Neurofibromatosis Type IIPhase 1/2NCT07131722

Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II

A Phase 1/2 study testing Trineumin in people with Neurofibromatosis Type II and NF2, run by PRG Science & Technology Co., Ltd..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record

Search all trials

Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Neurofibromatosis Type II, NF2
What is being tested?: Trineumin
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 25 participants
Where: No sites listed yet
Study dates: Jul 2026to Jul 2028
Phase: Phase 1/2
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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).
Subjects with progressive tumors (VS, non-VS, meningiomas, ependymomas) confirmed on MRI within 36 months prior to screening (2) Subjects with clinical symptoms (decreased function of affected nerves, such as hearing loss, uncontrolled pain, shortness of breath, difficulty swallowing, decreased motor function, and decreased gait) as judged by the investigator 4) Subjects with ECOG performance status 0 - 1 or Karnofsky performance status 70 or higher 5) Subjects who have appropriate hematological, liver, renal, and blood coagulation functions confirmed based on the following criteria at screening: 6) Subjects who have appropriate cardiac and pulmonary functions confirmed based on the following criteria at screening: 7) Subjects who agree to use sunscreen during the clinical study period. 8) Subjects (or the subject's legal representative) who voluntarily consent and provide written informed consent to participate in this clinical study.
9) (Only for Phase 2a) Subjects with one or more measurable NF2-related tumors confirmed on MRI at screening

Who cannot join

1) Subjects who have the following past or current medical history confirmed during screening:
Malignant tumor requiring treatment (chemotherapy or radiotherapy) or with disease progression within 2 years prior to screening (2) The following heart-related history
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

Show all 21Show fewer

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)
Other diseases that are sufficient to affect the clinical study results at the investigator's discretion.
2) Subjects who have confirmed or need the following drug treatments:
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)
Other investigational drugs or devices within 4 weeks prior to screening
Corticosteroids such as prednisone and prednisolone within 1 week prior to screening (but, allowing if treated with low doses)
Substrate of strong inhibitor, inducer, or transporter (OCT2, MATE1, MATE2-K) of CYP enzymes (CYP2C8, CYP3A) (but, allowing only if more than 5 times the half-life from baseline has elapsed) 3) Subjects who performed a major surgery within 4 weeks prior to screening (but, allowing minor surgical procedures such as catheter replacement therapy and local biopsy).
4) Subjects who received radiation therapy for the purpose of treating tumors due to NF2 within 24 weeks prior to screening or who require total body irradiation during the clinical study period.
5) Subjects with prosthetics or orthopedic devices that may interfere with the volumetric analysis of target lesions via MRI.
6) Subjects with a known severe hypersensitivity to PRG-N-01 or a concomitant medication or the ingredients or a history of allergic reactions to compounds of similar chemical or biological composition.
7) Female subjects who are pregnant or breastfeeding 8) Women of childbearing potential or men who are unwilling to use an appropriate method of contraception from the date of written consent to 12 weeks after the last dose of PRG-N-01.
9) Other subjects who are deemed unsuitable for participation in this clinical study at the investigator's discretion

About this study

Official title: 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

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

Read the detailed description

This is an open-label, Phase 1/2a clinical study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of Trineumin(Code name:PRG-N-01) in subjects with Neurofibromatosis Type II(NF2)-related tumors

Phase 1 Subjects who voluntarily provide written informed consent will be screened according to predefined inclusion and exclusion criteria. The Phase 1 portion is designed to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of Trineumin.

A total of six dose levels are planned. Dose escalation follows an accelerated titration design (ATD) for Cohort 1, a standard 3+3 design for Cohort 2, and a rolling six design for subsequent cohorts. Trineumin is administered orally once daily. Each subject is assigned to a cohort in the order of enrollment. The dose-limiting toxicity (DLT) observation period is 12 weeks following initial dosing. Subjects without DLT or who recover from DLT during this period may continue treatment.

Phase 2a In the Phase 2a portion, eligible subjects who provide informed consent will be randomized to receive one of two selected doses of Trineumin orally once daily. Randomization is stratified based on predefined criteria.

Conditions: Neurofibromatosis Type II, NF2
Being tested: Trineumin
Medical subject headings: Neurofibromatosis 2

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TrineuminDrug
Trineumin(Code name: PRG-N-01) is administered orally once daily. The study includes six dose levels.
Dose escalation decisions are based on observed DLTs.
TrineuminDrug
Trineumin(Code name: PRG-N-01) is administered orally once daily at the lower dose selected from Phase 1 results.
TrineuminDrug
Trineumin(Code name: PRG-N-01) is administered orally once daily at the higher dose selected from Phase 1 results.

