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Study of NIB101 in Participants With Advanced Solid Tumors

Phase 1
Terminated
Conditions
Solid Tumor
Interventions
Registration Number
NCT05192174
Lead Sponsor
Noile-Immune Biotech, Inc
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.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
3
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
NIB101 Expansion CohortNIB101Recommended dose determined on dose escalation phase will be administered intravenously on Day 0.
NIB101 Dose Level 2NIB1011 x 10\^8 cells/body as CAR positive viable cells will be administered intravenously on Day 0.
NIB101 Dose Level 1NIB1011 x 10\^7 cells/body as chimeric antigen receptor (CAR) positive viable cells will be administered intravenously on Day 0.
Primary Outcome Measures
NameTimeMethod
Adverse Events2 years from NIB101 infusion

Number of participants with adverse events

Dose Limiting Toxicities28 days after NIB101 infusion

Specific adverse events defined in the protocol and related to NIB101 infusion

Secondary Outcome Measures
NameTimeMethod
Time To Response2 years from NIB101 infusion

Time from NIB101 infusion to the initial documented response (PR or better)

Progression Free Survival2 years from NIB101 infusion

Time from NIB101 infusion to the date of disease progression or death from any cause.

Overall Survival2 years from NIB101 infusion

Time from NIB101 infusion to time of death due to any cause

Pharmacokinetics (Tlast)2 years from NIB101 infusion

Time of Clast observed

Immunogenicity2 years from NIB101 infusion

Evaluation of an anti-CAR antibody response

Objective Response2 years from NIB101 infusion

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

Disease Control Rate2 years from NIB101 infusion

Percentage of subjects who achieved SD or better

Pharmacokinetics (Cmax)2 years from NIB101 infusion

Maximum peak of NIB101

Overall Response Rate2 years from NIB101 infusion

Percentage of subjects who achieved PR or better

Duration of Response2 years from NIB101 infusion

Time from first documentation of response (PR or better) to first documentation of disease progression or death from any cause

Pharmacokinetics (AUC)2 years from NIB101 infusion

Area under the curve of NIB101

Pharmacokinetics (Tmax)2 years from NIB101 infusion

Time to maximum peak of NIB101

Replication Competent Retrovirus (RCR)2 years from NIB101 infusion

Number of cases of positive RCR

Pharmacokinetics (T1/2)2 years from NIB101 infusion

Half life of NIB101

Pharmacokinetics (Clast)2 years from NIB101 infusion

Concentration of last quantified NIB101

Trial Locations

Locations (2)

National Cancer Center Hospital East

🇯🇵

Kashiwa, Chiba, Japan

National Cancer Center Hospital

🇯🇵

Chuo Ku, Tokyo, Japan

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