NCT06834438

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

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences · Academic / Other
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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. Using a 3+3 dose escalation design, three dose groups are formulated, and three to six patients are expected to be enrolled in each dose group. A total of nine to eighteen - patients with NF2 gene mutation related solid tumors will be enrolled.

Conditions

• NF2 Deficiency

Interventions

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

Timeline

• Start date: 2025-02-15
• Primary completion: 2026-02-01
• Completion: 2027-02-01
• First posted: 2025-02-19
• Last updated: 2025-02-19

Locations

1 site across 1 country: China