Solid TumorPhase 1/2NCT07245901
Safety and Tolerability of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination With Anti-PD-1 Monoclonal Antibody in Patients With Advanced Solid Tumors
A Phase 1/2 study testing circFAM53B mRNA vaccine and toripalimab in people with Solid Tumor, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University.
Trial summary
This is an Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination with anti-PD-1 monoclonal antibody in Patients with Advanced Solid Tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Solid Tumor
- What is being tested?
- circFAM53B mRNA vaccine and toripalimab
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 5 Dec 2025to 5 Dec 2030
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntary participation: The patient voluntarily agrees to participate in the study, signs a written informed consent form (ICF), and is willing and able to comply with the study protocol requirements.
- Age and sex: Male or female patients aged ≥18 years.
- Life expectancy: Expected survival of ≥3 months.
- Diagnosis: Histologically or cytologically confirmed unresectable or metastatic advanced solid malignancies, including but not limited to pancreatic cancer, breast cancer, non-small cell lung cancer, melanoma, and colorectal cancer.
- Tumor tissue availability: Patients must have adequate fresh tumor tissue obtained via biopsy or surgery prior to treatment, or available formalin-fixed paraffin-embedded (FFPE) samples. The expression of circFAM53B-219aa in the specimen must be confirmed by RT-qPCR analysis and/or immunohistochemistry.
- HLA genotyping will be performed using peripheral blood samples, employing polymerase chain reaction-sequence-based typing (PCR-SBT) combined with Sanger sequencing, with allele assignment referenced to the IMGT/HLA database, or alternatively, using PCR with sequence-specific oligonucleotide (SSO) probes, sequence-specific primers (SSP), or next-generation sequencing (NGS) technologies. Subsequently, NetMHCpan and MHCflurry predictive models will be applied to identify tumor-specific circFAM53B-39aa-derived peptides that bind to HLA class I molecules (HLA-A, -B, and -C) and to evaluate their binding affinity. Patients with HLA genotypes showing a predicted binding rank value <2 will be eligible for enrollment.
Show all 30Show fewer
- Measurable disease: At least one measurable lesion or bone-only lesion as defined by RECIST v1.1 (see Appendix 4: Response Evaluation Criteria in Solid Tumors).
- Prior therapy: Patients who have failed standard therapy, have no available standard treatment, or cannot tolerate standard treatment-related toxicities.
- Performance status: ECOG performance status 0-2.
- Adequate organ function as defined below:
- Hematology:
- Absolute neutrophil count (ANC) ≥ 1.5 × 10⁹/L;
- Platelet count (PLT) ≥ 75 × 10⁹/L;
- Hemoglobin (Hb) ≥ 90 g/L.
- Hepatic function:
- AST, ALT, and alkaline phosphatase ≤ 2.5 × ULN; total bilirubin (TBIL) ≤ 1.5 × ULN.
- Exceptions:
- Patients with confirmed liver metastases: AST and/or ALT ≤ 5 × ULN;
- Patients with confirmed liver or bone metastases: alkaline phosphatase ≤ 5 × ULN;
- Patients with confirmed Gilbert's syndrome: total bilirubin ≤ 3.0 mg/dL.
- Renal function: Serum creatinine ≤ 1.5 × ULN.
- Coagulation: APTT ≤ 1.5 × ULN, and INR or PT ≤ 1.5 × ULN.
- Cardiac function: Left ventricular ejection fraction (LVEF) ≥ 50% by echocardiography.
- Pulmonary function: FEV₁ ≥ 60%.
- Contraception: Fertile patients who are not surgically sterile must agree to use at least one medically approved contraceptive method (e.g., intrauterine device, oral contraceptives, or condoms) during the study treatment period and for 1 year after study completion. Female patients of childbearing potential must have a negative serum hCG test within 7 days prior to cell injection.
- Recovery from prior therapy: Any toxicities from previous treatments must have resolved to CTCAE v5.0 Grade ≤1 or met protocol-specified laboratory criteria, except for:
- Grade 2 peripheral neuropathy, alopecia, or vitiligo;
- Hypothyroidism controlled with hormone replacement therapy;
- Type I diabetes well controlled with insulin;
- Other irreversible toxicities deemed by the investigator unlikely to worsen with vaccination.
Who cannot join
- Participants meeting any of the following criteria will be excluded from the study:
- Active infection requiring systemic therapy.
- Uncontrolled tumor-associated pain, as determined by the investigator. Patients requiring analgesic therapy must be on a stable pain management regimen prior to enrollment. Symptomatic lesions amenable to palliative radiotherapy should be treated before study entry.
- Severe cardiac disease or cardiac dysfunction expected to preclude tolerance to treatment, including but not limited to: life-threatening arrhythmias or high-grade atrioventricular block, unstable angina, clinically significant valvular heart disease, electrocardiographic evidence of transmural myocardial infarction, or uncontrolled hypertension.
