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Burkitt LymphomaPhase 1/2NCT07069699

Testing the Safety of Anti-Cancer Drug, CX-5461 (Pidnarulex), in Treating Lymphoma With Specific Changes in the MYC Gene

A Phase 1/2 study testing Biopsy Procedure, Biospecimen Collection and Computed Tomography in people with Burkitt Lymphoma and Double-Expressor Lymphoma, run by National Cancer Institute (NCI) at 8 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This phase Ib/II trial tests the safety, side effects, best dose and how well giving CX-5461 works for the treatment of patients with B-cell non-Hodgkin lymphoma. CX-5461 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biopsy Procedure, Biospecimen Collection and Computed Tomography
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
8 sitesUnited States
Study dates
11 Jan 2027to 31 Jan 2030
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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

  • Patients must have one of the following subtypes of aggressive B-cell non-Hodgkin lymphomas: double-expressor lymphoma (DEL), high-grade B-cell lymphoma (HGBL) with MYC and BCL2 and/or BCL6 rearrangement, or Burkitt lymphoma (BL). Eligible patients must have received at least two prior lines of treatment for diffuse large B-cell lymphoma (DLBCL) or at least one prior line of therapy for Burkitt Lymphoma and must have disease for which no standard curative or palliative treatment options exist or remain effective (Quin et al., 2016)
  • Age ≥ 18 years. Because no dosing or adverse event (AE) data are currently available on the use of CX-5461 (Pidnarulex) in patients < 18 years of age, children are excluded from this study
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 60%). ECOG 3 is allowed if directly related to lymphoma per treating provider
  • Absolute neutrophil count ≥ 1,000/mcL
  • Platelets ≥ 50,000/mcL
  • Total bilirubin ≤ 1.5 institutional upper limit of normal (ULN)
Show all 19Show fewer
  • Patients with documented Gilbert's syndrome may be included if total bilirubin is ≤ 3 × ULN and direct bilirubin is within normal limits
  • Aspartate aminotransferase (AST)(serum glutamic oxaloacetic transaminase [SGOT])/ alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT]) ≤ 3 × institutional upper limit of normal (ULN)
  • Glomerular filtration rate (GFR) ≥ 60 mL/min/, calculated by multiplying the estimated (e)GFR (mL/min/1.73 m^2) by the individual's body surface area (BSA, calculated using an accepted formula) and dividing by 1.73 m^2
  • Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
  • For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
  • Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
  • Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
  • Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class II or better
  • Patients with cytopenia related to abnormal bone marrow function in the setting of bone marrow involvement with lymphoma or post chimeric antigen receptor (CAR) T-cell are allowed to enroll if deemed safe by treating provider
  • Patients without clinical evidence of central nervous system (CNS) lymphoma
  • The effects of CX-5461 (Pidnarulex) on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) 14 days prior to study entry and for the duration of study participation and for at least 6 months after the last dose of study drug. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women should not breastfeed while taking CX-5461 (Pidnarulex) and for 6 months after cessation of treatment. Men treated or enrolled on this protocol must also agree to use adequate contraception 14 days prior to the study, for the duration of study participation, and 6 months after completion of CX-5461 (Pidnarulex) administration. Women of childbearing age should not donate egg(s) and men should not donate sperm for the duration of study participation and 6 months after completion of the last dose CX-5461 (Pidnarulex)
  • Willingness to provide blood and biopsy samples for research purposes
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Patients must have recovered from clinically significant adverse events (AEs) of their most recent cancer immunotherapy to grade 1 or less (with the exception for alopecia or lymphopenia)
  • Eligibility of subjects receiving any medications or substances known to affect or with the potential to affect the activity of CX-5461 (Pidnarulex) will be determined based on their potential to interact with the CYP3A4 isozyme. Specifically, subjects taking strong CYP3A4 inhibitors or strong CYP3A4 inducers will be excluded from participation in the trial. For medications or substances not listed, or in cases of uncertainty, the Principal Investigator may consult with a medical expert or a pharmacologist to make an informed decision regarding eligibility
  • Patients with a baseline corrected QT (QTc) interval > 480 msec
  • Patients who are receiving any other investigational agents
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to CX-5461 (Pidnarulex)
  • Patients with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous
  • Pregnant women are excluded from this study because CX-5461 (Pidnarulex) is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with CX-5461 (Pidnarulex), breastfeeding should be discontinued if the mother is treated with CX-5461 (Pidnarulex)

Trial locations and contacts

8 sites in 1 country. Contact the study team or a site near you to ask about taking part.

