A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy

NCT06351904 | Recruiting Phase 1 FDA Drug

• 1 other identifier: RAG-01-01
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Non-Muscle-Invasive Bladder Cancer (NMIBC)

Outcome Measures

Primary Outcomes (2)

• Safety 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)

  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)

  Within 21 days after first instillation

Study Arms (1)

RAG-01 dose escalation and dose expansion

EXPERIMENTAL

The starting dose of RAG-01 is 30mg, and there are 4 dose cohorts, including 30mg, 100mg, 300mg, 600mg, respectively. Each eligible subject will be distributed into one cohort. Safety and pharmacokinetics are assessed at each dose level.

Drug: RAG-01

Interventions

RAG-01 DRUG

RAG-01 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands.

RAG-01 dose escalation and dose expansion

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ability to understand the study and have signed the informed consent form;
• Any adult ≥ 18 years old;
• Pathologically confirmed high grade NMIBC defined as grade 2 or grade 3 disease;
• Expected survival ≥ 6 months;
• ECOG PS ≤2;
• Sufficient organ functions, as defined below:
• Investigations Hematology Absolute Neutrophil Count (ANC): ≥ 1.5 x 109/L Hemoglobin: ≥ 90 g/L Platelet: ≥ 100 x 109/L Liver Function Serum bilirubin: ≤ 1.25×ULN or 2.5×ULN（with Gilbert syndrome） AST \& ALT: ≤ 2.5×ULN Renal Function Creatinine Clearance (Cockcroft-Gault equation): ≥ 30 mL/min
• Subject must be able to tolerate catheterization;
• Female subject of childbearing potential and her spouse must use adopt effective contraception (non-pharmacological contraception required) from signing informed consent to within 6 months after the last instillation;

You may not qualify if:

• Subject who is allergy to RAG-01 or similar products;
• Except for TURBT, the subject received other anti-tumor treatments, and the last administration date is within ≤ 21 days or 5 half-lifes whichever is shorter from the date of signing ICF;
• Subject with imaging examination diagnosed extravesical metastasis, including ureter and urethra;
• Subject has other malignancies within the past 3 years, except for adequately treated carcinoma of the cervix, basal or squamous cell carcinomas of the skin, or adenocarcinoma of the prostate that has been surgically treated with a post-treatment PSA that is non-detectable;
• The following illnesses have not been relieved to CTCAE 0-1:
• Uncontrolled acute and chronic infections, such as pneumonia, biliary tract infection, hepatitis B virus infection and hepatitis C virus infection;
• Dyspnea;
• Acute and chronic kidney injury, and inflammation;
• Urinary incontinence;
• Urinary frequency;
• Urinary tract obstruction (except benign prostatic hypertrophy);
• Subject could not hold the urine for at least 90 mins due to any reason;
• New York Heart Association (NYHA) 3 or 4 grade;
• Coronary heart disease related symptoms have not been relieved to CTCAE 0-1, including: myocardial infarction, unstable angina pectoris, congestive heart failure and arrhythmia;
• Subject with QTc \>470 msec.

Sponsors & Collaborators

• Ractigen Therapeutics.: lead

Study Sites (3)

GenesisCare North Shore

St Leonards, New South Wales, 2065, Australia

RECRUITING

The Royal Melbourne Hospital

Melbourne, Victoria, 3050, Australia

RECRUITING

Peninsula & South Eastern Haematology and Oncology Group

Melbourne, Victoria, 3199, Australia

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

• Laurence Krieger, MBCHB

  GenesisCare, 7 Westbourne Street, St Leonards, NSW 2065, Australia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Long-Cheng Li

CONTACT

+86 18051622388; lilc@ractigen.com

Study Design

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

Study Record Dates

• First Submitted: April 2, 2024
• First Posted: April 8, 2024
• Study Start: April 3, 2024
• Primary Completion (Estimated): August 31, 2028
• Study Completion (Estimated): August 31, 2028
• Last Updated: September 11, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3)