Bladder Cancer
Conditions
Keywords
Newly diagnosed or recurrent bladder tumors
Brief summary
This is a phase II, single center open label to determine safety, dose tolerance, PK and PD of the recommended Phase 2 dose (RP2D) of CPX-POM administered in patients with any newly diagnosed or recurrent bladder tumors.
Detailed description
This will be an open-label study to determine the safety, dose tolerance, pharmacokinetics, and pharmacodynamics of CPX-POM in patients with newly diagnosed or recurrent, untreated or intravesical treatment completed \>6 months before the current diagnosis, resectable tumors. Approximately 12 patients will be enrolled and treated with 900 mg/m2 CPX-POM administered IV over 20 minutes once per day for 5 days followed by TURBT on Day 5 after the fifth dose. TURBT will be performed 2 to 6 hours following drug administration on Day 5. Pretreatment bladder tumor tissues will be obtained at the time of in-office cystoscopy by cold cup biopsy within 4 weeks of TURBT. Posttreatment bladder tumor tissues will be obtained at TURBT. Bladder tumor tissues will undergo pathological evaluation at each site. Prior to administration of the first CPX-POM dose on Day 1, pre-dose blood (plasma) and urine (clean catch) samples will be collected. At the time of TURBT on Day 5, one 3-mL blood (plasma) sample and a urine specimen will be collected for measurement of CPX-POM concentrations. Patients will be followed for at least 30 days after the last dose of CPX-POM for safety
Interventions
DRUGCPX-POM
CPX-POM
Sponsors
CicloMed LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patient is male or female aged ≥18 years. 2. Patient provided signed and dated informed consent prior to initiation of any study procedures. 3. Patient is likely to have a new bladder tumor based on clinical presentation or is at high risk for tumor recurrence based on previous history. 4. Patient has a cystoscopically confirmed bladder tumor and will be scheduled to undergo TURBT. 5. Patient has not received prior treatment for bladder cancer or completed their last intravesical therapy \>6 months before screening. 6. Patient has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 (fully active, able to carry out all pre-disease activities without restriction) or 1 (unable to perform physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature). 7. Patient has a predicted life expectancy of ≥3 months. 8. Patient has adequate renal function (creatinine ≤1.5 × the upper limit of the normal range (ULN) or an estimated glomerular filtration rate (eGFR) of \>30 mL/min/1.73 m2). 9. Patient has adequate hepatic function, as evidenced by a total bilirubin ≤1.5 × ULN, aspartate aminotransferase (AST) ≤3 × ULN and /or alanine aminotransferase (ALT) ≤3 × ULN. 10. Patient has adequate bone marrow function, as evidenced by hemoglobin ≥9.0 g/dL in the absence of transfusion within the previous 72 hours, platelet count ≥100×10\^9cells/L, and absolute neutrophil count (ANC) ≥1.5×10\^9 cells/L. 11. Patient has no significant ischemic heart disease or myocardial infarction within 6 months before the first dose of CPX-POM and currently has adequate cardiac function, as evidenced by a left ventricular ejection fraction of \>50% as assessed by multi-gated acquisition (MUGA) or ultrasound/echocardiography (ECHO); and corrected QT interval by Fridericia's correction formula (QTcF) \<450 msec for males and \<470 msec for females. The eligibility of patients with ventricular pacemakers for whom the QT interval may not be accurately measurable will be determined on a case-by-case basis by the Sponsor in consultation with the Medical Monitor. 12. Patient and his/her partner agree to use adequate contraception after providing written informed consent through 3 months after the last dose of CPX-POM, as follows: 1. For women: Negative pregnancy test during Screening and at Day 1 of each treatment cycle and compliant with a medically-approved contraceptive regimen during and for 3 months after the treatment period or documented to be surgically sterile or postmenopausal. 2. For men: Compliant with a medically-approved contraceptive regimen during and for 3 months after the treatment period or documented to be surgically sterile. Men whose sexual partners are of child-bearing potential must agree to use 2 methods of contraception prior to study entry, during the study, and for 3 months after the treatment period. 13. Patient is willing and able to participate in the study and comply with all study requirements.
Exclusion criteria
Patients who meet any of the following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Any Serious Adverse Events (SAEs) | 35 days | Incidence of Serious Adverse Events in subjects receiving CPX-POM as assessed by (CTCAE) version 5.0 |
| Number of Participants With Any Adverse Events (AEs) | 35 days | Incidence of Adverse Events in subjects receiving CPX-POM as assessed by (CTCAE) version 5.0 |
| Evaluate the Dose Limiting Toxicities (DLTs) of CPX-POM | 35 days | A DLT will include some Grade 3 or 4 AEs if deemed related to study drug. In addition, any patient who is unable to receive 80% of the expected dose of CPX-POM (i.e., patients who are unable to receive at least 4 of the 5 scheduled doses) because of AEs will be considered to have a DLT. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment Related Adverse Events | 35 days | To determine the number of subjects with treatment related AEs |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| CPX-POM IV over 20 minutes once per day
CPX-POM: CPX-POM | 12 |
| Total | 12 |
Baseline characteristics
| Characteristic | CPX-POM |
|---|---|
| Age, Continuous | 69.41 years STANDARD_DEVIATION 10.01 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 12 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 9 / 12 |
| serious Total, serious adverse events | 1 / 12 |
Outcome results
Primary
Evaluate the Dose Limiting Toxicities (DLTs) of CPX-POM
A DLT will include some Grade 3 or 4 AEs if deemed related to study drug. In addition, any patient who is unable to receive 80% of the expected dose of CPX-POM (i.e., patients who are unable to receive at least 4 of the 5 scheduled doses) because of AEs will be considered to have a DLT.
Time frame: 35 days
Population: Safety population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CPX-POM | Evaluate the Dose Limiting Toxicities (DLTs) of CPX-POM | 2 Participants |
Primary
Number of Participants With Any Adverse Events (AEs)
Incidence of Adverse Events in subjects receiving CPX-POM as assessed by (CTCAE) version 5.0
Time frame: 35 days
Population: safety population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CPX-POM | Number of Participants With Any Adverse Events (AEs) | 9 Participants |
Primary
Number of Participants With Any Serious Adverse Events (SAEs)
Incidence of Serious Adverse Events in subjects receiving CPX-POM as assessed by (CTCAE) version 5.0
Time frame: 35 days
Population: safety population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CPX-POM | Number of Participants With Any Serious Adverse Events (SAEs) | 1 Participants |
Secondary
Incidence of Treatment Related Adverse Events
To determine the number of subjects with treatment related AEs
Time frame: 35 days
Population: Safety population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CPX-POM | Incidence of Treatment Related Adverse Events | 4 Participants |