- Receipt of radiotherapy, chemotherapy, or endocrine therapy within 4 weeks prior to enrollment, or current participation in another interventional clinical trial.
- Pregnant or breastfeeding women.
Show all 20Show fewer
- Active autoimmune disease, a history of autoimmune disease, or conditions requiring systemic corticosteroids or immunosuppressive therapy (>10 mg/day prednisone or equivalent).
- Exceptions: hormone replacement therapy (e.g., thyroxine, insulin, or physiological corticosteroid replacement for adrenal or pituitary insufficiency) is permitted.
- History of congenital or acquired immunodeficiency, including HIV seropositivity.
- Active tuberculosis (TB) infection within 1 year prior to enrollment, or a history of active TB infection more than 1 year ago that was not adequately treated.
- Active hepatitis B or C infection.
- Patients who are HBsAg or HBcAb positive may be enrolled if HBV DNA is below the lower limit of normal (LLN) at the study site.
- Patients who are HCV antibody positive may be enrolled if HCV RNA is below the LLN at the study site.
- Carriers participating in the study should receive antiviral therapy as appropriate, with periodic quantitative nucleic acid testing during the study.
- Active syphilis.
- Receipt of any form of vaccination within 4 weeks prior to enrollment, or planned vaccination during the study period.
- Previous allogeneic bone marrow transplantation or solid organ transplantation.
- History of hypersensitivity or allergic reactions to any component of the investigational products, including but not limited to the mRNA vaccine, toripalimab, or their formulation excipients.
- Concurrent use of prohibited concomitant medications, such as systemic corticosteroids or live vaccines, unless discontinued at least 7 days prior to enrollment.
- Any other condition that, in the investigator's judgment, may compromise patient safety, treatment compliance, or study integrity.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination With Anti-PD-1 Monoclonal Antibody in Patients With Advanced Solid Tumors
This is an Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination with anti-PD-1 monoclonal antibody in Patients with Advanced Solid Tumors. Preclinical studies of this project have identified that circFAM53B, which is specifically overexpressed in tumor cells, possesses the ability to encode a cryptic peptide, FAM53B-219aa, representing a potential tumor vaccine target. In this study, the sequence encoding the cryptic antigen from circFAM53B will be linearized and formulated as an mRNA vaccine (circFAM53B mRNA injection). The vaccine will be administered via lipid nanoparticle (LNP) encapsulation, and its safety and efficacy will be evaluated in combination with anti-PD-1 monoclonal antibody therapy.
Read the detailed description
This investigator-initiated clinical trial is designed to evaluate the safety and preliminary efficacy of the circFAM53B mRNA vaccine in combination with anti-PD-1 monoclonal antibody therapy using a "3+3" dose-escalation design. The trial consists of two phases: a dose-escalation phase and a dose-expansion phase.
In the dose-escalation phase, patients with advanced solid tumors exhibiting high circFAM53B-219aa expression who have failed prior standard therapies will be enrolled. Approximately 10-20 participants will receive the circFAM53B mRNA vaccine to determine the safety profile (DLT) and identify the maximum tolerated dose (MTD).
Based on the results from the dose-escalation phase, the dose-expansion phase will be conducted at the identified MTD. This phase will enroll participants from two different tumor types, with approximately 10 patients per cohort, to receive the circFAM53B mRNA vaccine in combination with toripalimab.
- Conditions
- Solid Tumor
- Being tested
- circFAM53B mRNA vaccine, toripalimab
- Keywords
- Tumor vaccine, mRNA vaccine, solid tumor, immunotherapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
circFAM53B mRNA vaccineDrug
Also called circFAM53B mRNA injection
IM injection on Day 1 of each 21-day cycle for up to 9 cycles
toripalimabDrug
Also called anti-PD-1 monoclonal antibody
Intravenous infusion once every 21 days
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation phase | Experimental | A single dose of the PD-1 monoclonal antibody will be administered first, followed by combination treatment with the mRNA vaccine and the PD-1 antibody 21 days later. Participants will receive circFAM53B mRNA injection via an intramuscular (IM) injection on Day 1 of each 21-day cycle for up to 9 cycles, combined with PD-1 monoclonal antibody therapy for up to 35 cycles. Receives: Drug: circFAM53B mRNA vaccine; Drug: toripalimab |
| Dose Expansion phase | Experimental | A single dose of the PD-1 monoclonal antibody will be administered first, followed by combination treatment with the mRNA vaccine and the PD-1 antibody 21 days later. Participants will receive circFAM53B mRNA injection via an intramuscular (IM) injection on Day 1 of each 21-day cycle for up to 9 cycles, combined with PD-1 monoclonal antibody therapy for up to 35 cycles. Receives: Drug: circFAM53B mRNA vaccine; Drug: toripalimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Safety and tolerability as determined by assessment of dose limiting toxicities and the maximum tolerated dose or maximal assessed dose per protocol of the vaccine with cancers.
