NCT07131722

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

Not yet recruiting Phase 1/Phase 2 Last updated 21 January 2026

What this trial tests

Phase 1/Phase 2 trial testing Trineumin in Neurofibromatosis Type II in 25 participants. Not yet recruiting.

Timeline

1 July 2026

Primary endpoint
1 July 2028

1 July 2028

Quick facts

Lead sponsor	PRG Science & Technology Co., Ltd.
Phase	Phase 1/Phase 2
Status	Not yet recruiting
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	25
Start date	1 July 2026
Primary completion	1 July 2028
Estimated completion	1 July 2028

Drugs / interventions tested

• Trineumin — full drug profile →
• Trineumin — full drug profile →
• Trineumin — full drug profile →

Conditions studied

• Neurofibromatosis Type II — all drugs for Neurofibromatosis Type II →
• NF2 — all drugs for NF2 →

Sponsor

PRG Science & Technology Co., Ltd. — full company profile →

Who can join

18 and older, any sex, with Neurofibromatosis Type II or NF2. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Phase 1_Incidence of Dose-Limiting Toxicities (DLTs)
  Time frame: Each treatment group at the 12-week time point after IP administration
  Incidence of DLTs will be assessed to determine the MTD and RP2D based on predefined criteria.
• Phase 1_Maximum Tolerated Dose (MTD)
  Time frame: Each treatment group at the 12-week time point after IP administration
  it determine the MTD based on predefined criteria.
• Phase 1_ Recommended Phase 2 Dose (RP2D)
  Time frame: Each treatment group at the 12-week time point after IP administration
  it determine the RP2D based on predefined criteria.
• Phase2a_Maximum tumor size change rate of Radiographic Tumor Response
  Time frame: From baseline excluding week 24 to week 96 at 12-week intervals
  Based on best overall response (BOR), ORR, DOR, PFS, and changes in tumor size from baseline using up to 4 target lesions.
• Phase2a_best overall response (BOR) of Radiographic Tumor Response
  Time frame: From baseline excluding week 24 to week 96 at 12-week intervals
  Based on best overall response (BOR), ORR, DOR, PFS, and changes in tumor size from baseline using up to 4 target lesions.
• Phase2a_Objective response rate (ORR) of Radiographic Tumor Response
  Time frame: From baseline excluding week 24 to week 96 at 12-week intervals
  Based on best overall response (BOR), ORR, DOR, PFS, and changes in tumor size from baseline using up to 4 target lesions.

Sponsor's own description

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

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Vestibular schwannoma: genetic and epigenetic mechanisms, hearing loss, and emerging therapies.
   Otaner F, Himic V, Vargas LO, Abikenari M, et al · · 2026 · PMID 42143176 · DOI 10.1007/s11060-026-05621-4