A Study to Evaluate the Efficacy and Safety of RAG-01 as Monotherapy and in Combination With Chemotherapy in Patients With Non-muscle Invasive Bladder Cancer (NMIBC).

NCT07777848 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: RAG010201
• Study Type: interventional
• Target: 114
• Locations: 1 country
• Sites: 1

Brief Summary

This is a randomized, controlled, multicenter clinical study to evaluate the efficacy and safety of RAG-01 monotherapy and in combination with chemotherapy in patients with NMIBC.

Conditions

Non-muscle Invasive Bladder Cancer (NMIBC)

Outcome Measures

Primary Outcomes (1)

• Efficacy of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)

  Event-Free Survival rate

  24 months

Study Arms (2)

Cohort A: RAG-01 Monotherapy and Combination

EXPERIMENTAL

High-risk NMIBC patients will be enrolled in a safety lead-in phase to evaluate the safety, tolerability, and initial efficacy of RAG-01 as monotherapy and in combination with a chemotherapy agent, and to determine the recommended dose. Approximately 12-24 patients will be enrolled in this lead-in phase, and these patients will receive non-randomized treatment. Following completion of the safety assessment during the safety lead-in phase and confirmation of acceptable safety results, additional eligible patients will be enrolled into a randomized controlled phase. In the randomized phase, patients will be randomized 1:1 to either RAG-01 monotherapy or RAG-01 plus a chemotherapy agent.

Drug: RAG-01; Drug: Chemotherapy agent

Cohort B: RAG-01 Monotherapy

EXPERIMENTAL

Intermediate-risk NMIBC patients will receive RAG-01 monotherapy.

Drug: RAG-01

Interventions

Chemotherapy agent DRUG

Chemotherapy agent administered in combination with RAG-01.

Cohort A: RAG-01 Monotherapy and Combination

RAG-01 DRUG

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

Cohort A: RAG-01 Monotherapy and Combination; Cohort B: RAG-01 Monotherapy

Eligibility Criteria

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

You may qualify if:

• Voluntarily agrees to participate in this study and provides written informed consent prior to the initiation of any study-specific procedures.
• Is aged ≥18 years at the time of signing the informed consent form (ICF).
• Has histologically confirmed tumor tissue obtained within 12 weeks prior to the first study treatment, with urothelial carcinoma as the predominant histologic subtype (≥90%), and is classified according to the American Urological Association (AUA) guidelines as follows:
• Cohort A: high-risk non-muscle-invasive bladder cancer (NMIBC).
• Cohort B: intermediate-risk NMIBC.
• Regardless of prior bacillus Calmette-Guérin (BCG) treatment, is confirmed before enrollment to be BCG-intolerant, ineligible for BCG, unwilling to receive BCG, or unable to access BCG treatment for any reason.
• Has no resectable lesion, as confirmed by cystoscopy and/or transurethral resection of bladder tumor (TURBT), within 12 weeks prior to the first administration of study treatment.
• Is willing to provide pathological tissue specimens.
• Has an Eastern Cooperative Oncology Group (ECOG) performance status of ≤1.
• Has adequate hematologic and organ function within 14 days prior to enrollment.
• Female participants must agree to use effective contraception during treatment or be surgically sterile or postmenopausal. Women of childbearing potential must have a negative pregnancy test during the screening period; however, pregnancy testing is also required for participants with bilateral tubal ligation. Male participants must be surgically sterile or agree to use effective contraception during treatment. Participants must use contraception from the time of signing the ICF until 3 months after completion of study treatment. The definition of effective contraception will be based on the investigator's judgment.

You may not qualify if:

• Low-risk NMIBC.
• Very high-risk NMIBC.
• Tumors staged as T2 or higher, or urothelial carcinoma of the upper urinary tract or urethra (renal pelvis, ureter, or urethra).
• Predominantly non-urothelial histology, including squamous cell carcinoma, adenocarcinoma, or other non-urothelial carcinomas.
• Receipt of antitumor intravesical therapy between the most recent transurethral resection of bladder tumor (TURBT) and the first administration of study treatment, except for a single immediate postoperative intravesical chemotherapy instillation.
• Receipt of any antitumor therapy within 4 weeks prior to the first administration of study treatment.
• Participation in another clinical trial involving an antitumor investigational drug within 4 weeks prior to the first administration of study treatment.
• Prior treatment with small nucleic acid-based antitumor agents, p21-targeted antitumor agents, or cyclin-dependent kinase (CDK) inhibitors.
• Major surgery within 4 weeks prior to signing the informed consent form (ICF).
• History of malignancy within 2 years prior to enrollment, except for curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix; or ongoing requirement for antitumor therapy.
• Any Grade ≥3 hemorrhage, as defined by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 6.0, at any site within 6 weeks prior to enrollment, regardless of whether the event has resolved.
• Chronic renal injury or inflammation, or renal injury or inflammation not recovered to Grade ≤1 according to NCI CTCAE version 6.0.
• Any of the following within 6 months prior to enrollment: myocardial infarction; severe or unstable angina; coronary or peripheral arterial bypass grafting; symptomatic congestive heart failure; cerebrovascular accident or transient ischemic attack; or pulmonary embolism.
• Uncontrolled hypertension.
• New York Heart Association (NYHA) Class III or IV heart failure, or left ventricular ejection fraction (LVEF) \<50%.

Sponsors & Collaborators

• Ractigen Therapeutics.: lead

Study Sites (1)

Fudan University Shanghai Cancer Center

Shanghai, Shanghai Municipality, 200032, China

Location

MeSH Terms

Conditions

Non-Muscle Invasive Bladder Neoplasms

Interventions

Antineoplastic Agents

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

Intervention Hierarchy (Ancestors)

Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Dingwei Ye

  270 Dong'an Road, Xuhui District, Shanghai, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Long-Cheng Li

CONTACT

+86 18051622388; lilc@ractigen.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2026
• First Posted: August 20, 2026
• Study Start: September 1, 2026
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): March 1, 2029
• Last Updated: September 15, 2026

Record last verified: 2026-09

Locations

CN (1)