CAR-pNK Cell Immunotherapy in MUC1 Positive Relapsed or Refractory Solid Tumor

NCT02839954 — Phase 1 / Phase 2, Unknown, 10 participants, PersonGen BioTherapeutics (Suzhou) Co., Ltd.

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Study Evaluating the Efficacy and Safety of Chimeric Antigen Receptor-Modified pNK Cells in MUC1 Positive Advanced Refractory or Relapsed Solid Tumor

Registry	NCT02839954
Status	Unknown
Phase	Phase 1 / Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	10 (Estimated)
Sponsor	PersonGen BioTherapeutics (Suzhou) Co., Ltd. (Industry); collaborators: The First People's Hospital of Hefei, Hefei Binhu Hospital
Conditions	Hepatocellular Carcinoma, Non-small Cell Lung Cancer, Pancreatic Carcinoma, Triple-Negative Invasive Breast Carcinoma, Malignant Glioma of Brain, Colorectal Carcinoma, Gastric Carcinoma
Interventions	anti-MUC1 CAR-pNK cells (Biological)
Dates	start 2016-07-01, primary completion 2017-07-01 (estimated), completion 2018-07-01 (estimated), first posted 2016-07-21, last updated 2016-12-06
Locations	1 site in China

-Summary

The purpose of this study is to evaluate the safety and effectiveness of CAR-pNK cell immunotherapy in patients with MUC1 positive relapsed or refractory solid tumor.

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+Arms and interventions

Arm	Type	Interventions	Description
CAR-pNK Cell immunotherapy	Experimental	Biological: anti-MUC1 CAR-pNK cells	Enrolled patients will receive CAR-pNK cell immunotherapy with a novel specific chimeric antigen receptor targeting MUC1 antigen by infusion.

anti-MUC1 CAR-pNK cells (Biological), also known as Chimeric antigen receptor NK cells with specificity for MUC1

+Outcome measures

Primary outcomes

Phase I: Adverse events attributed to the administration of the anti-MUC1 CAR-pNK cells [2 years]: Determine the toxicity profile of the MUC1 targeted CAR-pNK cells with Common Toxicity Criteria for Adverse Effects (CTCAE) version 4.0.

Secondary outcomes

Phase II: Objective Response Rate [2 years]: The objective response rate (ORR) is defined as the proportion of patients who achieve radiographic partial or complete response (PR or CR) according to the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 guideline.

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

Male and female subjects with MUC1+ malignancies in patients with no available curative treatment options who have limited prognosis (several months to < 2 year survival) with currently available therapies will be enrolled:

1. Eligible diseases: MUC1+ malignant glioma of brain, colorectal carcinoma, gastric carcinoma, hepatocellular carcinoma, non-small cell lung cancer, pancreatic carcinoma and triple-negative basal-like breast carcinoma.
2. Patients 18 years of age or older, and must have a life expectancy > 12 weeks.
3. MUC1 is expressed in malignancy tissues by immuno-histochemical (IHC).
4. Eastern cooperative oncology group (ECOG) performance status of 0-2 or karnofsky performance status (KPS) score is higher than 60.
5. Presence of measurable disease by RECIST.
6. Females of child-bearing potential must have a negative pregnancy test and all subjects must agree to use an effective method of contraception for up to two weeks after the last infusion of CAR-pNK cells.
7. Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements: White blood cell count (WBC) ≥ 2500c/ml, Platelets ≥ 50×10\^9/L, Hb ≥ 9.0g/dL, lymphocyte (LY) ≥ 0.7×10\^9/L, LY% ≥ 15%, Alb ≥ 2.8g/dL, serum lipase and amylase < 1.5×upper limit of normal, serum creatinine ≤ 2.5mg/dL, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 5×upper limit of normal, serum total bilirubin ≤ 2.0mg/dL. These tests must be conducted within 7 days prior to registration.
8. Ability to give informed consent.

Exclusion Criteria:

1. Patients with symptomatic central nervous system (CNS) involvement.
2. Pregnant or nursing women may not participate.
3. Active HIV, hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
4. Serious illness or medical condition which would not permit the patient to be managed according to the protocol, including active uncontrolled infection, major cardiovascular, coagulation disorders, respiratory or immune system, myocardial infarction, cardiac arrhythmias, obstructive/restrictive pulmonary disease, or psychiatric or emotional disorders.
5. Concurrent use of systemic steroids. Recent or current use of inhaled steroids is not exclusionary.
6. The existence of unstable or active ulcers or gastrointestinal bleeding.
7. Patients with a history of organ transplantation or are waiting for organ transplantation.
8. Patients need anticoagulant therapy (such as warfarin or heparin).
9. Patients need long-term antiplatelet therapy (aspirin at a dose > 300mg/d; clopidogrel at a dose > 75mg/d).

+Locations (1)

Facility	City	Country	Status
PersonGen BioTherapeutics (Suzhou) Co., Ltd.	Suzhou, Jiangsu	China	Recruiting

+Publications

Results publication: Lin J, Xu W, Xia L, Zhang C, Ge L, Gao J, Bao J, Xu Y, Sun X, Li H, Wang Y. A phase I trial of combination CAR-NK92MI immunotherapy by dual targeting MUC-1 and PD-L1 for patients with relapsed or refractory solid tumors: focus on non-small cell lung cancer. BMC Pulm Med. 2025 Oct 21;25(1):482. doi: 10.1186/s12890-025-03944-y. PMID 41121171
Results publication: Del Zotto G, Marcenaro E, Vacca P, Sivori S, Pende D, Della Chiesa M, Moretta F, Ingegnere T, Mingari MC, Moretta A, Moretta L. Markers and function of human NK cells in normal and pathological conditions. Cytometry B Clin Cytom. 2017 Mar;92(2):100-114. doi: 10.1002/cyto.b.21508. Epub 2017 Feb 12. PMID 28054442

+Investigators

Lin Yang, Ph.D., Principal investigator, PersonGen BioTherapeutics (Suzhou) Co., Ltd.