Non-muscle Invasive Bladder Cancer (NMIBC)Phase 2NCT07777848
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).
A Phase 2 study testing RAG-01 and Chemotherapy agent in people with Non-muscle Invasive Bladder Cancer (NMIBC), run by Ractigen Therapeutics. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-muscle Invasive Bladder Cancer (NMIBC)
- What is being tested?
- RAG-01 and Chemotherapy agent
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 114 participants
- Where
- 1 siteChina
- Study dates
- Sep 2026to Mar 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 11Show fewer
- 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.
Who cannot join
- 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.
Show all 26Show fewer
- 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%.
- Prolonged corrected QT (QTc) interval.
- Active infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), or tuberculosis at screening, including HIV antibody positivity; hepatitis B surface antigen (HBsAg) positivity with detectable HBV DNA; or HCV antibody positivity with detectable HCV RNA.
- Severe infection within 4 weeks prior to the first administration of study treatment, or active infection of Grade ≥2 per CTCAE requiring systemic antibiotic treatment within 1 week prior to the first administration of study treatment.
- Intolerance of or inability to receive intravesical instillation, or inability to retain intravesical study treatment for the duration required by the protocol for any reason.
- Anuria, urinary incontinence, or urinary tract obstruction within 4 weeks prior to enrollment.
- Persistent gross hematuria despite treatment within 1 week prior to enrollment.
- Uncontrolled pleural effusion, pericardial effusion, or ascites.
- Severe pulmonary disease of significant clinical relevance.
- Confirmed vesicoureteral reflux or bladder perforation.
- Any other serious acute or chronic medical condition that, in the investigator's judgment, may increase the risk associated with study participation or study treatment, or may interfere with the interpretation or assessment of study results.
- Severe psychological or psychiatric disorder that, in the investigator's judgment, is likely to result in inadequate compliance with study requirements.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Fudan University Shanghai Cancer Center
Shanghai, Shanghai Municipality, 200032
Dingwei Ye
About this study
Official title: A Randomized, Controlled, Multicenter Clinical Study to Evaluate the Efficacy and Safety of RAG-01 Monotherapy and RAG-01 in Combination With Chemotherapy in Intermediate/High-risk Patients With Non-muscle Invasive Bladder Cancer (NMIBC).
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.
- Being tested
- RAG-01, Chemotherapy agent
- Medical subject headings
- Non-Muscle Invasive Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAG-01Drug
RAG-01 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands.
Chemotherapy agentDrug
Chemotherapy agent administered in combination with RAG-01.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: RAG-01; Drug: Chemotherapy agent |
| Cohort B: RAG-01 Monotherapy | Experimental | Intermediate-risk NMIBC patients will receive RAG-01 monotherapy. Receives: Drug: RAG-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)
Event-Free Survival rate
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionMar 2029 (estimated)
- Study completionMar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-muscle Invasive Bladder Cancer (NMIBC)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 114 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ractigen Therapeutics.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dingwei Ye, Principal investigator, 270 Dong'an Road, Xuhui District, Shanghai, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07777848
- Sponsor's protocol ID
- RAG010201
- Status last verified
- Sep 2026
- First submitted
- 17 Aug 2026
- Last updated
- 15 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Controlled, Multicenter Clinical Study to Evaluate the Efficacy and Safety of RAG-01 Monotherapy and RAG-01 in Combination With Chemotherapy in Intermediate/High-risk Patients With Non-muscle Invasive Bladder Cancer (NMIBC).
- ClinicalTrials.gov ID
- NCT07777848
- Acronym / sponsor ID
- RAG010201
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 114 participants (estimated)
- Lead sponsor
- Ractigen Therapeutics.University, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Mar 2029 (estimated)
- Study completion
- Mar 2029 (estimated)
- First posted
- 20 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A: RAG-01 Monotherapy and Combination | Experimental | Drug: RAG-01, Drug: Chemotherapy agentHigh-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. |
| Cohort B: RAG-01 Monotherapy | Experimental | Drug: RAG-01Intermediate-risk NMIBC patients will receive RAG-01 monotherapy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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:
- 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.
Key exclusion criteria (26 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RAG-01RAG-01 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands. | Drug | Cohort A: RAG-01 Monotherapy and Combination, Cohort B: RAG-01 Monotherapy |
| Chemotherapy agentChemotherapy agent administered in combination with RAG-01. | Drug | Cohort A: RAG-01 Monotherapy and Combination |
Primary outcome
Efficacy of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)
Time frame: 24 months
Event-Free Survival rate
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
Related trials for Non-muscle Invasive Bladder Cancer (NMIBC)
RecruitingPhase 1
A Study to Learn About the Study Medicine PF-08052667 in People With Bladder Cancer
Non-muscle Invasive Bladder Cancer
Recruiting
Using Urine Tumor DNA to De-Intensify Surveillance in Non-Muscle Invasive Bladder Cancer
Non-muscle Invasive Bladder Cancer (NMIBC)
Active, not recruitingPhase 3
A Study of TAR-200 Versus Intravesical Chemotherapy in Participants With Recurrent High-Risk Non-Muscle-Invasive Bladder Cancer (HR-NMIBC) After Bacillus Calmette-Guérin (BCG)
Non-Muscle Invasive Bladder Neoplasms
RecruitingPhase 2
Disitamab Vedotin Plus Intravesical Mitomycin C in HER2-Expressing Intermediate- to High-Risk Non-Muscle-Invasive Bladder Cancer
Non-Muscle-Invasive Bladder Cancer (NMIBC)
RecruitingPhase 3
A Study to Evaluate TAR-210 Versus Single Agent Intravesical Cancer Treatment in Participants With Bladder Cancer
Non-Muscle Invasive Bladder Neoplasms
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Dingwei Ye principal investigator
270 Dong'an Road, Xuhui District, Shanghai, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RAG-01 and Chemotherapy agent in people with Non-muscle Invasive Bladder Cancer (NMIBC), run by Ractigen Therapeutics. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Long-Cheng Li (+86 18051622388, lilc@ractigen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC) (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2026.