40 Participants Needed

Pidnarulex for Cancer

Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: National Cancer Institute (NCI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

This phase I trial tests the safety, side effects, and best dose of pidnarulex (CX-5461) in treating patients with solid tumors that have spread from where it first started (primary site) to other places in the body (metastatic). Pidnarulex is an oral inhibitor of ribonucleic acid polymerase I, with potential antineoplastic activity. It blocks a certain enzyme needed for cell division and deoxyribonucleic acid (DNA) repair and may kill cancer cells.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you cannot participate if you are taking strong CYP3A4 inhibitors or inducers, as they may interact with the study drug. It's best to discuss your current medications with the trial team to determine eligibility.

What data supports the effectiveness of the drug Pidnarulex for cancer?

The research does not provide direct evidence about Pidnarulex, but it highlights the effectiveness of paclitaxel, a similar cancer drug, which has shown significant activity in various cancers, including ovarian and breast cancer. Paclitaxel's success in combination therapies suggests that Pidnarulex might also be effective when used with other agents.12345

Is Pidnarulex safe for humans?

Pidnarulex, also known as CX-5461, has been shown to cause significant genetic changes in human cells, which could be harmful. This suggests potential safety concerns, as these changes are more extensive than those caused by known environmental cancer-causing agents.678910

Research Team

AP

A P Chen

Principal Investigator

National Cancer Institute LAO

Eligibility Criteria

This trial is for adults with solid tumors that have spread and worsened after at least one prior treatment. They must have a tumor measurable by medical imaging, be able to undergo a biopsy, and be in decent physical shape (able to perform light activities). Blood counts and liver function need to meet specific levels.

Inclusion Criteria

My total bilirubin levels are within the normal range.
I have Gilbert's syndrome with a bilirubin level up to 3 mg/dl.
My cancer has spread and worsened after at least one treatment.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment

Patients receive pidnarulex IV over 60 minutes on days 1 and 8 of each 28-day cycle. Patients also undergo CT or MRI, biopsy, and collection of blood samples throughout the trial.

Up to 3 years
2 visits per cycle (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

4 weeks
1 visit (in-person)

Treatment Details

Interventions

  • Pidnarulex
Trial Overview The trial is testing the safety, side effects, and best dose of Pidnarulex (CX-5461), an oral drug designed to block enzymes involved in cell division and DNA repair. This could potentially kill cancer cells. The study includes tests like echocardiography, MRI, CT scans, biopsies, and biospecimen collection.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Treatment (pidnarulex)Experimental Treatment6 Interventions
Patients receive pidnarulex IV over 60 minutes on days 1 and 8 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo CT or MRI, biopsy, and collection of blood samples throughout the trial. Patients may undergo ECHO at screening and then as clinically indicated.

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Findings from Research

Panobinostat, a histone deacetylase inhibitor, shows significant synergy with gemcitabine and paclitaxel, and particularly strong synergistic effects when combined with doxorubicin and carboplatin in treating metastatic ovarian cancer cell lines.
The study utilized in vitro assays on three ovarian cancer cell lines (SK-OV3, CaOV-3, and ES-2) to identify effective drug combinations, suggesting that these combinations could enhance treatment efficacy and warrant further clinical investigation.
The histone deacetylase inhibitor panobinostat demonstrates marked synergy with conventional chemotherapeutic agents in human ovarian cancer cell lines.Budman, DR., Tai, J., Calabro, A., et al.[2021]
In a study of 138 patients with relapsing epithelial ovarian cancer, paclitaxel treatment resulted in a 28% overall response rate, with 14% achieving a complete response, indicating its potential efficacy as a second-line treatment.
The treatment was well-tolerated with no serious toxicity reported, and patients with platinum-sensitive tumors and good performance status had significantly better survival outcomes, highlighting the importance of patient selection for optimal results.
Long-term results from a phase II study of single agent paclitaxel (Taxol) in previously platinum treated patients with advanced ovarian cancer: the Nordic experience.Tropé, C., Hogberg, T., Kaern, J., et al.[2020]
The novel paclitaxel-aminoguanidine conjugate significantly improves water solubility compared to paclitaxel alone, enhancing its potential effectiveness against metastatic breast cancer.
This conjugate not only releases both paclitaxel and aminoguanidine within cancer cells but also demonstrates stronger cytotoxicity, increased apoptosis, and superior anti-metastatic effects compared to either drug used separately.
Synthesis and Biological Evaluation of Paclitaxel-aminoguanidine Conjugates for Suppressing Breast Cancer.Dai, Y., Zhang, Y., Zhang, L., et al.[2023]

References

The histone deacetylase inhibitor panobinostat demonstrates marked synergy with conventional chemotherapeutic agents in human ovarian cancer cell lines. [2021]
Long-term results from a phase II study of single agent paclitaxel (Taxol) in previously platinum treated patients with advanced ovarian cancer: the Nordic experience. [2020]
3.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Synthesis and Biological Evaluation of Paclitaxel-aminoguanidine Conjugates for Suppressing Breast Cancer. [2023]
Paclitaxel (Taxol®): A new natural product with major anticancer activity. [2012]
Virtual clinical trials identify effective combination therapies in ovarian cancer. [2021]
The chemotherapeutic drug CX-5461 is a potent mutagen in cultured human cells. [2023]
Risk of serious adverse event and fatal adverse event with molecular target anticancer drugs in cancer patients: A meta-analysis. [2020]
MetaADEDB 2.0: a comprehensive database on adverse drug events. [2021]
ADReCS-Target: target profiles for aiding drug safety research and application. [2019]
Real-world safety profiles of pirfenidone and nintedanib in idiopathic pulmonary fibrosis patients. [2022]
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