Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002

NCT06834438 | Not Yet Recruiting

• 1 other identifier: ST002-IIT-2024-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

NF2 Deficiency

Outcome Measures

Primary Outcomes (1)

• Safety evaluation

  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.

  From baseline to Week 24 after administration

Secondary Outcomes (2)

• Efficacy evaluation

  Day 21 after administration, and at the end of study follow-up, and at early withdrawal. The frequency of checks during the research period can be adjusted according to the researcher's judgement.

• Efficacy evaluation

  Day 21 after injection

Study Arms (1)

ST002

EXPERIMENTAL

Subjects will receive with a single multi-point intra-tumoral injection of ST002 from 1x10\^7 to 1x10\^8 TU into the tumor.

Genetic: ST002

Interventions

ST002 GENETIC

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

ST002

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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;
• 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);
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

Study Sites (1)

Shuhang Wang

Beijing, Beijing Municipality, 100021, China

Location

Study Officials

• Shuhang Wang, PhD

  National Cancer Center of China

  STUDY DIRECTOR

Central Study Contacts

Ning Li, M.D.

CONTACT

+86 01087788919; lining@cicas.ac.cn

Shuhang Wang, PhD

CONTACT

13581809307; wangshuhang@cicams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Cancer Hospital Chinese Academy of Medical Sciences

Study Record Dates

• First Submitted: February 7, 2025
• First Posted: February 19, 2025
• Study Start: February 15, 2025
• Primary Completion: February 1, 2026
• Study Completion (Estimated): February 1, 2027
• Last Updated: February 19, 2025

Record last verified: 2025-02

Locations

CN (1)