Study of NIB101 in Participants With Advanced Solid Tumors

An Open-Label Non-Randomized Phase 1 Dose Escalation and Dose Expansion Study of NIB101 in Participants With Advanced Solid Tumors

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.

Study Overview

• Status: Terminated
• Conditions: Solid Tumor
• Intervention / Treatment: Biological: 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.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Chiba
    • Kashiwa, Chiba, Japan
      • National Cancer Center Hospital East
  • Tokyo
    • Chuo Ku, Tokyo, Japan
      • National Cancer Center Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NIB101 Dose Level 1; 1 x 10^7 cells/body as chimeric antigen receptor (CAR) positive viable cells will be administered intravenously on Day 0.	Biological: NIB101; NIB101
Experimental: NIB101 Dose Level 2; 1 x 10^8 cells/body as CAR positive viable cells will be administered intravenously on Day 0.	Biological: NIB101; NIB101
Experimental: NIB101 Expansion Cohort; Recommended dose determined on dose escalation phase will be administered intravenously on Day 0.	Biological: NIB101; NIB101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose Limiting Toxicities	Specific adverse events defined in the protocol and related to NIB101 infusion	28 days after NIB101 infusion
Adverse Events	Number of participants with adverse events	2 years from NIB101 infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response	Complete Response (CR), Partial Response (PR), Stable Disease (SD) or Progressive Disease (PD) determined by the investigator per RECIST version 1.1 or modified RECIST	2 years from NIB101 infusion
Overall Response Rate	Percentage of subjects who achieved PR or better	2 years from NIB101 infusion
Disease Control Rate	Percentage of subjects who achieved SD or better	2 years from NIB101 infusion
Duration of Response	Time from first documentation of response (PR or better) to first documentation of disease progression or death from any cause	2 years from NIB101 infusion
Time To Response	Time from NIB101 infusion to the initial documented response (PR or better)	2 years from NIB101 infusion
Progression Free Survival	Time from NIB101 infusion to the date of disease progression or death from any cause.	2 years from NIB101 infusion
Overall Survival	Time from NIB101 infusion to time of death due to any cause	2 years from NIB101 infusion
Pharmacokinetics (Cmax)	Maximum peak of NIB101	2 years from NIB101 infusion
Pharmacokinetics (Tmax)	Time to maximum peak of NIB101	2 years from NIB101 infusion
Pharmacokinetics (T1/2)	Half life of NIB101	2 years from NIB101 infusion
Pharmacokinetics (Clast)	Concentration of last quantified NIB101	2 years from NIB101 infusion
Pharmacokinetics (Tlast)	Time of Clast observed	2 years from NIB101 infusion
Pharmacokinetics (AUC)	Area under the curve of NIB101	2 years from NIB101 infusion
Immunogenicity	Evaluation of an anti-CAR antibody response	2 years from NIB101 infusion
Replication Competent Retrovirus (RCR)	Number of cases of positive RCR	2 years from NIB101 infusion

Collaborators and Investigators

• Sponsor: Noile-Immune Biotech, Inc
• Investigators: Study Director: Noile-Immune Biotech, Inc., Noile-Immune Biotech, Inc

Study record dates

Study Major Dates

• Study Start (Actual): January 24, 2022
• Primary Completion (Actual): May 16, 2025
• Study Completion (Actual): May 16, 2025

Study Registration Dates

• First Submitted: December 24, 2021
• First Submitted That Met QC Criteria: January 10, 2022
• First Posted (Actual): January 14, 2022

Study Record Updates

• Last Update Posted (Actual): May 23, 2025
• Last Update Submitted That Met QC Criteria: May 20, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: NIB101-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No