Trial Outcomes & Findings for Safety, Dose Tolerance, Pharmacokinetics, and Pharmacodynamics Study of CPX-POM in Patients With Advanced Solid Tumors (NCT NCT04608045)

Last Updated: 2025-03-03

Results Overview

Tumors will be assessed in a standard manner and given grade/stage according to the American Joint Commission on Cancer (AJCC) criteria. Efficacy will be assessed based on pathologic criteria and evidence of pharmacologic activity in the target tissue.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

9 participants

Primary outcome timeframe

56 days

Results posted on

2025-03-03

Participant Flow

CPX-POM-001EXP (NCT04608045) was submitted to and approved by FDA as a protocol amendment to CPX-POM-001 (dose escalation study, NCT03348514), the Phase 1 Dose Escalation Study. The dosage regimen was determined to be the Recommended Phase 2 Dose (RP2D) defined by the CPX-POM-001 Dose Escalation Study (NCT03348514) conducted in advanced solid tumor patients. The nine patients enrolled in CPX-POM-001EXP (NCT04608045) received one dosage regimen of 900 mg/m\^2 via a 20-minute infusion.

CPX-POM-001EXP was conducted in muscle-invasive bladder cancer (MIBC) patients scheduled for cystectomy. MIBC, cisplatin-ineligible MIBC and cisplatin-eligible MIBC patients received fosciclopirox for 2 or 3, 21-day treatment, fosciclopirox alone prior to cystectomy for 2, 21-day treatment cycles or fosciclopirox for 3, 21-day treatment cycles in combination with standard-of-care gemcitabine and cisplatin prior to cystectomy. The dosage regimen was administered in a neoadjuvant setting.

Participant milestones

Participant milestones
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion CPX-POM: CPX-POM
Overall Study STARTED	9
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion CPX-POM: CPX-POM
Overall Study Physician Decision	1

Baseline Characteristics

Safety, Dose Tolerance, Pharmacokinetics, and Pharmacodynamics Study of CPX-POM in Patients With Advanced Solid Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=9 Participants CPX-POM: CPX-POM
Age, Continuous	68.6 years STANDARD_DEVIATION 11.06 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 56 days

Outcome measures

Outcome measures
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=8 Participants CPX-POM: CPX-POM	Post-treatment: CPX-POM, 900 mg/m^2 by 20-minute IV Infusion Post-treatment
Determine Disease Response Following 2 or 3 CPX-POM Treatment Cycles by Assessing the Complete and Partial Pathologic Response Rate at the Time of Radical Cystectomy (RC) Complete pathological response	2 Participants	—
Determine Disease Response Following 2 or 3 CPX-POM Treatment Cycles by Assessing the Complete and Partial Pathologic Response Rate at the Time of Radical Cystectomy (RC) Pathologic downstaging	4 Participants	—

PRIMARY outcome

Timeframe: 56 days

Immunohistochemistry (IHC) analyses of pre-treatment (at TURBT) and post-treatment (at radical cystectomy) bladder tumor tissues were performed to determine whether neoadjuvant fosciclopirox treatment affected the Notch cell signaling pathway. Immunohistochemistry staining intensity score as a measure of protein expression with four categories: negative (0), weak (1), moderate (2), and strong (3). Staining intensity scores were assigned by a blinded pathologist who specializes in genitourinary cancers. Pre-treatment and post-treatment bladder tumor samples were available for IHC analysis for six of the nine patients studied. For two patients, pre-treatment bladder tumor tissue samples obtained from TURBT at referring sites were not processed for IHC analysis. One patient was discovered to have metastatic MIBC after enrollment, and therefore, did not undergo RC, hence, no post-treatment sample was available for IHC analysis.

Outcome measures

Outcome measures
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=6 Participants CPX-POM: CPX-POM	Post-treatment: CPX-POM, 900 mg/m^2 by 20-minute IV Infusion n=6 Participants Post-treatment
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Notch 1	2.2 score on a scale Standard Deviation 0.8	1.3 score on a scale Standard Deviation 1.5
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Notch 2	1.0 score on a scale Standard Deviation 0.9	0.2 score on a scale Standard Deviation 0.4
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Notch 3	2.5 score on a scale Standard Deviation 1.2	1.5 score on a scale Standard Deviation 1.6
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Jagged 1	2.0 score on a scale Standard Deviation 1.3	1.0 score on a scale Standard Deviation 1.5
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Presenilin 1	1.3 score on a scale Standard Deviation 1.5	1.0 score on a scale Standard Deviation 1.3
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Nicastrin	2.5 score on a scale Standard Deviation 0.2	1.5 score on a scale Standard Deviation 1.6
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Hes 1	1.5 score on a scale Standard Deviation 1.2	0.5 score on a scale Standard Deviation 1.2
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. Cyclin D1	0.0 score on a scale Standard Deviation 0.0	0.2 score on a scale Standard Deviation 0.4
Immunohistochemistry (IHC) Analyses of Pre-treatment (at TURBT) and Post-treatment (at Radical Cystectomy) Bladder Tumor Tissues Were Performed to Determine Whether Neoadjuvant Fosciclopirox Treatment Affected the Notch Cell Signaling Pathway. CD8+	1.5 score on a scale Standard Deviation 0.5	0.5 score on a scale Standard Deviation 0.5

SECONDARY outcome

Timeframe: 56 days

Population: Safety population

Incidence of Serious Adverse Events in subjects receiving CPX-POM.

