A Study Evaluating the Safety and Efficacy of PRG-N-01 in Patients with Neurofibromatosis Type II (NF2)
- Conditions
- Congenital malformations, deformations and chromosomal abnormalities
- Registration Number
- KCT0009520
- Lead Sponsor
- PRG S&Tech
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 36
1. Common to phase 1 and 2a
1) Adult men and women aged 19 years or older at the time of providing written consent.
2) Individuals clinically or genetically diagnosed with NF2 according to the updated I-NF-DC 2022 diagnostic criteria.
3) Tumors caused by NF2 meeting one or more of the following criteria and requiring treatment but deemed ineligible for surgical intervention due to the high risk of surgical side effects (such as nerve function impairment):
(1) Those with progressive tumors (vestibular schwannomas, non-vestibular schwannomas, meningiomas, ependymomas) confirmed by MRI within 36 months before screening.
(2) Subjects exhibiting clinical symptoms (such as deteriorating function of the affected nerve, such as hearing loss, uncontrolled pain, difficulty breathing, swallowing difficulties, decreased motor function, and impaired gait) as determined by the investigator.
4) ECOG performance status 0 – 1
5) Individuals whose hematological, liver, kidney, and blood coagulation functions were confirmed to be appropriate according to the following criteria at the time of screening.
6) Individuals with appropriate heart and lung function confirmed by the following criteria at the screening.
7) Individuals who agree to use sunscreen during the clinical trial period.
8) A test subject (or a test subject's representative) who voluntarily agrees and provides written consent to participate in this clinical trial.
2. Phase 2a
1) Patients with at least one measurable tumor due to NF2 confirmed on MRI at baseline.
1. Common to phase 1 and 2a
1) Individuals with the following past or current medical history confirmed at the time of screening:
(1) Malignant tumor requiring treatment (chemotherapy or radiation therapy) or with disease progression within 2 years of screening.
(2) The following heart-related history:
- Uncontrolled hypertension at screening (DBP 100 mmHg or higher or SBP 160 mmHg or higher despite treatment).
- Acute coronary syndrome (ACS), clinically significant arrhythmia, cardiomyopathy, unstable angina, NYHA stage II-IV heart failure, severe valvular heart disease within 24 weeks of baseline.
(3) Interstitial lung disease or pulmonary fibrosis.
(4) Cystitis or urinary obstruction within 12 weeks of screening.
(5) Blood coagulation disorders.
(6) Severe or active infectious disease requiring administration of antibiotics, antivirals, etc., within 4 weeks of baseline.
(7) Gastrointestinal disease that currently presents difficulties in oral administration or may affect absorption (celiac disease, Crohn's disease, intestinal resection, etc.).
(8) Other diseases deemed to affect clinical trial results according to the judgment of the investigator.
2) Individuals with confirmed or requiring the following drug treatment history:
(1) Chemotherapy, immunotherapy, myelosuppressive chemotherapy within 4 weeks of screening (however, in the case of nitrosourea or mitomycin C, registration is possible after more than 6 weeks have elapsed).
(2) Monoclonal antibody therapy within 12 weeks of screening (however, registration is possible if more than 3 times the half-life has elapsed), stem cell transplantation (however, registration is possible if there is no evidence of active graft-versus-host disease and more than 12 weeks have passed since transplantation).
(3) Other investigational drugs or investigational medical devices within 4 weeks of baseline.
(4) Corticosteroids such as prednisone, prednisolone, etc., within 1 week of baseline (only permitted if treated with low dose).
(5) Strong inhibitor, inducer, or substrate of transporter (OCT2, MATE1, MATE2-K) of CYP enzymes (CYP2C8, CYP3A) (however, registration is possible if the period has elapsed, and the half-life is more than 5 times that of the baseline).
3) Individuals who have undergone major surgery within 4 weeks of baseline (however, minor surgical procedures such as catheter replacement therapy and local biopsy are allowed).
4) Individuals who have received radiation therapy for the purpose of treating tumors caused by NF2 within 24 weeks of baseline or who require whole-body radiation therapy during the clinical trial period.
5) Individuals with implants or orthopedic braces that may interfere with volumetric analysis of target lesions through MRI.
6) Individuals with known severe hypersensitivity to PRG-N-01 or concomitant drugs or components, or a history of allergic reactions due to compounds of similar chemical or biological composition.
7) Pregnant or lactating women.
8) Women or men of childbearing potential who are unwilling to use appropriate contraception from the date of written consent until 12 weeks after the last dose of PRG-N-01.
9) Other individuals deemed unsuitable for participation in this clinical trial according to the judgment of the investigator.
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method