Non-Small Cell Lung CancerPhase 1NCT06934590
Evaluate Safety and Tolerability of RX001 in Patients With KRAS Mutant Advanced NSCLC
A Phase 1 study testing RX001 in people with Non-Small Cell Lung Cancer, run by GeneCraft Inc. at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Non-Small Cell Lung Cancer
- What is being tested?
- RX001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteSouth Korea
- Study dates
- May 2025to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Subjects must not meet any of the following conditions:
- Subjects with a history of or current comorbidities including:
- Hematologic malignancies, such as lymphoma, or any other malignant tumors apart from the indication of this study.
- Subjects with the cardiovascular conditions such as unstable angina and/or myocardial infarction within 1 year prior to screening
- Subjects with the pulmonary baseline conditions:
- Blood coagulation disorder
Show all 16Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea1 site
Konkuk University Medical Center
Seoul, 05030
Lee, Professor, 82 02 2030-7521, kyleemd@kuh.ac.kr
About this study
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
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.
- Conditions
- Non-Small Cell Lung Cancer
- Being tested
- RX001
- Keywords
- KRAS, NSCLC, RUNX3, AAV
- Medical subject headings
- Carcinoma, Non-Small-Cell Lung
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RX001Drug
Subjects will receive a single RX001 (AAV-hRUNX3) treatment via Intra-tumoral injection
| Study group | Role | What they receive |
|---|---|---|
| RX001 | Experimental | RX001 Receives: Drug: RX001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-related serious adverse event (SAE) rate
Incidence of SAEs throughout the study
SAE: an adverse event or suspected adverse reaction is considered "serious" if, in the view of the investigator, it results in any of the following outcomes:
Death or a life-threatening adverse event Inpatient hospitalization or prolongation of existing hospitalization A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions A congenital anomaly/birth defect Any other important medical event that does not fit the criteria above but, based upon appropriate medical judgment, may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above
Measured: Observation for 12 weeks following a single dose
Secondary outcomes (3)
Objective response rate (ORR)
- ORR = percentage of participants with complete response + partial response.
- Complete response (CR): Disappearance of all target lesions.
- Partial response (PR): At least a 30% decrease in the diameters of target lesions, taking as reference the baseline diameters.
Measured: Observation for 12 weeks following a single dose
Progression-free survival (PFS)
PFS is defined as the time from date of treatment initiation to disease progression or death from any cause, whichever occurs first.
Measured: Observation for 12 weeks following a single dose
Target Tumor Size
Changes in the maximum length of the target lesion
Measured: Observation for 12 weeks following a single dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2025
- Study startMay 2025 (estimated)
- Primary completionSep 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-Small Cell Lung CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GeneCraft Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06934590
- Sponsor's protocol ID
- GC-LCP1001
- Status last verified
- Apr 2025
- First submitted
- 2 Apr 2025
- Last updated
- 23 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The current position is "No" due to pending internal policy decisions. However, revision of internal regulations is under review to allow for potential future data sharing and registry compliance.
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06934590
- Acronym / sponsor ID
- GC-LCP1001
- Conditions
- Non-Small Cell Lung Cancer
- Keywords
- KRAS, NSCLC, RUNX3, AAV
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- GeneCraft Inc.Industry (pharma, biotech, devices)
- Study start
- May 2025 (estimated)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 18 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RX001 | Experimental | Drug: RX001RX001 |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (16 listed)
- Subjects must not meet any of the following conditions:
- Subjects with a history of or current comorbidities including:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RX001Subjects will receive a single RX001 (AAV-hRUNX3) treatment via Intra-tumoral injection | Drug | RX001 |
Primary outcome
Incidence of treatment-related serious adverse event (SAE) rate
Time frame: Observation for 12 weeks following a single dose
Incidence of SAEs throughout the study SAE: an adverse event or suspected adverse reaction is considered "serious" if, in the view of the investigator, it results in any of the following outcomes: Death or a life-threatening adverse event Inpatient hospitalization or prolongation of existing hospitalization A persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions A congenital anomaly/birth defect Any other important medical event that does not fit the criteria above but, based upon appropriate medical judgment, may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above
3 secondary outcomes
Objective response rate (ORR)
Time frame: Observation for 12 weeks following a single dose
Progression-free survival (PFS)
Time frame: Observation for 12 weeks following a single dose
Target Tumor Size
Time frame: Observation for 12 weeks following a single dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2025.
- Individual participant dataSharing planned: No. The current position is "No" due to pending internal policy decisions. However, revision of internal regulations is under review to allow for potential future data sharing and registry compliance.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RX001 in people with Non-Small Cell Lung Cancer, run by GeneCraft Inc. at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 23 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kyoungmi Jung, PhD, Seoul (82 2-400-8890, kmjung@genecraft.co.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-related serious adverse event (SAE) rate (time frame: Observation for 12 weeks following a single dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2025.