CEL001 injection for adults with advanced solid tumors: safety and tolerability

A Phase I Clinical Study Evaluating the Safety and Tolerability of CEL001 Injection in the Treatment of Advanced Solid Tumors

This trial will try injecting CEL001, an NK cell therapy, in adults with advanced solid tumors who have exhausted standard treatments to see if it is safe, tolerable, and shows early signs of benefit.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 13 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Guangzhou Xiling Biotechnology Co., Ltd. (Industry-sponsored)
• Drugs / interventions: vediximab, edatinib, chemotherapy, immunotherapy
• Locations: 1 site (Beijing)
• Trial ID: NCT07259889 on ClinicalTrials.gov

What this trial studies

This first-in-human, open-label Phase 1 trial uses a dose-escalation and expansion design to determine the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary efficacy of CEL001, an NK cell preparation, in patients with advanced solid tumors. The protocol includes screening, a single-dose period with a 14-day DLT observation window, a clearing period, a multiple-dose period with a 28-day DLT window, and up to one year of cumulative dosing followed by two years of follow-up. Investigators will monitor adverse events, collect blood and tumor biomarkers, measure drug levels, and evaluate tumor responses by RECIST 1.1. Dose adjustments and cohort escalation follow predefined safety rules in this open-label format.

Who should consider this trial

Good fit: Adults (≥18) with histologically or cytologically confirmed advanced or metastatic solid tumors, measurable disease by RECIST 1.1, ECOG 0–1, expected survival >3 months, adequate organ function, and who have failed or cannot tolerate standard treatments are the intended candidates.

Not a fit: Patients who still have effective standard treatment options, who do not meet organ function or performance status criteria, or who lack measurable disease are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, CEL001 could provide a new immunotherapy option that is safe and may help control or shrink tumors for patients with few or no standard treatments left.

How similar studies have performed: Cellular NK therapies have produced clear benefits in some hematologic cancers but have shown limited and inconsistent success in solid tumors, so this first-in-human CEL001 approach remains exploratory.

Eligibility criteria

Inclusion Criteria:

* Subjects must meet all of the following criteria to enter this study:

  1. Age ≥ 18 years old, gender not limited;
  2. ECOG physical fitness status score: 0-1 points;
  3. Subjects with advanced or metastatic tumors diagnosed by histology or cytology, who have failed\* or unable to tolerate standard treatment according to CSCO guidelines or NCCN guidelines, or lack effective treatment methods;
  4. Male subjects should weigh no less than 50 kilograms, and female subjects should weigh no less than 45 kilograms;
  5. Expected survival time exceeds 3 months;
  6. There must be at least one measurable lesion, defined as measurable according to the RECIST 1.1 standard;
  7. Prior to treatment, the main organ function meets the following criteria (no blood transfusion, long-acting EPO, or long-acting G-CSF treatment received within 14 days prior to the administration of the investigational drug, which can be reduced to 7 days for short acting EPO or G-CSF):

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  1. Blood routine: Absolute neutrophil count (ANC) ≥ 1.5 × 10\^9/L, platelets ≥ 90 × 10\^9/L, hemoglobin ≥ 90 g/L or ≥ 5.6 mmol/L;
  2. Kidney: serum creatinine ≤ 1.5 x upper limit of normal range (ULN) or Ccr ≥ 50 mL/min (estimated according to the Cockcroft Gault formula);
  3. Liver: Total bilirubin ≤ 1.5 × ULN (including liver metastasis or liver cancer subjects), AST and ALT ≤ 2.5 × ULN (including liver metastasis or liver cancer subjects ≤ 5 × ULN);
  4. Coagulation: International Normalized Ratio (INR) or Prothrombin Time (PT) ≤ 1.5 × ULN, Partially Activated Thromboplastin Time (APTT) ≤ 1.5 × ULN; 8. Women should agree to take appropriate contraceptive measures (such as intrauterine devices \[IUDs\], birth control pills, or condoms) during the study period and within 6 months after the end of the study. They must have a negative serum pregnancy test within 7 days prior to enrollment in the study and must be non lactating subjects; Men should agree to take appropriate contraceptive measures during the study period and within 6 months after the end of the study.

