through study completion, an average of ~90 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Apr 2021
  2. Study start14 Sep 2021
  3. Primary completion28 Oct 2024
  4. Study completion28 Oct 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Model description
For Part A dose-escalation, patients will be enrolled in cohorts of 3 to 6 patients to each dose level. Six patients will be treated at the maximum tolerated dose (MTD) and/or recommended phase II dose (RP2D).
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04836195
Sponsor's protocol ID
PCLX-001-01
Status last verified
Mar 2025
First submitted
29 Mar 2021
Last updated
17 Apr 2025
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Beauchamp E, Yap MC, Iyer A, Perinpanayagam MA, Gamma JM, Vincent KM, Lakshmanan M, Raju A, Tergaonkar V, Tan SY, Lim ST, Dong WF, Postovit LM, Read KD, Gray DW, Wyatt PG, Mackey JR, Berthiaume LG. Targeting N-myristoylation for therapy of B-cell lymphomas. Nat Commun. 2020 Oct 22;11(1):5348. doi: 10.1038/s41467-020-18998-1.

    PubMed 33093447
  • Sangha R, Jamal R, Spratlin J, Kuruvilla J, Sehn LH, Beauchamp E, Weickert M, Berthiaume LG, Mackey JR. A first-in-human phase I trial of daily oral zelenirstat, a N-myristoyltransferase inhibitor, in patients with advanced solid tumors and relapsed/refractory B-cell lymphomas. Invest New Drugs. 2024 Aug;42(4):386-393. doi: 10.1007/s10637-024-01448-w. Epub 2024 Jun 5.

    PubMed 38837078

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing PCLX-001 - 20mg, PCLX-001 - 40mg and PCLX-001 - 70mg in people with B-cell Non Hodgkin Lymphoma and Advanced Solid Tumor, run by Pacylex Pharmaceuticals at 4 sites in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To Determine, During the Dose Escalation Phase, the Recommended Dose of PCLX-001 for the Dose Expansion Phase of the Trial. (time frame: Through study completion, an average of ~90 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04836195, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2025.

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