8 sites in 1 country

United States8 sites, 8 recruiting

  • University of Kansas Clinical Research Center

    Fairway, Kansas, 66205

    Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu

    Aung M. Tun

    Recruiting

  • UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care

    Irvine, California, 92612

    Site Public Contact, 877-827-8839, ucstudy@uci.edu

    Nicole Foley

    Recruiting

  • University of Kansas Cancer Center

    Kansas City, Kansas, 66160

    Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu

    Aung M. Tun

    Recruiting

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

    Site Public Contact, 405-271-8777, ou-clinical-trials@ouhsc.edu

    Sami Ibrahimi

    Recruiting

  • UC Irvine Health/Chao Family Comprehensive Cancer Center

    Orange, California, 92868

    Site Public Contact, 877-827-8839, ucstudy@uci.edu

    Nicole Foley

    Recruiting

  • University of Kansas Hospital-Indian Creek Campus

    Overland Park, Kansas, 66211

    Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu

    Aung M. Tun

    Recruiting

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

    Site Public Contact, 412-647-8073

    Jing-Zhou Hou

    Recruiting

  • University of Kansas Hospital-Westwood Cancer Center

    Westwood, Kansas, 66205

    Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu

    Aung M. Tun

    Recruiting

Other trials in United States

About this study

Official title: Phase 1b/2 Trial of Pidnarulex in MYC Aberrant Lymphoma

This phase Ib/II trial tests the safety, side effects, best dose and how well giving CX-5461 works for the treatment of patients with B-cell non-Hodgkin lymphoma. CX-5461 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CX-5461 may be safe, tolerable and/or effective in treating patients with B-cell non-Hodgkin lymphoma.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To define dose limiting toxicity (DLT). (Phase 1) II. To assess toxicity profile. (Phase 1) III. To determine the recommended phase 2 dose (RP2D). (Phase 1) IV. To perform pharmacokinetic (PK) studies. (Phase 1) V. To examine the effect of CX-5461 (Pidnarulex) on gene expression profile of MYC aberrant lymphomas. (Phase 1) VI. To determine the objective response rate (ORR) (complete response [CR] and partial response [PR]). (Phase 2)

SECONDARY OBJECTIVE:

  1. To observe and record anti-tumor activity. (Phase 1 and 2)

EXPLORATORY OBJECTIVES:

  1. To determine the mechanisms of response and resistance using circulating tumor deoxyribonucleic acid (ctDNA). (Phase 1 and 2) II. To observe cerebral spinal fluid (CSF) distribution of CX-5461 (Pidnarulex). (Phase 1 and 2)

OUTLINE: This is a phase 1 dose-escalation study of CX-5461 followed by a phase 2 study.

Patients receive CX-5461 intravenously (IV) over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo lumbar puncture with cerebrospinal fluid (CSF) collection on study and positron emission tomography (PET) scan/computed tomography (CT) scan, tumor biopsy, and blood sample collection throughout the study.

After completion of study treatment, patients are followed up at 30 days, every 3-4 months for the first 2 years, and every 6 months for years 3-5.

Being tested
Biopsy Procedure, Biospecimen Collection, Computed Tomography, Lumbar Puncture, Pidnarulex
Medical subject headings
Burkitt Lymphoma

What participants receive

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

  • Biopsy ProcedureProcedure or surgery

    Also called Biopsy, BIOPSY_TYPE, Bx

    Undergo tumor biopsy

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo CSF and blood sample collection

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo PET/CT scan

  • Lumbar PunctureProcedure or surgery

    Also called LP, Spinal Tap

    Undergo lumbar puncture

  • PidnarulexDrug

    Also called CX-5461, CX5461, Pol I Inhibitor CX5461, RNA Pol I Inhibitor CX5461

    Given IV

  • Positron Emission TomographyProcedure or surgery

    Also called Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure)

    Undergo PET/CT scan

Study groups in NCT07069699
Study groupRoleWhat they receive
Treatment (CX-5461)ExperimentalPatients receive CX-5461 IV over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo lumbar puncture with CSF collection on study and PET scan/CT scan, tumor biopsy, and blood sample collection throughout the study.
Receives: Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Procedure: Computed Tomography; Procedure: Lumbar Puncture; Drug: Pidnarulex; Procedure: Positron Emission Tomography

What is being measured

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

  1. Incidence of adverse events (Phase 1)

    Assessed by the Common Terminology Criteria for Adverse Events version 5. Safety will be summarized overall, by actual dose level in Phase 1b.

    Measured: Up to 30 days after last dose of study treatment

  2. Recommended phase 2 dose (RP2D) (Phase 1)

    Measured: Up to 5 years

  3. Pharmacokinetic (PK) levels (Phase 1)

    Assessed using Liquid Chromatography Mass Spectrometry (LC-MS).

    Measured: At cycle (C) 1 day (D) 8 at pre-dose, end of infusion, and 2 hours (hrs) post-dose; on C1D9 at 24 hrs post C1D8 infusion; on C1D10 at 48 hrs post C1D8 infusion; on C1D11 at 72 hrs post C1D8 infusion; and on C1D15 at 167 hrs post C1D8 infusion

  4. Gene expression (Phase 1)

    Assessed by ribonucleic acid sequencing complemented by whole exome sequencing using an average absolute log2 fold change relative to baseline with a significance threshold of p < 0.05.

    Measured: At baseline, C1D9 or C1D10 (within 24-48 hrs of C1D8 dosing),and at progression

  5. Overall response rate (Phase 2)

    Assessed using standard radiographic criteria according to Lugano criteria. Phase 2 results will be reported at the RP2D.

    Measured: Up to 5 years

Secondary outcomes (1)

  1. Response to therapy (Phase 1 and 2)

    Assessed according to Lugano criteria.

    Measured: Up to 5 years

Timeline

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

  1. First posted17 Jul 2025
  2. Study start11 Jan 2027 (estimated)
  3. Primary completion31 Jan 2030 (estimated)
  4. Study completion31 Jan 2030 (estimated)

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07069699
Sponsor's protocol ID
NCI-2025-04787
Other IDs
NCI-2025-04787, 10717, 10717, UM1CA186689
Status last verified
Oct 2026
First submitted
16 Jul 2025
Last updated
14 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Biopsy Procedure, Biospecimen Collection and Computed Tomography in people with Burkitt Lymphoma and Double-Expressor Lymphoma, run by National Cancer Institute (NCI) at 8 sites in United States.

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 19 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events (Phase 1) (time frame: Up to 30 days after last dose of study treatment). The study lists 5 primary outcomes in all. 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 NCT07069699.

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 14 Oct 2026.

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