     * Standard treatment failure:

       * Non small cell lung cancer: (1) Subjects with metastatic non driver gene mutations: disease progression or recurrence after at least second-line treatment (including platinum based chemotherapy); (2) Subjects with driver gene mutations such as EGFR, ROS1, ALK in tumors should have received targeted therapy for these mutations that failed, followed by at least second-line treatment (including platinum based chemotherapy) for disease progression or recurrence;
       * Small cell lung cancer: disease progression or recurrence after receiving at least second-line treatment in the past;
       * Colorectal cancer: disease progression or recurrence after at least second-line treatment in the past (standard chemotherapy regimens include fluorouracil or its derivatives, oxaliplatin, and irinotecan, Subjects with BRAF V600E mutation have used BRAF inhibitors, and subjects with MSI-H/dMMR need to have used PD-1/PD-L1 treatment);
       * Head and neck squamous cell carcinoma: disease progression or recurrence after receiving at least second-line treatment (including platinum based chemotherapy) in the past;
       * Urethral epithelial cancer: disease progression or recurrence after receiving at least second-line treatment in the past (recommended treatment regimens in guidelines include PD-1/PD-L1 therapy, platinum based chemotherapy regimen, paclitaxel based chemotherapy regimen, vediximab, and vinblastine, and subjects with FGFR2/3 mutations have already used edatinib);
       * Esophageal cancer: disease progression or recurrence after receiving at least second-line treatment (including platinum based chemotherapy) in the past;
       * Cervical cancer: disease progression or recurrence after receiving at least second-line treatment in the past (including platinum based chemotherapy, subjects who meet PD-L1 positive or TMB-H or MSI-H/dMMR criteria must have received PD-1/PD-L1 treatment);
       * Hepatocellular carcinoma: disease progression or recurrence after receiving at least second-line treatment in the past;
       * Renal cell carcinoma: disease progression or recurrence after receiving at least second-line treatment in the past;
       * For other unspecified malignant tumors, refer to the latest guidelines of CSCO or NCCN.

Exclusion Criteria:

* Subjects who meet any of the following criteria will not be eligible to enter this study:

  1. Individuals allergic to any component of CEL001 injection, including those allergic to penicillin;
  2. Have received anti-tumor treatments such as chemotherapy, radiotherapy, biotherapy, endocrine therapy, targeted therapy, immunotherapy, or participated in other clinical trials within 4 weeks or 5 known drug half lives (whichever is shorter) before the first use of the investigational drug;
  3. Have received traditional Chinese medicine or modern Chinese medicine preparations with anti-tumor indications in the instructions within 14 days before the first administration;
  4. Within the past 5 years, have had malignant tumors other than those treated in this study (excluding cured thyroid cancer, basal cell carcinoma of the skin, and cervical carcinoma in situ);
  5. The adverse reactions of previous anti-tumor treatments have not yet recovered to NCI CTCAE v5.0 grade evaluation ≤ 1 (excluding toxicity judged by researchers to have no safety risks such as hair loss);
  6. Have undergone surgical procedures within 4 weeks prior to receiving treatment or have not fully recovered from any previous invasive procedures;
  7. If there are clinical symptoms of central nervous system metastasis or meningeal metastasis, or if there is other evidence indicating that the participant's central nervous system metastasis or meningeal metastasis has not been controlled, the researcher determines that it is not suitable for inclusion;
  8. Individuals with active infection (NCI CTCAE v5.0 ≥ 2) or any other suspected infection risk assessed by researchers;
  9. Have a history of autoimmune diseases, immunodeficiency, including HIV testing positive, or other acquired or congenital immunodeficiency diseases, or a history of organ transplantation;
  10. Subjects with active hepatitis B or active hepatitis C;
  11. Individuals who have previously received immunotherapy;
  12. Have a history of serious cardiovascular disease, such as severe cardiac rhythm or conduction abnormalities (requiring clinical intervention for ventricular arrhythmias, grade II-III atrioventricular block, etc.), myocardial infarction, history of coronary artery bypass surgery, heart failure, New York Heart Association (NYHA) classification of grade II or above, left ventricular ejection fraction (LVEF) ≤ 50% and thrombotic findings, male QTcF\>450msec or female QTcF\>470msec, etc; Subjects with a history of severe cerebrovascular disease such as stroke;
  13. It is necessary to combine other anti-tumor treatments (including various radiotherapy, chemotherapy, immunotherapy, targeted therapy, traditional Chinese medicine treatment, etc.);
  14. Have a clear history of neurological or mental disorders, including epilepsy or dementia;
  15. The researchers believe that there are other reasons why the subjects are not suitable to participate in this clinical study.

Where this trial is running

Beijing

• Cancer Hospital Chinese Academy of Medical Sciences — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Li Ning, Ph.D. — Cancer Institute and Hospital, Chinese Academy of Medical Sciences
• Study coordinator: Li Ning, Ph.D.

Conditions Advanced Solid Tumors