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

PHASE1RECRUITING

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.

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Study details:

The study is planned to include a "3+3" dose escalation phase. The primary study period includes a screening period, a treatment and Dose-Limiting Toxicity (DLT) assessment period, and a follow-up period.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Ability to understand the study and have signed the informed consent form;

≥ 18, ≤ 75 years old, male or female;

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.

Exclusion criteria

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: 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);

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.

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2024-04-03

Primary completion: 2025-06-30

Study completion finish: 2025-08-31

Study type

TREATMENT

Phase

PHASE1

Trial ID

NCT06351904

Intervention or treatment

DRUG: RAG-01

Conditions

• Non-Muscle-Invasive Bladder Cancer (NMIBC)

Image related to Non-Muscle-Invasive Bladder Cancer (NMIBC)

Condition: Non-Muscle-Invasive Bladder Cancer (NMIBC)
DRUG: RAG-01
St Leonards, New South Wales, Australia and more
Sponsor: Ractigen Therapeutics.

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

GenesisCare North Shore

Research sites nearby

Select from list below to view details:

GenesisCare North Shore
St Leonards, New South Wales, Australia
The Royal Melbourne Hospital
Melbourne, Victoria, Australia
Peninsula & South Eastern Haematology and Oncology Group
Melbourne, Victoria, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
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.	DRUG: RAG-01 RAG-01 is a therapeutic small activating RNA (saRNA).

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
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

Secondary outcome

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

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