Study groups in NCT07131722
Study group	Role	What they receive
Trineumin Dose Escalation Arm	Experimental	Subjects receive Trineumin(Code: name: PRG-N-01) orally once daily across six sequential dose cohorts. Dose escalation follows an accelerated titration design (Cohort 1), a 3+3 design (Cohort 2), and a rolling 6 design thereafter. Dosing proceeds unless dose-limiting toxicities (DLTs) are observed. Receives: Drug: Trineumin
Trineumin Low Dose (Phase 2a)	Experimental	Subjects receive Trineumin(Code: name: PRG-N-01) at the selected lower dose (e.g., RP2D or sub-RP2D level) once daily. Subjects are randomized in Phase 2a. Receives: Drug: Trineumin
Trineumin High Dose (Phase 2a)	Experimental	Subjects receive Trineumin(Code: name: PRG-N-01) at a higher dose once daily in Phase 2a. Subjects are randomized to this arm. Receives: Drug: Trineumin

What is being measured

The primary outcome is the main question the study is designed to answer.

Phase 1_Incidence of Dose-Limiting Toxicities (DLTs)
Incidence of DLTs will be assessed to determine the MTD and RP2D based on predefined criteria.
Measured: Each treatment group at the 12-week time point after IP administration
Phase 1_Maximum Tolerated Dose (MTD)
it determine the MTD based on predefined criteria.
Measured: Each treatment group at the 12-week time point after IP administration
Phase 1_ Recommended Phase 2 Dose (RP2D)
it determine the RP2D based on predefined criteria.
Measured: Each treatment group at the 12-week time point after IP administration
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.
Measured: From baseline excluding week 24 to week 96 at 12-week intervals
Phase2a_best overall response (BOR) 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.
Measured: From baseline excluding week 24 to week 96 at 12-week intervals
Phase2a_Objective response rate (ORR) 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.
Measured: From baseline excluding week 24 to week 96 at 12-week intervals
Phase2a_Duration of response (DOR) 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.
Measured: From baseline excluding week 24 to week 96 at 12-week intervals
Phase2a_Progression-free survival (PFS) 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.
Measured: From baseline excluding week 24 to week 96 at 12-week intervals

Secondary outcomes (10)

Phase1_Progression-free survival (PFS) of Radiographic Tumor Response
Measured: From baseline to week 96 at 12-week intervals
Phase1_Duration of response (DOR) of Radiographic Tumor Response
Measured: From baseline to week 96 at 12-week intervals
Phase1_Objective response rate (ORR) of Radiographic Tumor Response
Measured: From baseline to week 96 at 12-week intervals
Phase1_best overall response (BOR) of Radiographic Tumor Response
Measured: From baseline to week 96 at 12-week intervals
Phase1_Maximum tumor size change rate of Radiographic Tumor Response
Measured: From baseline to week 96 at 12-week intervals
Phase1,2a_ Pure Tone Audiometry (PTA) in Functional Hearing Response
Measured: From baseline to week 96 at 12-week intervals
Phase1,2a_Word Recognition Score (WRS) in Functional Hearing Response
Measured: From baseline to week 96 at 12-week intervals
Phase1,2a_Change of NFTI-QOL scores in Quality of Life
Measured: From baseline to week 96 at 12-week intervals
Phase1,2a_Change of PAN-QOL scores in Quality of Life
Measured: From baseline to week 96 at 12-week intervals
Phase1,2a_Incidence and Severity of Adverse Events
adverse events will be recorded and graded according to CTCAE v5.0
Measured: Up to 108 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2026Jul 2028

First posted20 Aug 2025
Study startJul 2026 (estimated)
Primary completionJul 2028 (estimated)
Study completionJul 2028 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Neurofibromatosis Type IIStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Model description: Sequential Assignment, non-Randomized (Phase 1), then Parallel Assignment, Randomized (Phase 2a)
Masking: None (open label)
Enrolment: 25 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07131722
Sponsor's protocol ID: PRG-NF-001
Status last verified: Aug 2025
First submitted: 1 Jul 2025
Last updated: 23 Jan 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Interested in taking part?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

