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

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorRactigen Therapeutics.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • RAG-01

    Drug

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

Treatment groups

72 Participants
are divided into 1 treatment group
Group A: RAG-01 dose escalation and dose expansionExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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)

Time frame
From the screening to 6 months after the first instillation of RAG-01
2

Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of RAG-01

Dose-limiting toxicity (DLT)

Time frame
Within 21 days after first instillation

Secondary outcomes

Other outcomes

Sponsors and contacts

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