Phase 1 Study of NIB101 in Participants with Advanced Solid Tumors

JRCT ID: jRCT2033210499

Registered date:18/12/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Solid tumor
• Date of first enrollment: 27/12/2021
• Target sample size: 42
• Study type: Interventional

Intervention(s)

-Dose level 1: 1 x 10^7 cells/body as CAR positive viable cells will be administered intravenously on Day 0.
-Dose level 2: 1 x 10^8 cells/body as CAR positive viable cells will be administered intravenously on Day 0.
-Dose expansion: Recommended dose determined on dose escalation phase will be administered intravenously on Day 0.

Outcome(s)

Primary Outcome

-Dose escalation phase
DLTs, SAEs, AEs leading to discontinuation, AEs of special interest, deaths, AEs, and laboratory abnormalities

-Dose expansion phase
SAEs, AEs leading to discontinuation, AEs of special interest, deaths, AEs, and laboratory abnormalities

Secondary Outcome

-Dose escalation phase
Cellular kinetics/Immunogenicity parameters
Tumor shrinkage, BOR, ORR, DCR, DOR, TTR
The number of RCR positive

-Dose expansion phase
Cellular kinetics/Immunogenicity parameters
Tumor shrinkage, BOR, ORR, DCR, PFS, OS, DOR, TTR
The number of RCR positive

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Participant with histologically or cytologically confirmed solid tumor.
2) Participants who failed or are intolerable to available standard of cares (regardless of the number of prior lines of therapy) at the investigator's discretion.
3) Participant whose tumor tissues express GM2 membrane as determined by immunohistochemistry.
4) Participant who has measurable lesions.
5) Eastern Cooperative Oncology Group (ECOG) performance status 0-1.
6) Life expectancy >=12 weeksfrom the signing screening ICF.
7) Participant with adequate organ functions.
8) Participant who can undergo apheresis at the investigator's discretion.
9) Participant must agree to use adequate contraception methods
10) Patipant who is willing to sign a written informed consent.

Exclude criteria

1) Active brain metastasis on the screening MRI (in case of MRI contradiction, CT is acceptable).
2) Participant with an active, known or suspected autoimmune disease requiring immune suppressive agents other than hormonal replacement therapy.
3) Prior malignancy (other than targeted GM2 positive malignancy) within the previous 3 years the signing screening ICF.
4) Suspected malignant lymphoma or leukemia
5) Participant with known or suspected interstitial pneumonia
6) Active infections requiring treatments
7) Participant with an active, known or suspected gangliosidosis.
8) Other concurrent serious diseases that may interfere with planned study intervention per investigator's discretion.
9) Prior treatment with engineered T-cell therapy/gene therapy.
10) Prior treatment with any GM2, IL-7 or CCL19 targeted therapy.
11) Participant with a condition requiring systemic treatment with either corticosteroids (>= 10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days prior to apheresis. Inhaled or topical steroids, and adrenal replacement steroid doses <10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.
12) Participant with adverse events due to prior therapy have not recovered to grade 1 or baseline, except for non-clinically significant adverse events at the investigator's discretion such as alopecia.
13) Anti-neoplasm treatment within 14 days prior to apheresis
14) Radiation therapy within 14 days prior to apheresis
15) Participant currently requiring ganciclovir, valganciclovir, and so on (the drug that provides HSV-TK substrate) treatment. Participants currently receiving prophylaxis treatment can be enrolled if the prophylaxis treatment is completed before apheresis.
16) Major surgery within 4 weeks prior to screening informed consent.
17) Prior treatment with any investigational study drug/investigational study cell and gene therapies within 28 days before signing screening ICF.
18) Positive human immunodeficiency virus (HIV) and/or HTLV-1 antibody test on the screening prior to apheresis.
19) Positive HBsAg or HCV antibody test on the screening prior to apheresis. Participant who has positive HBs antibody or HBc antibody can be enrolled if HBV-DNA is undetectable.
20) Any symptoms of suspected syphilis
21) Pregnant or breastfeeding
22) History of allergy or hypersensitivity to components of NIB101 or materials used for manufacturing NIB101.
23) Hypersensitivity or contraindicated to study intervention components.

Related Information

• Primary Sponsor: Tanaka Michihiko