Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Ne… (NCT07131722) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II
25 participantsStarted 2026-07
Plain-language 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
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Male or female subjects aged 18 years or older at screening
✓. Subjects diagnosed with NF2 clinically or genetically, according to the I-NF-DC 2022 updated NF2 diagnostic criteria
✓. Subjects with tumors due to NF2 who require treatment if they meet one of more of the criteria below but cannot undergo surgical treatment due to high risk of side effects from surgery (e.g., damage to nerve function).
Exclusion criteria
✕. Interstitial lung disease or pulmonary fibrosis
✕. Cystitis or urinary obstruction within 12 weeks prior to screening
✕. Blood coagulation disorder
✕. Severe or active infectious disease requiring antibiotics, antivirals, etc. within 4 weeks prior to screening
✕. Gastrointestinal disease that currently makes oral administration difficult or may affect absorption (e.g., celiac disease, Crohn's disease, intestinal resection)
✕
What they're measuring
1
Phase 1_Incidence of Dose-Limiting Toxicities (DLTs)
Timeframe: Each treatment group at the 12-week time point after IP administration
2
Phase 1_Maximum Tolerated Dose (MTD)
Timeframe: Each treatment group at the 12-week time point after IP administration
3
Phase 1_ Recommended Phase 2 Dose (RP2D)
Timeframe: Each treatment group at the 12-week time point after IP administration
4
Phase2a_Maximum tumor size change rate of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals
5
Phase2a_best overall response (BOR) of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals
6
Phase2a_Objective response rate (ORR) of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals
7
Phase2a_Duration of response (DOR) of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals
. Other diseases that are sufficient to affect the clinical study results at the investigator's discretion.
✕. Chemotherapy, immunotherapy, or myelosuppressive chemotherapy within 4 weeks prior to screening (but, within 6 weeks prior to screening for nitrosourea agents or mitomycin C)
✕. Monoclonal antibody therapy within 12 weeks prior to screening (but, allowing if more than 3 half-lives have elapsed); stem cell transplantation (but, allowing if there is no evidence of active graft-versus-host disease and more than 12 weeks have elapsed since transplantation)
Phase2a_Progression-free survival (PFS) of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals