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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06351904




Registration number
NCT06351904
Ethics application status
Date submitted
2/04/2024
Date registered
8/04/2024
Date last updated
28/06/2024

Titles & IDs
Public title
A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy
Scientific title
A Phase ?, 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
Secondary ID [1] 0 0
RAG-01-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Non-Muscle-Invasive Bladder Cancer (NMIBC) 0 0
Condition category
Condition code
Cancer 0 0 0 0
Bladder - transitional cell cancer
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RAG-01

Experimental: RAG-01 - The starting dose of RAG-01 is 30 mg, and there are 4 dose cohorts, including 30 mg, 100 mg, 300 mg and 600 mg, respectively. Each eligible subject will be distributed into one dose cohort.


Treatment: Drugs: RAG-01
RAG-01 is a therapeutic small activating RNA (saRNA).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)
Timepoint [1] 0 0
From the screening to 6 months after the first instillation of RAG-01
Primary outcome [2] 0 0
Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of RAG-01
Timepoint [2] 0 0
Within 21 days after first instillation

Eligibility
Key inclusion criteria
1. Ability to understand the study and have signed the informed consent form;
2. = 18, = 75 years old, male or female;
3. Pathologically confirmed high grade NMIBC defined as grade 2 or grade 3 disease;
4. Expected survival = 6 months;
5. ECOG PS =2;
6. 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
7. Subject must be able to tolerate catheterization;
8. 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.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subject who is allergy to RAG-01 or similar products;
2. 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;
3. Subject with imaging examination diagnosed extravesical metastasis, including ureter and urethra;
4. 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;
5. The following illnesses have not been relieved to CTCAE 0-1:

1. Uncontrolled acute and chronic infections, such as pneumonia, biliary tract infection, hepatitis B virus infection and hepatitis C virus infection;
2. Dyspnea;
3. Acute and chronic kidney injury, and inflammation;
4. Urinary incontinence;
5. Urinary frequency;
6. Urinary tract obstruction (except benign prostatic hypertrophy);
6. Subject could not hold the urine for at least 90 mins due to any reason;
7. New York Heart Association (NYHA) 3 or 4 grade;
8. Coronary heart disease related symptoms have not been relieved to CTCAE 0-1, including: myocardial infarction, unstable angina pectoris, congestive heart failure and arrhythmia;
9. Subject with QTc >470 msec.
10. Cerebrovascular accidents have not been relieved to CTCAE 0-1;
11. 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.
12. Subject is pregnant or lactating during the treatment period;
13. History of central nervous system or psychiatric disorders, e.g., epilepsy or dementia;
14. Other severe systemic diseases which might compromise the subject's compliance to the study, e.g., uncontrolled diabetes, gastrointestinal disorders, and renal diseases;
15. Any other situations which are judged by the investigator, would exposure the subject to unnecessary risk, therefore he/she is not eligible.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/08/2025
Actual
Sample size
Target
15
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
GenesisCare North Shore - St Leonards
Recruitment hospital [2] 0 0
The Royal Melbourne Hospital - Melbourne
Recruitment hospital [3] 0 0
Peninsula & South Eastern Haematology and Oncology Group - Melbourne
Recruitment postcode(s) [1] 0 0
2065 - St Leonards
Recruitment postcode(s) [2] 0 0
3050 - Melbourne
Recruitment postcode(s) [3] 0 0
3199 - Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Ractigen Therapeutics.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06351904
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Laurence Krieger, MBCHB
Address 0 0
GenesisCare, 7 Westbourne Street, St Leonards, NSW 2065, Australia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Long-Cheng Li
Address 0 0
Country 0 0
Phone 0 0
+86 18051622388
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06351904