Clinical trial NCT06934590: Evaluate Safety and Tolerability of RX001 in Patients With KRAS Mutant Advanced NSCLC
Official title: An Open-label, Dose Escalation, Phase I Study to Evaluate Safety and Tolerability of RX001 in Patients With KRAS Mutant Advanced Non-small Cell Lung Cancer
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Non-Small Cell Lung Cancer
Keywords: KRAS
NSCLC
RUNX3
AAV
Lead sponsor: GeneCraft Inc.
Sponsor class: INDUSTRY
First posted: 2025-04-18
Enrollment: 18
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is an open-label, dose-escalation Phase I study to evaluate the safety and tolerability of RX001 monotherapy in patients with advanced KRAS mutant NSCLC. Subjects aged ≥19 years with advanced NSCLC with KRAS mutations will be enrolled. Based on the investigator's judgment of the lung tumor location, RX001 will be administered as a single intratumoral treatment via bronchoscopy or CT-guided percutaneous injection. Additionally, after conducting safety, tolerability, and preliminary anti-tumor efficacy assessments for all subjects up to Week 12 (D84) following IP administration, the collected data will be reviewed by the SRC for a comprehensive evaluation. If the IP is designated for long-term follow-up studies, monitoring for malignant tumor formation and other SAEs specified by the Ministry of Food and Drug Safety (MFDS) will be conducted for a minimum of 5 years.


Eligibility criteria: Inclusion Criteria:

* Adults aged ≥19 years, male or female
* Subject with histologically and/or cytologically confirmed, unresectable advanced NSCLC for radical cure that are confirmed as PD for the standard of care currently known to have clinical benefits, or for which no currently available standard of care exists due to intolerance, ineligibility, treatment refusal, etc.
* Subjects must have at least one evaluable and injectable lesion as assessed on CT images based on RECIST version 1.1.
* Subjects with valid biopsy results documented the presence of KRAS mutation.
* Subjects with an ECOG performance status of 0 or 1 and an expected survival of at least 12 weeks at the time of study participation.
* Subjects with adequate hematologic and terminal organ function.
* Subjects must voluntarily sign an informed consent form approved by the IRB. Subjects must have the willingness and ability to comply with all procedures required by the protocol.

Exclusion Criteria:

* Subjects must not meet any of the following conditions:
* Subjects with a history of or current comorbidities including:

  1. Hematologic malignancies, such as lymphoma, or any other malignant tumors apart from the indication of this study.
  2. Subjects with the cardiovascular conditions such as unstable angina and/or myocardial infarction within 1 year prior to screening
  3. Subjects with the pulmonary baseline conditions:
  4. Blood coagulation disorder
  5. Active bleeding disorders (including gastrointestinal bleeding)
* Subjects who have received antithrombotics, including antiplatelets or anticoagulants
* Subjects who have received anticancer therapies within 28 days prior to trial participation, or who have not recovered or stabilized from all AEs caused by such treatments to baseline levels
* Subjects who have received radical radiotherapy within 6 weeks prior to trial participation etc.
* Subjects who have received live vaccines or attenuated vaccines within 4 weeks prior to study participation
* Subjects with active HBV or HCV, or HIV positive
* Subjects with severe infections requiring antibiotics, antifungal agents, or antiviral agents, or those with uncontrolled active infectious diseases
* Subjects with any form of primary immunodeficiency or active autoimmune diseases
* Subjects who have previously received intratumoral therapy for the target lesion
* Ineligibility or inability to participate in the study at the investigator's discretion

Primary outcomes:
measure: Incidence of treatment-related serious adverse event (SAE) rate
timeFrame: Observation for 12 weeks following a single dose
Secondary outcomes:
measure: Objective response rate (ORR)
timeFrame: Observation for 12 weeks following a single dose
measure: Progression-free survival (PFS)
timeFrame: Observation for 12 weeks following a single dose
measure: Target Tumor Size
timeFrame: Observation for 12 weeks following a single dose
Other outcomes:
measure: Immune response assessment
timeFrame: Observation for 12 weeks following a single dose
measure: Viral Shedding Profile Assessment
timeFrame: Observation for 12 weeks following a single dose
Locations:
city: Seoul
country: Korea, Republic of
facility: Konkuk University Medical Center
Study officials:
References: