# A Phase I trial of cancer-targeting micelles for non-myoinvasive bladder cancer

> **NIH VA I01** · VA BOSTON HEALTH CARE SYSTEM · 2024 · —

## Abstract

The goal of this project is to conduct a Phase I clinical trial to determine the recommended Phase II dose
(RP2D) of bladder cancer-targeting micelles loaded with a chemotherapeutic drug paclitaxel (PTX). With a VA
Career Development Award-2 and VA Merit grant (PI: Pan), we developed a bladder cancer-specific targeting
ligand named PLZ4, and a PLZ4-coated PTX-loaded nanoscale micelle (PPM) platform that can specifically
deliver the drug load into bladder cancer cells both in vitro and in vivo. This proposed clinical trial is to conduct
a first-in-human trial to use PPM for the treatment of non-myoinvasive bladder cancer (NMIBC). Bladder cancer
is one of the ten most common cancers in the United States. NMIBC accounts for around 75% of bladder
cancer cases at diagnosis. The current first-line treatment approach of transurethral resection followed by
intravesical instillation of therapeutic agents, usually Bacillus Calmette-Guérin (BCG), is associated with a
recurrence rate of around 70% at two years. Pembrolizumab and valrubicin are the only second-line drug
approved by the Food and Drug Administration (FDA) with a response rate of 40% and 20-40%, respectively.
Considering that bladder cancer is among the top ten most common cancers and over 70% of bladder cancers
are diagnosed as NMIBC, there are huge unmet medical needs for developing more effective therapies. This
primary objective of the proposed Phase I trial is to determine the recommended Phase II dose (RP2D) of PPM.
The secondary objectives are to assess the toxicity, obtain preliminary efficacy information of PPM, and
determine systemic absorption after intravesical instillation of PPM. Up to 25 patients with recurrent or refractory
NMIBC after a standard first-line intravesical therapy pembrolizumab treatment will be recruited. PPM will be
given as intravesical instillation once weekly for six weeks. The toxicity will be assessed by the NCI Common
Terminology Criteria for Adverse Events (CTCAE) version 5.0. The efficacy will be determined by urine cytology
and cystoscopy two weeks after finishing treatment. Molecular correlative studies will be performed.

## Key facts

- **NIH application ID:** 10909807
- **Project number:** 5I01CX002247-04
- **Recipient organization:** VA BOSTON HEALTH CARE SYSTEM
- **Principal Investigator:** CHONG-XIAN PAN
- **Activity code:** I01 (R01, R21, SBIR, etc.)
- **Funding institute:** VA
- **Fiscal year:** 2024
- **Award amount:** —
- **Award type:** 5
- **Project period:** 2020-10-01 → 2026-09-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10909807

## Citation

> US National Institutes of Health, RePORTER application 10909807, A Phase I trial of cancer-targeting micelles for non-myoinvasive bladder cancer (5I01CX002247-04). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10909807. Licensed CC0.

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