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All Neurofibromatosis Type II trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT07131722
Acronym / sponsor ID: PRG-NF-001
Conditions: Neurofibromatosis Type II, NF2
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Not yet recruiting
Enrolment: 25 participants (estimated)
Lead sponsor: PRG Science & Technology Co., Ltd.Industry (pharma, biotech, devices)
Study start: Jul 2026 (estimated)
Primary completion: Jul 2028 (estimated)
Study completion: Jul 2028 (estimated)
First posted: 20 Aug 2025 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 3
Model description: Sequential Assignment, non-Randomized (Phase 1), then Parallel Assignment, Randomized (Phase 2a)

Arm	Type	Receives
Trineumin Dose Escalation Arm	Experimental	Drug: TrineuminSubjects receive Trineumin(Code: name: PRG-N-01) orally once daily across six sequential dose cohorts. Dose escalation follows an accelerated titration design (Cohort 1), a 3+3 design (Cohort 2), and a rolling 6 design thereafter. Dosing proceeds unless dose-limiting toxicities (DLTs) are observed.
Trineumin Low Dose (Phase 2a)	Experimental	Drug: TrineuminSubjects receive Trineumin(Code: name: PRG-N-01) at the selected lower dose (e.g., RP2D or sub-RP2D level) once daily. Subjects are randomized in Phase 2a.
Trineumin High Dose (Phase 2a)	Experimental	Drug: TrineuminSubjects receive Trineumin(Code: name: PRG-N-01) at a higher dose once daily in Phase 2a. Subjects are randomized to this arm.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

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).
Subjects with progressive tumors (VS, non-VS, meningiomas, ependymomas) confirmed on MRI within 36 months prior to screening (2) Subjects with clinical symptoms (decreased function of affected nerves, such as hearing loss, uncontrolled pain, shortness of breath, difficulty swallowing, decreased motor function, and decreased gait) as judged by the investigator 4) Subjects with ECOG performance status 0 - 1 or Karnofsky performance status 70 or higher 5) Subjects who have appropriate hematological, liver, renal, and blood coagulation functions confirmed based on the following criteria at screening: 6) Subjects who have appropriate cardiac and pulmonary functions confirmed based on the following criteria at screening: 7) Subjects who agree to use sunscreen during the clinical study period. 8) Subjects (or the subject's legal representative) who voluntarily consent and provide written informed consent to participate in this clinical study.
9) (Only for Phase 2a) Subjects with one or more measurable NF2-related tumors confirmed on MRI at screening

Key exclusion criteria (21 listed)

1) Subjects who have the following past or current medical history confirmed during screening:
Malignant tumor requiring treatment (chemotherapy or radiotherapy) or with disease progression within 2 years prior to screening (2) The following heart-related history
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
4) Subjects who received radiation therapy for the purpose of treating tumors due to NF2 within 24 weeks prior to screening or who require total body irradiation during the clinical study period.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TrineuminTrineumin(Code name: PRG-N-01) is administered orally once daily. The study includes six dose levels. Dose escalation decisions are based on observed DLTs.	Drug	Trineumin Dose Escalation Arm
TrineuminTrineumin(Code name: PRG-N-01) is administered orally once daily at the lower dose selected from Phase 1 results.	Drug	Trineumin Low Dose (Phase 2a)
TrineuminTrineumin(Code name: PRG-N-01) is administered orally once daily at the higher dose selected from Phase 1 results.	Drug	Trineumin High Dose (Phase 2a)

Primary outcome

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.
Phase2a_Duration of response (DOR) 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_Progression-free survival (PFS) 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.

10 secondary outcomes

Phase1_Progression-free survival (PFS) of Radiographic Tumor Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1_Duration of response (DOR) of Radiographic Tumor Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1_Objective response rate (ORR) of Radiographic Tumor Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1_best overall response (BOR) of Radiographic Tumor Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1_Maximum tumor size change rate of Radiographic Tumor Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1,2a_ Pure Tone Audiometry (PTA) in Functional Hearing Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1,2a_Word Recognition Score (WRS) in Functional Hearing Response
Time frame: From baseline to week 96 at 12-week intervals
Phase1,2a_Change of NFTI-QOL scores in Quality of Life
Time frame: From baseline to week 96 at 12-week intervals
Phase1,2a_Change of PAN-QOL scores in Quality of Life
Time frame: From baseline to week 96 at 12-week intervals
Phase1,2a_Incidence and Severity of Adverse Events
Time frame: Up to 108 weeks

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2026.

Related trials for Neurofibromatosis Type II

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Trineumin in people with Neurofibromatosis Type II and NF2, run by PRG Science & Technology Co., Ltd..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 23 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1_Incidence of Dose-Limiting Toxicities (DLTs) (time frame: Each treatment group at the 12-week time point after IP administration). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07131722.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2026.