Measured: 12 months
Recommended Phase 2 Dose (RP2D)
To determine a recommended phase 2 dose of the vaccine for further development by evaluating number of patients with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0)
Measured: 12 months
Maximum Tolerated Dose (MTD)
Determine the maximum tolerated dose by assessing the Incidence of Dose Limiting Toxicities (DLTs), treatment emergent and treatment related adverse events (assessed by CTCAE v5.0).
Measured: 12 months
Secondary outcomes (4)
Overall Response Rate (ORR): Number of Participants with Tumor Response (Partial or Complete)
ORR is defined as the proportion of participants whose best overall response is complete response (CR) or partial response (PR).
Measured: Baseline through disease progression by Response Evaluation Criteria of Solid Tumors Version 1.1 (RECIST 1.1), start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Duration of Response (DoR)
DoR is defined as time from first tumor response (partial or complete) until either radiological disease progression, clinical/symptomatic disease progression or death (whichever is sooner).
Measured: Baseline through disease progression by RECIST 1.1, start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Progression Free Survival (PFS)
PFS is defined as time between the date of first dose of Toripalimab and the date of either radiological disease progression, clinical/symptomatic disease progression or death (whichever is sooner).
Measured: Baseline through disease progression by RECIST 1.1, start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Overall Survival (OS)
OS is defined as time between the date of the first dose of study drug and the date of death due to any cause.
Measured: Baseline to death of any cause (up to approximately 3 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2025
- Study start5 Dec 2025 (estimated)
- Primary completion5 Dec 2028 (estimated)
- Study completion5 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Solid TumorStudies of the same condition, open and under way.
- More trials run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Zenith Institute of Medical Sciences, Guangzhou
- Responsible party
- Principal investigator, Erwei Song, M.D., Ph.D., Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
- Investigators
- Erwei Song, Study chair, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07245901
- Sponsor's protocol ID
- SYSKY-2025-591-02
- Status last verified
- Oct 2025
- First submitted
- 17 Nov 2025
- Last updated
- 24 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination With Anti-PD-1 Monoclonal Antibody in Patients With Advanced Solid Tumors
- ClinicalTrials.gov ID
- NCT07245901
- Acronym / sponsor ID
- SYSKY-2025-591-02
- Conditions
- Solid Tumor
- Keywords
- Tumor vaccine, mRNA vaccine, solid tumor, immunotherapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityUniversity, hospital or other
- Collaborators
- Zenith Institute of Medical Sciences, Guangzhou
- Study start
- 5 Dec 2025 (estimated)
- Primary completion
- 5 Dec 2028 (estimated)
- Study completion
- 5 Dec 2030 (estimated)
- First posted
- 24 Nov 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 Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination with anti-PD-1 monoclonal antibody in Patients with Advanced Solid Tumors. Preclinical studies of this project have identified that circFAM53B, which is specifically overexpressed in tumor cells, possesses the ability to encode a cryptic peptide, FAM53B-219aa, representing a potential tumor vaccine target. In this study, the sequence encoding the cryptic antigen from circFAM53B will be linearized and formulated as an mRNA vaccine (circFAM53B mRNA injection). The vaccine will be administered via lipid nanoparticle (LNP) encapsulation, and its safety and efficacy will be evaluated in combination with anti-PD-1 monoclonal antibody therapy.
Detailed description
This investigator-initiated clinical trial is designed to evaluate the safety and preliminary efficacy of the circFAM53B mRNA vaccine in combination with anti-PD-1 monoclonal antibody therapy using a "3+3" dose-escalation design. The trial consists of two phases: a dose-escalation phase and a dose-expansion phase.
In the dose-escalation phase, patients with advanced solid tumors exhibiting high circFAM53B-219aa expression who have failed prior standard therapies will be enrolled. Approximately 10-20 participants will receive the circFAM53B mRNA vaccine to determine the safety profile (DLT) and identify the maximum tolerated dose (MTD).
Based on the results from the dose-escalation phase, the dose-expansion phase will be conducted at the identified MTD. This phase will enroll participants from two different tumor types, with approximately 10 patients per cohort, to receive the circFAM53B mRNA vaccine in combination with toripalimab.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation phase | Experimental | Drug: circFAM53B mRNA vaccine, Drug: toripalimabA single dose of the PD-1 monoclonal antibody will be administered first, followed by combination treatment with the mRNA vaccine and the PD-1 antibody 21 days later. Participants will receive circFAM53B mRNA injection via an intramuscular (IM) injection on Day 1 of each 21-day cycle for up to 9 cycles, combined with PD-1 monoclonal antibody therapy for up to 35 cycles. |
| Dose Expansion phase | Experimental | Drug: circFAM53B mRNA vaccine, Drug: toripalimabA single dose of the PD-1 monoclonal antibody will be administered first, followed by combination treatment with the mRNA vaccine and the PD-1 antibody 21 days later. Participants will receive circFAM53B mRNA injection via an intramuscular (IM) injection on Day 1 of each 21-day cycle for up to 9 cycles, combined with PD-1 monoclonal antibody therapy for up to 35 cycles. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (30 listed)
- Voluntary participation: The patient voluntarily agrees to participate in the study, signs a written informed consent form (ICF), and is willing and able to comply with the study protocol requirements.
