Immuno-NF2 Rescue Study

JRCT ID: jRCT2031240608

Registered date:15/01/2025

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Patients with NF2 diagnosed with progressive schwannoma and HLA-A*24:02
• Date of first enrollment: 21/02/2025
• Target sample size: 26
• Study type: Interventional
• Intervention(s): OCV-101/OTS-102 were emulsified together with incomplete Freund's adjuvant (Montanide ISA-51 VG, SEPPIC, Paris). OCV-101/OTS-102 were injected subcutaneously into infra-axillary and inguinal regions, once a week for 4 weeks and then once a month for 11 months (total of 15 injections over 12 months).

Outcome(s)

• Primary Outcome: Toxicity profile (frequency and severity)

Secondary Outcome

Efficacy endpoint
1) Proportion of subjects with a response of PR, MR, or SD at Visit 16 compared to baseline (Disease control rate)
2) Proportion of subjects with a response of PR, or MR at Visit 16 compared to baseline
3) Improvement rate of hearing at Visit 10 and 16 compared to baseline (WRS:10%<=)
4) Percentage change in target and non-target tumor volume from baseline
5) Percentage change in total tumor volume from baseline (target and non-target vestibular schwannoma)
6) Change in ABR measurements at Visit 16 compared to baseline
7) Change in stabilometry measurements at Visit 16 compared to baseline
8) Change in the following test values at Visit 16 compared to baseline
- EuroQol 5 Dimension (EQ-5D-5L)
- The Penn Acoustic Neuroma Quality-of-Life scale (PANQOL)
- Dizziness Handicap Inventory (DHI)
- Tinnitus Handicap Inventory (THI)
9) The time from start of treatment until disease progression (PD, vestibular schwannoma)

Exploratory endpoint
- PTA

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: <= 79age old
• Gender: Both

Include criteria

1) Written informed consent obtained from participant. For children below the age of 20, a parent(s) or legal guardian(s) can consent to the treatment of the child.
2) Patients who participated in the main study and received the investigational drug (at least once)
3) Participant whose target tumors are evaluated as PD at the end of the main study, or participant whose main study is discontinued due to the target tumor progression (PD) during the main stud
4) Participant who wish to receive the investigational drug
5) Eastern Cooperative Oncology Group (ECOG) performance status (PS): 0-2 (3, depending on the situation)
6) Laboratory test values prior to vaccination (supportive therapy is not permitted)
- Hemoglobin level >=10 g/dl
- Platelet count >=75,000/microL
- Neutrophil count >=1500/microL
- Lymphocyte count >=1500/microL
- AST and ALT <= 3.0x the institutional normal upper limits
- Creatinine <= 1.5mg/dL

Exclude criteria

1) Malignant tumor requiring treatment (exc. carcinoma in situ and intramucosal carcinoma)
2) Participant who is unable to eat orally due to gastrointestinal lesions and require intravenous infusion, tube feeding, or high-calorie infusion for 24 hours or more.
3) Myelodysplastic syndrome and myeloproliferative disease
4) Moderate pleural effusion, ascites, and pericardial effusion (needing puncture)
5) Prior allogeneic hematopoietic stem cell transplantation after the main study
6) Presence of uncontrollable severe infectious diseases
- HBs antigen, HBs antibody, HBc antibody, or HCV antibody-positive participant
- HIV antibody-positive participant
7) Severe functional disorder (>= CTCAE [ver. 5.0] Grade 3)
8) Severe psychiatric, cognitive, or consciousness disorder
9) Uncontrolled diabetes
10) Enteroparalysis
11) Interstitial pneumonia or idiopathic pulmonary fibrosis (after the main study)
12) Myocardial infarction, severe unstable angina, congestive heart failure, cerebrovascular disease, pulmonary embolism, deep venous thrombosis, other severe thrombo-embolisms and coronary artery bypass grafting (after the main study)
13) Treatment-resistant hypertension
14) Arrhythmia heart failure requiring treatment
15) Unhealed wound (including fracture)
16) Hemorrhagic history such as blood coagulation disorder
17) Participant requiring continuous systemic administration of steroids or immunosuppressants (however, local administration, administration of prednisolone for acute sensorineural hearing loss including sudden deafness [<=60 mg/day, 2 weeks], and administration before imaging examinations for allergies are permitted)
18) Pregnancy or planning to become pregnant after the main study (male: 180 days after the last vaccination, female: 120 days after the last vaccination).
19) Participating in other trials (non-interventional clinical is permitted)
20) Participant who is eligible for surgery or radiation therapy
21) Decision of unsuitability by the principal investigator or the physician in charge

Related Information

• Primary Sponsor: Toda Masahiro
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development (AMED)