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 range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. 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)
. 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)
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals
8
Phase2a_Progression-free survival (PFS) of Radiographic Tumor Response
Timeframe: From baseline excluding week 24 to week 96 at 12-week intervals