Clinical trial · Interventional
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
An Open- Label, Single-arm, Dose-escalation Pilot Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ST002 in the Treatment of Patients with NF2 Mutation-related Solid Tumors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. ST002 injection is a gene therapy product designed for NF2. By reinserting the normal XXX gene into genetically deficient tumor cells, the product expresses Merlin. This regulates gene transcription in tumor cells, controls the tumor microenvironment, and inhibits tumor growth and invasion, achieving therapeutic effects.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| NF2 Deficiency | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| ST002 | Genetic | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- ST002
- description
- Subjects will receive with a single multi-point intra-tumoral injection of ST002 from 1x10\^7 to 1x10\^8 TU into the tumor.
- interventionNames
- Genetic: ST002
Primary outcomes (1)
- measure
- Safety evaluation
- timeFrame
- From baseline to Week 24 after administration
- description
- Evaluate the incidence of drug-related adverse events (AEs) and adverse events (SAEs) from baseline to Week 24 after administration, with a focus on neurotoxicity and genome-wide integration carcinogenic risks.
Secondary outcomes (2)
- measure
- Efficacy evaluation
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 16 Years
Show eligibility criteria text
Inclusion Criteria: * age ≥16 years old, gender not limited; * Patients must meet the diagnostic criteria for NF2 gene mutations, which are benign or malignant solid tumors with confirmed NF2 gene mutations or no expression of the NF2 protein product Merlin in peripheral blood or tumor tissue, and no standard treatment or with standard treatment failure; * at least one measurable and injectable superficial lesion (according to RECIST or itRECIST criteria); * Having sufficient organ and bone marrow function: 1. Blood routine (no transfusion or treatment with colony-stimulating factor within 14 days): neutrophil count (ANC) ≥ 1.5 × 10 9 /L, hemoglobin (Hb) ≥ 90g/L, platelet count (PLT) ≥ 75 × 10 9 /L; White blood cell count (WBC)\>3.0 × 10 9 /L; 2. Liver function: Serum total bilirubin (TBIL) ≤ 1.5 × ULN, ALT≤2.5×ULN， AST ≤ 2.5 × ULN (ALT ≤ 5 × ULN, AST ≤ 5 × ULN in patients with liver metastasis); 3. Renal function: serum creatinine (Cr) ≤ 1.5 × ULN, or creatinine clearance rate (CrCl) ≥ 50ml/min; d) Coagulation function: Prothrombin time (PT) ≤ 1.5 × ULN, activated partial thromboplastin time (APTT) ≤ 1.5 × ULN, international normalized ratio (INR) ≤ 1.5 × ULN; * Patients must voluntarily participate in clinical trials, demonstrate adherence to the study protocol, cooperate well with researchers, and sign a written informed consent form. Exclusion Criteria: * Has participated in or is currently participating in other clinical studies NF2 targeting gene therapy; * History of severe neurological disorders such as epilepsy (excluding neurofibromatosis); * Patients who are allergic to lentiviral vectors and their excipients; * Patients with poorly controlled hypertension (systolic blood pressure\>150 mmHg and/or diastolic blood pressure\>100 mmHg under regular medication control), as well as those with a history of hypertensive crisis or hypertensive brain disease; * Suffering from unstable angina or acute myocardial infarction, or having a history of both within the past six months; * Patients with a history of malignant tumors within the past 5 years, except for cured basal cell carcinoma, squamous cell carcinoma of the skin, cervical cancer, and gastrointestinal cancer; * During the study period, premenopausal women who are pregnant, breastfeeding, has a positive pregnancy test, or are unwilling to take contraceptive measures.
References
Publications (0)
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