Non-Muscle-Invasive Bladder Cancer (NMIBC)Phase 1NCT06351904
A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy
A Phase 1 study testing RAG-01 in people with Non-Muscle-Invasive Bladder Cancer (NMIBC), run by Ractigen Therapeutics. at 3 sites in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Non-Muscle-Invasive Bladder Cancer (NMIBC)
- What is being tested?
- RAG-01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 3 sitesAustralia
- Study dates
- 3 Apr 2024to 31 Aug 2028
- Phase
- Phase 1
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- 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;
Who cannot join
- 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;
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- 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.
- Cerebrovascular accidents have not been relieved to CTCAE 0-1;
- HIV positive; Active hepatitis B or C. For active HBV, it's defined as HBsAg positive with HBV DNA ≥ ULN, for active HCV, it's defined as HCV antibody positive with HCV RNA ≥ ULN, respectively.
- Subject is pregnant or lactating during the treatment period;
- History of central nervous system or psychiatric disorders, e.g., epilepsy or dementia;
- Other severe systemic diseases which might compromise the subject's compliance to the study, e.g., uncontrolled diabetes, gastrointestinal disorders, and renal diseases;
- Any other situations which are judged by the investigator, would exposure the subject to unnecessary risk, therefore he/she is not eligible.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia3 sites, 3 recruiting
- Recruiting
The Royal Melbourne Hospital
Melbourne, Victoria, 3050
Paul Anderson, MBBS FRACS, 0393427294, mr.paul.anderson@gmail.com
- Recruiting
Peninsula & South Eastern Haematology and Oncology Group
Melbourne, Victoria, 3199
Sanjeev Sewak, MBBS, ECFMG, 0397815244, sewaks01@yahoo.com
- Recruiting
GenesisCare North Shore
St Leonards, New South Wales, 2065
Laurence Krieger, MBCHB, +61 2 8037 4100, laurence_krieger@hotmail.com
About this study
Official title: A Phase Ⅰ, Open Label, Multi-center Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy
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.
Read the detailed description
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.
- Being tested
- RAG-01
- 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: RAG-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
Measured: Within 21 days after first instillation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2024
- Study start3 Apr 2024
- Primary completion31 Aug 2028 (estimated)
- Study completion31 Aug 2028 (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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ractigen Therapeutics.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Laurence Krieger, MBCHB, Principal investigator, GenesisCare, 7 Westbourne Street, St Leonards, NSW 2065, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06351904
- Sponsor's protocol ID
- RAG-01-01
- Status last verified
- Sep 2025
- First submitted
- 2 Apr 2024
- Last updated
- 11 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ⅰ, Open Label, Multi-center Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy
- ClinicalTrials.gov ID
- NCT06351904
- Acronym / sponsor ID
- RAG-01-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Ractigen Therapeutics.University, hospital or other
- Study start
- 3 Apr 2024 (actual)
- Primary completion
- 31 Aug 2028 (estimated)
- Study completion
- 31 Aug 2028 (estimated)
- First posted
- 8 Apr 2024 (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 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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RAG-01 dose escalation and dose expansion | Experimental | Drug: RAG-01The 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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;
Key exclusion criteria (21 listed)
- 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:
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 | RAG-01 dose escalation and dose expansion |
Primary outcome
Safety and tolerability of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)
Time frame: From the screening to 6 months after the first instillation of RAG-01
Adverse events (AEs), serious adverse events (SAEs), and adverse events during treatment (TEAEs)
Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of RAG-01
Time frame: Within 21 days after first instillation
Dose-limiting toxicity (DLT)
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 11 Sep 2025.
- Individual participant dataSharing planned: No
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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.
Laurence Krieger, MBCHB principal investigator
GenesisCare, 7 Westbourne Street, St Leonards, NSW 2065, Australia
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RAG-01 in people with Non-Muscle-Invasive Bladder Cancer (NMIBC), run by Ractigen Therapeutics. at 3 sites in Australia.
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 9 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Australia. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety and tolerability of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC) (time frame: From the screening to 6 months after the first instillation of RAG-01). The study lists 2 primary outcomes in all. 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 11 Sep 2025.