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NCT06834438

Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002

Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences

View on ClinicalTrials.gov

Summary

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.

Official title: 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

Key Details

Gender

All

Age Range

16 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2025-02-15

Completion Date

2027-02-01

Last Updated

2025-02-19

Healthy Volunteers

Conditions

NF2 Deficiency

Interventions

GENETIC

ST002

Single multi-point intra-tumoral injection of ST002, a lentiviral vector designed to drive expression of the NF2 protein product, Merlin.

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.

Locations (1)

Shuhang Wang

Beijing, Beijing Municipality, China