Outcome measures

Outcome measures
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=9 Participants CPX-POM: CPX-POM	Post-treatment: CPX-POM, 900 mg/m^2 by 20-minute IV Infusion Post-treatment
Number of Participant With Any Serious Adverse Events (SAEs) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 4.03)	0 Participants	—

SECONDARY outcome

Timeframe: 56 days

Population: Safety population

Incidence of Adverse Events in subjects receiving CPX-POM.

Outcome measures

Outcome measures
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=9 Participants CPX-POM: CPX-POM	Post-treatment: CPX-POM, 900 mg/m^2 by 20-minute IV Infusion Post-treatment
Number of Participant With Any Adverse Events (AEs) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 4.03)	9 Participants	—

Adverse Events

CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	CPX-POM, 900 mg/m^2 by 20 Minute IV Infusion n=9 participants at risk CPX-POM: CPX-POM
Gastrointestinal disorders Nausea	55.6% 5/9 • 56 days
Gastrointestinal disorders Constipation	33.3% 3/9 • 56 days
Gastrointestinal disorders Abdominal Distension	22.2% 2/9 • 56 days
Gastrointestinal disorders Abdominal Pain	22.2% 2/9 • 56 days
Gastrointestinal disorders Gastrooesophageal reflux disease	22.2% 2/9 • 56 days
Gastrointestinal disorders Vomiting	22.2% 2/9 • 56 days
Gastrointestinal disorders Diarrhoea	11.1% 1/9 • 56 days
Gastrointestinal disorders Epigastric Discomfort	11.1% 1/9 • 56 days
Gastrointestinal disorders Toothache	11.1% 1/9 • 56 days
General disorders Fatigue	66.7% 6/9 • 56 days
General disorders Gait disturbance	11.1% 1/9 • 56 days
General disorders Non-cardiac chest pain	11.1% 1/9 • 56 days
General disorders Peripheral swelling	11.1% 1/9 • 56 days
General disorders Pyrexia	11.1% 1/9 • 56 days
Renal and urinary disorders Haematuria	33.3% 3/9 • 56 days
Renal and urinary disorders Dysuria	22.2% 2/9 • 56 days
Renal and urinary disorders Micturition urgency	22.2% 2/9 • 56 days
Renal and urinary disorders Bladder spasm	11.1% 1/9 • 56 days
Renal and urinary disorders Hydronephrosis	11.1% 1/9 • 56 days
Renal and urinary disorders Pollakiuria	11.1% 1/9 • 56 days
Renal and urinary disorders Renal failure	11.1% 1/9 • 56 days
Renal and urinary disorders Urinary incontinence	11.1% 1/9 • 56 days
Nervous system disorders Dysgeusia	22.2% 2/9 • 56 days
Nervous system disorders Dizziness	11.1% 1/9 • 56 days
Nervous system disorders Headache	11.1% 1/9 • 56 days
Nervous system disorders Somnolence	11.1% 1/9 • 56 days
Infections and infestations COVID-19	11.1% 1/9 • 56 days
Infections and infestations Diverticulitis	11.1% 1/9 • 56 days
Infections and infestations Urinary Tract Infection	11.1% 1/9 • 56 days
Metabolism and nutrition disorders Decreased appetite	22.2% 2/9 • 56 days
Metabolism and nutrition disorders Dehydration	11.1% 1/9 • 56 days
Psychiatric disorders Anxiety	11.1% 1/9 • 56 days
Psychiatric disorders Confusional state	11.1% 1/9 • 56 days
Psychiatric disorders Dysphoria	11.1% 1/9 • 56 days
Reproductive system and breast disorders Pelvic pain	22.2% 2/9 • 56 days
Reproductive system and breast disorders Penile burnnig sensation	11.1% 1/9 • 56 days
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 1/9 • 56 days
Musculoskeletal and connective tissue disorders Myalgia	11.1% 1/9 • 56 days
Vascular disorders Embolism	11.1% 1/9 • 56 days
Vascular disorders Hypertension	11.1% 1/9 • 56 days
Blood and lymphatic system disorders Neutropenia	11.1% 1/9 • 56 days
Investigations Platelet count decreased	11.1% 1/9 • 56 days
Skin and subcutaneous tissue disorders Alopecia	11.1% 1/9 • 56 days

Additional Information

Dr. John A Taylor

University of Kansas Medical Center

Phone: 9135888170

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The PI and Sponsor plan to publish the data.
Publication restrictions are in place

Restriction type: OTHER