- Age and sex: Male or female patients aged ≥18 years.
- Life expectancy: Expected survival of ≥3 months.
- Diagnosis: Histologically or cytologically confirmed unresectable or metastatic advanced solid malignancies, including but not limited to pancreatic cancer, breast cancer, non-small cell lung cancer, melanoma, and colorectal cancer.
- Tumor tissue availability: Patients must have adequate fresh tumor tissue obtained via biopsy or surgery prior to treatment, or available formalin-fixed paraffin-embedded (FFPE) samples. The expression of circFAM53B-219aa in the specimen must be confirmed by RT-qPCR analysis and/or immunohistochemistry.
Key exclusion criteria (20 listed)
- Active infection requiring systemic therapy.
- Uncontrolled tumor-associated pain, as determined by the investigator. Patients requiring analgesic therapy must be on a stable pain management regimen prior to enrollment. Symptomatic lesions amenable to palliative radiotherapy should be treated before study entry.
- Severe cardiac disease or cardiac dysfunction expected to preclude tolerance to treatment, including but not limited to: life-threatening arrhythmias or high-grade atrioventricular block, unstable angina, clinically significant valvular heart disease, electrocardiographic evidence of transmural myocardial infarction, or uncontrolled hypertension.
- Receipt of radiotherapy, chemotherapy, or endocrine therapy within 4 weeks prior to enrollment, or current participation in another interventional clinical trial.
- Pregnant or breastfeeding women.
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 |
|---|---|---|
| circFAM53B mRNA vaccineAlso: circFAM53B mRNA injectionIM injection on Day 1 of each 21-day cycle for up to 9 cycles | Drug | Dose Escalation phase, Dose Expansion phase |
| toripalimabAlso: anti-PD-1 monoclonal antibodyIntravenous infusion once every 21 days | Drug | Dose Escalation phase, Dose Expansion phase |
Primary outcome
Safety and Tolerability
Time frame: 12 months
Safety and tolerability as determined by assessment of dose limiting toxicities and the maximum tolerated dose or maximal assessed dose per protocol of the vaccine with cancers.
Recommended Phase 2 Dose (RP2D)
Time frame: 12 months
To determine a recommended phase 2 dose of the vaccine for further development by evaluating number of patients with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0)
Maximum Tolerated Dose (MTD)
Time frame: 12 months
Determine the maximum tolerated dose by assessing the Incidence of Dose Limiting Toxicities (DLTs), treatment emergent and treatment related adverse events (assessed by CTCAE v5.0).
4 secondary outcomes
Overall Response Rate (ORR): Number of Participants with Tumor Response (Partial or Complete)
Time frame: Baseline through disease progression by Response Evaluation Criteria of Solid Tumors Version 1.1 (RECIST 1.1), start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Duration of Response (DoR)
Time frame: Baseline through disease progression by RECIST 1.1, start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Progression Free Survival (PFS)
Time frame: Baseline through disease progression by RECIST 1.1, start of new anti-cancer therapy, withdrawal of consent, death and last safety follow-up visit (up to approximately 3 years)
Overall Survival (OS)
Time frame: Baseline to death of any cause (up to approximately 3 years)
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 24 Nov 2025.
- Individual participant dataSharing planned: No
Related trials for Solid Tumor
RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
Active, not recruitingPhase 1
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK043 in Patients With Advanced Solid Tumor
Neoplasms
Active, not recruitingPhase 3
A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial
Solid Tumors
RecruitingPhase 1
IL-12 Genetically Engineered Myeloid Cells in Participants With Relapsed, Refractory Solid Tumors
Relapsed Solid Tumor Malignancies, Refractory Solid Tumor Malignancies
RecruitingObservational study
Immune Checkpoint Inhibitor Toxicity Risk Prediction in Solid Tumors
Malignant Solid Neoplasm
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.
Erwei Song study chair
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing circFAM53B mRNA vaccine and toripalimab in people with Solid Tumor, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University.
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 30 inclusion and 20 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 24 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Huang Di (0086-020-8133 2199, huangd63@mail.sysu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: 12 months). The study lists 3 primary outcomes in all. 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 24 Nov 2025.