Ignite Creation Date:
2025-12-24 @ 9:11 PM
Ignite Modification Date:
2025-12-28 @ 2:23 PM
Study NCT ID:
NCT06351904
Status:
RECRUITING
Last Update Posted:
2025-09-11
First Post:
2024-04-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000093284', 'term': 'Non-Muscle Invasive Bladder Neoplasms'}], 'ancestors': [{'id': 'D002277', 'term': 'Carcinoma'}, {'id': 'D009375', 'term': 'Neoplasms, Glandular and Epithelial'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D001749', 'term': 'Urinary Bladder Neoplasms'}, {'id': 'D014571', 'term': 'Urologic Neoplasms'}, {'id': 'D014565', 'term': 'Urogenital Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D001745', 'term': 'Urinary Bladder Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 72}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-04-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-04', 'studyFirstSubmitDate': '2024-04-02', 'studyFirstSubmitQcDate': '2024-04-02', 'lastUpdatePostDateStruct': {'date': '2025-09-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-04-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability of RAG-01 in patients with non-muscle-invasive bladder cancer (NMIBC)', 'timeFrame': 'From the screening to 6 months after the first instillation of RAG-01', 'description': 'Adverse events (AEs), serious adverse events (SAEs), and adverse events during treatment (TEAEs)'}, {'measure': 'Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of RAG-01', 'timeFrame': 'Within 21 days after first instillation', 'description': 'Dose-limiting toxicity (DLT)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Non-Muscle-Invasive Bladder Cancer (NMIBC)']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': "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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Ability to understand the study and have signed the informed consent form;\n2. Any adult ≥ 18 years old;\n3. Pathologically confirmed high grade NMIBC defined as grade 2 or grade 3 disease;\n4. Expected survival ≥ 6 months;\n5. ECOG PS ≤2;\n6. Sufficient organ functions, as defined below:\n\n 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\n7. Subject must be able to tolerate catheterization;\n8. 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;\n\nExclusion Criteria:\n\n1. Subject who is allergy to RAG-01 or similar products;\n2. 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;\n3. Subject with imaging examination diagnosed extravesical metastasis, including ureter and urethra;\n4. 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;\n5. The following illnesses have not been relieved to CTCAE 0-1:\n\n 1. Uncontrolled acute and chronic infections, such as pneumonia, biliary tract infection, hepatitis B virus infection and hepatitis C virus infection;\n 2. Dyspnea;\n 3. Acute and chronic kidney injury, and inflammation;\n 4. Urinary incontinence;\n 5. Urinary frequency;\n 6. Urinary tract obstruction (except benign prostatic hypertrophy);\n6. Subject could not hold the urine for at least 90 mins due to any reason;\n7. New York Heart Association (NYHA) 3 or 4 grade;\n8. Coronary heart disease related symptoms have not been relieved to CTCAE 0-1, including: myocardial infarction, unstable angina pectoris, congestive heart failure and arrhythmia;\n9. Subject with QTc \\>470 msec.\n10. Cerebrovascular accidents have not been relieved to CTCAE 0-1;\n11. 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.\n12. Subject is pregnant or lactating during the treatment period;\n13. History of central nervous system or psychiatric disorders, e.g., epilepsy or dementia;\n14. Other severe systemic diseases which might compromise the subject's compliance to the study, e.g., uncontrolled diabetes, gastrointestinal disorders, and renal diseases;\n15. Any other situations which are judged by the investigator, would exposure the subject to unnecessary risk, therefore he/she is not eligible."}, 'identificationModule': {'nctId': 'NCT06351904', 'briefTitle': 'A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy', 'organization': {'class': 'OTHER', 'fullName': 'Ractigen Therapeutics.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'RAG-01-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RAG-01 dose escalation and dose expansion', 'description': '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.', 'interventionNames': ['Drug: RAG-01']}], 'interventions': [{'name': 'RAG-01', 'type': 'DRUG', 'description': 'RAG-01 is a therapeutic small activating RNA (saRNA) duplex molecule comprised of two partially chemically modified complementary oligonucleotide strands.', 'armGroupLabels': ['RAG-01 dose escalation and dose expansion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2065', 'city': 'St Leonards', 'state': 'New South Wales', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Laurence Krieger, MBCHB', 'role': 'CONTACT', 'email': 'laurence_krieger@hotmail.com', 'phone': '+61 2 8037 4100'}], 'facility': 'GenesisCare North Shore', 'geoPoint': {'lat': -33.82344, 'lon': 151.19836}}, {'zip': '3050', 'city': 'Melbourne', 'state': 'Victoria', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Paul Anderson, MBBS FRACS', 'role': 'CONTACT', 'email': 'mr.paul.anderson@gmail.com', 'phone': '0393427294'}], 'facility': 'The Royal Melbourne Hospital', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': '3199', 'city': 'Melbourne', 'state': 'Victoria', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Sanjeev Sewak, MBBS, ECFMG', 'role': 'CONTACT', 'email': 'sewaks01@yahoo.com', 'phone': '0397815244'}], 'facility': 'Peninsula & South Eastern Haematology and Oncology Group', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}], 'centralContacts': [{'name': 'Long-Cheng Li', 'role': 'CONTACT', 'email': 'lilc@ractigen.com', 'phone': '+86 18051622388'}], 'overallOfficials': [{'name': 'Laurence Krieger, MBCHB', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'GenesisCare, 7 Westbourne Street, St Leonards, NSW 2065, Australia'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ractigen Therapeutics.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}