A Phase I Intravesical PPM Therapy for NMIBC

NCT05519241 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: ONCA-021-20SVA Merit
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical trial is to determine the safety and effectiveness of an investigational bladder cancer drug named "PLZ4-coated paclitaxel-loaded nanoscale micelle (PPM)." PPM is tiny particles that contain the chemotherapy drug paclitaxel. PLZ4 is a molecule that can possibly guide PPM to specifically target and deliver paclitaxel into and kill bladder cancer cells. In this trial, PPM will be instilled into the bladder cavity to treat bladder cancer that does not invade into the muscle layer of the bladder and that has failed the treatment of another drug BCG. Up to 29 patients will be enrolled into the trial. The main goal of this trial is to determine the dose of PPM for future clinical trials, assess the toxicity and obtain preliminary data regarding its effectiveness.

Conditions

Non-muscle-invasive Bladder Cancer

Keywords

non-muscle-invasive bladder cancer; BCG-refractory; paclitaxel; intravesical instillation

Outcome Measures

Primary Outcomes (1)

• adverse events

  CTCAE v5.0 will be used to determine any adverse events and assess the grade. All toxicity as well as Grade 3 or higher adverse events will be analyzed.

  up to 6 weeks after the last dose

Secondary Outcomes (1)

• complete response rate

  6 weeks after finishing the last dose of PPM

Other Outcomes (1)

• blood paclitaxel concentration

  within 6 hours after administration

Study Arms (1)

Phase I dose-escalation and expansion cohort

EXPERIMENTAL

There are three doses at the dose escalation stage: Dose level I: paclitaxel (PTX) 25 mg or 0.5 mg/ml; Dose Level II: PTX 50 mg or 1.0 mg/ml; Dose Level III: PTX 75 mg or 1.5 mg/ml. At the expansion cohort, up to 12 patients will be recruited and treated with PPM at the PTX dose of 50 mg or 1.0 mg/ml to determine the efficacy.

Drug: PLZ4-coated paclitaxel-loaded micelles (PPM)

Interventions

PLZ4-coated paclitaxel-loaded micelles (PPM) DRUG

PPM will be administrated weekly for 6 times through intravesical instillation into the bladder cavity

Phase I dose-escalation and expansion cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must meet all of the following criteria to be eligible for study entry.
• Histologically confirmed bladder carcinoma in situ (CIS) urothelial or urothelial carcinoma, with or without T1 cancer. Patients are eligible if the biopsy was done within 3 months of enrollment and a cystoscopy demonstrates no gross disease invasion into muscularis propria within 4 weeks of enrollment.

You may not qualify if:

• Persistent or recurrent CIS alone or with recurrent Ta/T1 (noninvasive papillary disease/tumor invades the subepithelial connective tissue) disease within 12 months of completion of adequate BCG therapy
• Recurrent high-grade Ta/T1 disease within 6 months of completion of adequate BCG therapy
• T1 high-grade disease at the first evaluation following an induction BCG course
• In this context, adequate BCG therapy is defined as at least one of the following:
• At least five of six doses of an initial induction course plus at least two of three doses of maintenance therapy
• At least five of six doses of an initial induction course plus at least two of six doses of a second induction course
• Refuse or intolerant of a radical cystectomy recommended by the treating urologist as the standard next therapy per urologic guidelines.
• Age 18 years.
• Performance status: ECOG performance status of 0, 1, or 2 (Appendix A) or Karnofsky performance status of 50 or higher (Appendix B).
• Patient with life expectancy greater than 24 months.
• No concurrent radiotherapy, chemotherapy, or other immunotherapy
• No scheduled radiotherapy, chemotherapy, other immunotherapy, or surgery before the scheduled response evaluation.
• Recovery from prior treatment side effects that might interfere with the study treatment, in the judgment of the participating urologist.
• Laboratory tests performed within 14 days of study enrollment:
• Absolute neutrophil count (AGC/ANC) 1,500/uL

Sponsors & Collaborators

• VA Office of Research and Development: lead
• University of California, Davis: collaborator

Study Sites (1)

VA Boston Healthcare System Jamaica Plain Campus, Jamaica Plain, MA

Boston, Massachusetts, 02130-4817, United States

RECRUITING

MeSH Terms

Conditions

Non-Muscle Invasive Bladder Neoplasms

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Urinary Bladder Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Study Officials

• Chong-Xian Pan, MD PhD

  VA Boston Healthcare System Jamaica Plain Campus, Jamaica Plain, MA

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chong-Xian Pan, MD PhD

CONTACT

(857) 203-6189; chong-xian.pan@va.gov

Lori Lerner, MD

CONTACT

(857) 364-6150; lori.lerner@va.gov

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: FED
• Responsible Party: SPONSOR

Model Details: This is a Phase I, single-group, dose-escalation trial

Study Record Dates

• First Submitted: August 18, 2022
• First Posted: August 29, 2022
• Study Start: October 10, 2022
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: September 2, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)