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CancerPhase 1APX3330NCT03375086

A Study of APX3330 in Patients With Advanced Solid Tumors

A Phase 1 study testing APX3330 in people with Cancer, run by Apexian Pharmaceuticals, Inc. at 3 sites in United States.

Completed

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

Official record
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Trial summary

This is a Phase 1, multi-center, open-label, dose-escalation oncology study of APX3330 in patients with advanced solid tumors. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cancer
What is being tested?
APX3330
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
3 sitesUnited States
Study dates
30 Jan 2018to 9 Jan 2019
Phase
Phase 1
Are there results?
None posted in the registry

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

  • Written informed consent must be obtained from the patient.
  • Patient must be > 18 years of age.
  • Patient must have recurrent or advanced cancer (i.e., solid tumors) for whom standard therapy offers no curative potential.
  • Evaluable disease by RECIST v1.1.
  • Performance status (PS) of 0-2 on the Eastern Cooperative Oncology Group (ECOG) scale. Note: PS 2 patients can only participate if, in the assessment of the clinical investigator, and with the consent of the medical monitor, the patient has the ability to participate in the clinical study for a minimum of at least 2 cycles.
  • > 21 days from therapeutic radiation or chemotherapy (>6 weeks from nitrosoureas and mitomycin C) and recovery to (NCI CTCAE v4.03) Grade ≤ 1 from all clinically significant toxicities related to prior therapies.
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  • Must have adequate organ function defined as:
  • Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L.
  • Platelet ≥ 100 x 10^9/L.
  • Hemoglobin ≥ 9 g/dL.
  • Activated partial thromboplastin time/ partial thromboplastin time (aPTT/PTT) ≤ 1.5 x ULN
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤ 2.5 × upper limit of normal (ULN). In the case of known (i.e., radiological or biopsy documented) liver metastasis, serum transaminase levels must be < 5 x ULN.
  • Total serum bilirubin ≤ 1.5 x ULN, (except for patients with known Gilbert's Syndrome ≤ 3 x ULN is permitted)
  • Renal: Serum creatinine < 2.0 x ULN or creatinine clearance ≥ 50 mL/min/1.73m^2 for patients with serum creatinine levels above 2 x ULN.
  • Agreement to use acceptable methods of contraception during the study and for at least 120 days after the last dose of APX3330 if sexually active and able to bear or beget children.

Who cannot join

  • Diagnosed with another malignancy within the past 2 years (excluding a history of carcinoma in situ of the cervix, superficial non-melanoma skin cancer, superficial bladder cancer, or endometrial cancer that has been adequately treated, or stage 1 prostate cancer that does not require treatment).
  • History of a major surgical procedure or a significant traumatic injury within 14 days prior to commencing treatment, or the anticipation of the need for a major surgical procedure during the course of the study.
  • Patients who have been treated with an investigational agent within 21 days prior to the first dose of study drug.
  • Concurrent serious (as determined by the Principal Investigator) medical conditions, including, but not limited to, New York Heart Association (NYHA) class III or IV congestive heart failure, history of congenital prolonged QT syndrome, uncontrolled infection, active hepatitis B, C or HIV, or other significant co-morbid conditions that in the opinion of the investigator would impair study participation or cooperation.
  • Impaired liver function Child-Pugh class B or C (score 7-15).
  • Women who are pregnant or lactating.
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  • Patients with evidence of symptomatic brain metastases. Patients with treated (surgically excised or irradiated) and stable brain metastases are eligible assuming the patient has adequately recovered from treatment, the treatment was at least 28 days prior to initiation of study drug, and baseline brain computed tomography (CT) with contrast, or magnetic resonance imaging (MRI) within 14 days of initiation of study drug, is negative for new or worsening brain metastases
  • Other concurrent chemotherapy, immunotherapy, radiotherapy or investigational therapy except for hormonal therapy (e.g., tamoxifen, etc.).
  • Patients requiring palliative radiotherapy to lesions that are defined as target lesions by RECIST criteria at the time of study entry.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • START Midwest

    Grand Rapids, Michigan, 49546

  • Indiana University Simon Cancer Center

    Indianapolis, Indiana, 46202

  • START San Antonio

    San Antonio, Texas, 78229

Other trials in United States

About this study

Official title: A Phase 1 Study of APX3330 in Patients With Advanced Solid Tumors

This is a Phase 1, multi-center, open-label, dose-escalation oncology study of APX3330 in patients with advanced solid tumors.

Read the detailed description

Apurinic/apyrimidinic endonuclease 1/redox factor-1 (APE1/Ref-1) is a protein that regulates multiple transcription factors involved in cancer cell signaling and APX3330 is a highly selective inhibitor of APE1/Ref-1 redox function.

