Clinical trial NCT05192174: Study of NIB101 in Participants With Advanced Solid Tumors
Official title: An Open-Label Non-Randomized Phase 1 Dose Escalation and Dose Expansion Study of NIB101 in Participants With Advanced Solid Tumors
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Solid Tumor
Lead sponsor: Noile-Immune Biotech, Inc
Sponsor class: INDUSTRY
First posted: 2022-01-14
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: NIB101-01 study is an open-label, non-randomized Phase 1 study in participants with GM2 positive advanced solid tumor, who failed to available standard of cares to evaluate the safety and tolerability of NIB101.

Detailed description: The screening begins by signing the informed consent form and determining the participant's initial eligibility. Alternatively, in advance, the prescreening enables participants to confirm GM2 expression using the archived samples by signing the prescreening ICF prior to the screening. After the consent is obtained and the eligibility of participants is confirmed, the participant will undergo apheresis. Lymphodepleting chemotherapy will be administered prior to NIB101 infusion. After manufacturing of NIB101, participants will receive a single dose of NIB101 intravenously on Day 0 and be followed for safety and efficacy.

Eligibility criteria: Inclusion Criteria:

1. Participant with histologically or cytologically confirmed solid tumor.
2. Participant 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 weeks from 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. Participant who is willing to sign a written informed consent.

Exclusion 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, Interleukin-7 (IL-7) or Chemokine (C-C motif) ligand 19 (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 Human T-cell leukemia virus-1 (HTLV-1) antibody test on the screening prior to apheresis.
19. Positive Hepatitis B surface (HBs) antigen or Hepatitis C virus (HCV) antibody test on the screening prior to apheresis. Participant who has positive HBs antibody or Hepatitis B core (HBc) antibody can be enrolled if Hepatitis B virus (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.

Primary outcomes:
measure: Dose Limiting Toxicities
timeFrame: 28 days after NIB101 infusion
measure: Adverse Events
timeFrame: 2 years from NIB101 infusion
Secondary outcomes:
measure: Objective Response
timeFrame: 2 years from NIB101 infusion
measure: Overall Response Rate
timeFrame: 2 years from NIB101 infusion
measure: Disease Control Rate
timeFrame: 2 years from NIB101 infusion
measure: Duration of Response
timeFrame: 2 years from NIB101 infusion
measure: Time To Response
timeFrame: 2 years from NIB101 infusion
measure: Progression Free Survival
timeFrame: 2 years from NIB101 infusion
measure: Overall Survival
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (Cmax)
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (Tmax)
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (T1/2)
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (Clast)
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (Tlast)
timeFrame: 2 years from NIB101 infusion
measure: Pharmacokinetics (AUC)
timeFrame: 2 years from NIB101 infusion
measure: Immunogenicity
timeFrame: 2 years from NIB101 infusion
measure: Replication Competent Retrovirus (RCR)
timeFrame: 2 years from NIB101 infusion
Other outcomes:
Locations:
city: Kashiwa
country: Japan
facility: National Cancer Center Hospital East
state: Chiba
city: Chuo Ku
country: Japan
facility: National Cancer Center Hospital
state: Tokyo
Study officials:
affiliation: Noile-Immune Biotech, Inc
name: Noile-Immune Biotech, Inc.
role: STUDY_DIRECTOR
References: