NCT07131722

Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II

An Open-Label, Dose-Finding, Phase 1/2a Study to Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: PRG Science & Technology Co., Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to learn if Trineumin(Code name:PRG-N-01) works to treat Neurofibromatosis Type II(NF2) in adults. It will also learn about the safety and tolerability and toxicity of PRG-N-01. The main questions it aims to answer are: * What dose was determined as the Maximum Tolerated Dose (MTD) of Trineumin? * What dose was explored as the optimal effective dose of Trineumin based on radiographic response? * Does Trineumin reduce tumor size or improve participants' quality of life, including hearing function? * What medical problems do participants have when taking Trineumin? Participants will: * Take Trineumin every day for 96 weeks * Visit the clinic once 1, 4, 8, 12, 18week and every 12 weeks and for checkups and tests

Detailed description

This is an open-label, Phase 1/2a clinical study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of Trineumin(Code name:PRG-N-01) in subjects with Neurofibromatosis Type II(NF2)-related tumors Phase 1 Subjects who voluntarily provide written informed consent will be screened according to predefined inclusion and exclusion criteria. The Phase 1 portion is designed to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of Trineumin. A total of six dose levels are planned. Dose escalation follows an accelerated titration design (ATD) for Cohort 1, a standard 3+3 design for Cohort 2, and a rolling six design for subsequent cohorts. Trineumin is administered orally once daily. Each subject is assigned to a cohort in the order of enrollment. The dose-limiting toxicity (DLT) observation period is 12 weeks following initial dosing. Subjects without DLT or who recover from DLT during this period may continue treatment. Phase 2a In the Phase 2a portion, eligible subjects who provide informed consent will be randomized to receive one of two selected doses of Trineumin orally once daily. Randomization is stratified based on predefined criteria.

Conditions

• Neurofibromatosis Type II
• NF2

Interventions

Type	Name	Description
DRUG	Trineumin	Trineumin(Code name: PRG-N-01) is administered orally once daily. The study includes six dose levels. Dose escalation decisions are based on observed DLTs.
DRUG	Trineumin	Trineumin(Code name: PRG-N-01) is administered orally once daily at the lower dose selected from Phase 1 results.
DRUG	Trineumin	Trineumin(Code name: PRG-N-01) is administered orally once daily at the higher dose selected from Phase 1 results.

Timeline

• Start date: 2026-07-01
• Primary completion: 2028-07-01
• Completion: 2028-07-01
• First posted: 2025-08-20
• Last updated: 2026-01-23

Regulatory

• FDA-regulated drug study