The anti-tumor effect of APX330 has been demonstrated in a variety of preclinical models and the human safety profile of APX3330 was established in prior clinical studies. Apexian Pharmaceuticals is developing APX3330 as an orally administered anti-cancer agent targeting the APE1/Ref-1 protein.

APX_CLN_0011 is a Phase 1, multi-center, open-label, dose-escalation oncology study in patients with advanced solid tumors. The study primary objective is to determine the recommended Phase 2 study dose of APX3330. Secondary objectives include assessment of APX3330 safety, anti-tumor activity, pharmacokinetic and pharmacodynamic profile.

Conditions
Cancer
Being tested
APX3330
Medical subject headings
Neoplasms

What participants receive

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

  • APX3330Drug

    Also called E3330

    APX3330 will be supplied as 60 and 120 mg orally administered tablets. Patients will receive a fixed dose of APX3330 twice daily (i.e., bid) each day of a 21-day cycle. The starting dose of APX3330 will be a daily dose of 240 mg (i.e., 120 mg/dose bid). Doses will be increased until identification of a maximum tolerated dose or bio-effective dose, whichever is lower.

Study groups in NCT03375086
Study groupRoleWhat they receive
Single armExperimentalPatients will receive APX3330 orally, twice per day until disease progression
Receives: Drug: APX3330

What is being measured

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

  1. Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0

    Identification of the safety and tolerability of APX3330

    Measured: 12 months

Secondary outcomes (1)

  1. Any anti-tumor activity that may occur in patients receiving APX3330 using RECIST criteria

    Using RECIST criteria

    Measured: 18 months

Timeline

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

  1. First posted15 Dec 2017
  2. Study start30 Jan 2018
  3. Primary completion18 Aug 2018
  4. Study completion9 Jan 2019

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
Not applicable
Intervention model
Single group
Model description
Open-label, multi-center, dose escalation.
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03375086
Sponsor's protocol ID
APX_CLN_0011
Acronym
APX3330
Status last verified
Sep 2020
First submitted
21 Nov 2017
Last updated
7 Sep 2020
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Shah F, Logsdon D, Messmann RA, Fehrenbacher JC, Fishel ML, Kelley MR. Exploiting the Ref-1-APE1 node in cancer signaling and other diseases: from bench to clinic. NPJ Precis Oncol. 2017;1:19. doi: 10.1038/s41698-017-0023-0. Epub 2017 Jun 8.

    PubMed 28825044

  • Jiang A, Gao H, Kelley MR, Qiao X. Inhibition of APE1/Ref-1 redox activity with APX3330 blocks retinal angiogenesis in vitro and in vivo. Vision Res. 2011 Jan;51(1):93-100. doi: 10.1016/j.visres.2010.10.008. Epub 2010 Oct 14.

    PubMed 20937296

  • Safi Shahda, Nehal J. Lakhani, Bert O'Neil, Drew W. Rasco, Jun Wan, Amber L Mosley, Hao Liu, Mark R. Kelley, Richard Adam Messmann. A phase I study of the APE1 protein inhibitor APX3330 in patients with advanced solid tumors. Journal of Clinical Oncology. Volume 37: Issue 15_suppl.

  • Kelley MR, Wan J, Liu S, Kpenu E, Wireman R, Mosley AL, Liu H, Lakhani NJ, Shahda S, O'Neil B, Opyrchal M, Messmann RA. A phase I study targeting the APE1/Ref-1 redox signaling protein with APX3330: first clinical agent targeting APE1/Ref-1 in cancer. Oncologist. 2026 Jan 17;31(2):oyaf423. doi: 10.1093/oncolo/oyaf423.

    PubMed 41423797

  • Kelley MR, Wan J, Liu S, Kpenu E, Wireman R, Mosley AL, Liu H, Lakhani NJ, Shahda S, O'Neil B, Opyrchal M, Messmann RA. A Phase I study targeting the APE1/Ref-1 redox signaling protein with APX3330: First clinical agent targeting APE1/Ref-1 in Cancer. medRxiv [Preprint]. 2025 Apr 4:2025.04.03.25325173. doi: 10.1101/2025.04.03.25325173.

    PubMed 40236444

  • Lalunio H, Stupka N, Goodman CA, Hayes A. The Potential of Targeting APE1/Ref-1 as a Therapeutic Intervention for Duchenne Muscular Dystrophy. Antioxid Redox Signal. 2025 May;42(13-15):641-654. doi: 10.1089/ars.2024.0620. Epub 2024 Dec 27.

    PubMed 39729027

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing APX3330 in people with Cancer, run by Apexian Pharmaceuticals, Inc. at 3 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 15 inclusion and 9 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 7 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 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 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 (time frame: 12 months). 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 NCT03375086, which also lists 6 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 7 Sep 2020.

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