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
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).
Uncontrolled hypertension at screening (DBP ≥100 mmHg or SBP ≥160 mmHg despite treatment)
Acute coronary syndrome (ACS) within 24 weeks of baseline, clinically significant arrhythmia, cardiomyopathy, unstable angina, NYHA II-IV heart failure, or severe valvular heart disease
Interstitial lung disease or pulmonary fibrosis
Cystitis or urinary obstruction within 12 weeks prior to screening
Sequential
Trineumin
DrugTrineumin(Code name: PRG-N-01) is administered orally once daily. The study includes six dose levels. Dose escalation decisions are based on observed DLTs.
Trineumin
DrugTrineumin(Code name: PRG-N-01) is administered orally once daily at the lower dose selected from Phase 1 results.
Trineumin
DrugTrineumin(Code name: PRG-N-01) is administered orally once daily at the higher dose selected from Phase 1 results.
Phase 1_Incidence of Dose-Limiting Toxicities (DLTs)
Incidence of DLTs will be assessed to determine the MTD and RP2D based on predefined criteria.
Phase 1_Maximum Tolerated Dose (MTD)
it determine the MTD based on predefined criteria.
Phase 1_ Recommended Phase 2 Dose (RP2D)
it determine the RP2D based on predefined criteria.
Phase2a_Maximum tumor size change rate of Radiographic Tumor Response
Based on best overall response (BOR), ORR, DOR, PFS, and changes in tumor size from baseline using up to 4 target lesions.
Phase1_Progression-free survival (PFS) of Radiographic Tumor Response
Phase1_Duration of response (DOR) of Radiographic Tumor Response
Phase1_Objective response rate (ORR) of Radiographic Tumor Response
Phase1_best overall response (BOR) of Radiographic Tumor Response
Phase2a_ Exploratory Analysis of Biomarker Expression in Blood
Blood samples will be collected and stored during Phase 2a for exploratory analysis of biomarkers.
Phase 1,2a_ AUC last of Day1
Phase 1,2a_ AUCinf of Day1
Phase 1,2a_ Cmax of Day1
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This trial is not recruiting at the moment. You can still explore other options:
