This is an open label, multi-center study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of RAG-01 in patients with NMIBC who have failed BCG therapy.
This study includes a dose escalation and a dose expansion. The dose escalation part will adopt a '3+3' dose escalation design. Additional subjects may be enrolled to have approximately 12 patients in each selected dose level for RDE determination. A randomized, open label, parallel-arm study will be conducted during the dose expansion part for further dose exploration. Approximately 40-60 subjects will be randomized in a 1:1 ratio into two treatment groups with dose levels selected from dose escalation part of the study. Randomization will be stratified based on disease type at enrollment (papillary-only vs. CIS). A dose selection analysis will be performed to assess safety, efficacy, PK, and PD, where applicable, to determine the optimal dose for RAG-01. The main study period includes the screening, initial treatment and DLT assessment period, second and third treatment period and the sequential follow-up period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
72
RAG-01 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands.
GenesisCare North Shore
St Leonards, New South Wales, Australia
RECRUITINGThe Royal Melbourne Hospital
Melbourne, Victoria, Australia
RECRUITINGPeninsula & South Eastern Haematology and Oncology Group
Melbourne, Victoria, Australia
RECRUITINGSafety and tolerability of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)
Adverse events (AEs), serious adverse events (SAEs), and adverse events during treatment (TEAEs)
Time frame: From the screening to 6 months after the first instillation of RAG-01
Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of RAG-01
Dose-limiting toxicity (DLT)
Time frame: Within 21 days after first instillation
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