Safety and Efficacy of CD160-Enhanced Autologous Antigen-Specific T-Cells (BTC-Ag-T) in Advanced Biliary Tract Cancer

NCT07614061 | Recruiting Phase 1

• 1 other identifier: ZSAB-AgT
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

BTC-Ag-T (ACH-AgT001) is an autologous experimental T-cell therapy designed for advanced biliary tract cancer. This is an open-label, single-arm Phase 1 study to evaluate the safety, tolerability, and preliminary efficacy of BTC-Ag-T in patients with advanced, unresectable, or metastatic biliary tract cancer who have failed standard-of-care therapy.

Conditions

Biliary Tract Neoplasms; Cholangiocarcinoma, Intrahepatic; Cholangiocarcinoma, Extrahepatic; Cholangiocarcinoma, Perihilar; Gallbladder Cancer

Keywords

Adoptive T-cell therapy; Antigen-specific T cells; CD160; Autologous T cell therapy; Biliary tract cancer; Cholangiocarcinoma; APTC - antigen-presenting tumor cell; Lymphodepletion; Phase 1; Investigator-initiated trial

Outcome Measures

Primary Outcomes (2)

• DLT incidence and MTD (Module A)

  Proportion of subjects with protocol-defined dose-limiting toxicities (Grade ≥3 cytokine release syndrome (CRS) or Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), persistent Grade 4 cytopenia, or specified Grade ≥3 non-hematologic toxicity attributed to BTC-Ag-T); MTD identified per 3+3 rules.

  DLT window: Day 0 through Day 28

• Safety of repeat lympho-depletion(LD)/re-induction (Module B)

  Incidence and severity of treatment-emergent adverse events graded per Common Terminology Criteria for Adverse Events (CTCAE) v6.0 and American Society for Transplantation and Cellular Therapy (ASTCT) criteria for CRS / ICANS, summarized by lymphodepletion cycle.

  Through Day 150; long-term follow-up up to 15 years

Secondary Outcomes (6)

• Objective Response Rate (ORR)

  24 months

• Duration of Response (DoR)

  24 months

• Disease Control Rate (DCR)

  24 months

• Progression-Free Survival (PFS)

  24 months

• Overall Survival (OS)

  36 months

Study Arms (1)

CD160-Enhanced Autologous BTC-Ag-T

EXPERIMENTAL

Autologous CD160-enhanced BTC-specific antigen-specific T cells (ACH-AgT001) administered IV after fludarabine/cyclophosphamide lymphodepletion. Module A uses a 3+3 dose escalation. Module B evaluates repeat lymphodepletion / re-induction at the selected dose.

Biological: BTC-Ag-T (ACH-AgT001); Drug: Cyclophosphamide and Fludarabine

Interventions

BTC-Ag-T (ACH-AgT001) BIOLOGICAL

CD160-enhanced autologous antigen-specific T cells. IV infusion.

Also known as: CD160-Ag-T; ACH-AgT001

CD160-Enhanced Autologous BTC-Ag-T

Cyclophosphamide and Fludarabine DRUG

Combination of cyclophosphamide and Fludarabine as part of lymphodepletion

CD160-Enhanced Autologous BTC-Ag-T

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must meet all of the following criteria to be enrolled:
• \. Age
• \- Age ≥ 18 years at the time of signing informed consent. 2. Diagnosis
• Histologically or cytologically confirmed biliary tract malignancy (intrahepatic, perihilar, or distal extrahepatic cholangiocarcinoma, or gallbladder cancer).
• \. Disease status
• Locally advanced unresectable or metastatic disease 4. Prior systemic therapy
• Patients (including those with refractory BTC and those with postoperative recurrence) must have received prior gemcitabine-based chemotherapy in combination with a PD-1/PD-L1 inhibitor.
• \. Measurable disease
• At least one measurable lesion per RECIST v1.1 at baseline imaging.
• Sufficient viable tumor tissue from biopsy for antigen-presenting tumor cell (APTC) manufacturing 6. Adequate venous access and overall condition to tolerate leukapheresis. 7. Washout and lymphocyte recovery before leukapheresis 8. ECOG performance status 0 or 1 9. Organ function
• Hematology (no growth-factor support or transfusion within 5 days of testing, unless otherwise stated): ANC ≥ 1.0 × 10⁹/L; platelets ≥ 75 × 10⁹/L; hemoglobin ≥ 8.0 g/dL (transfusion to reach this threshold is permitted).
• Hepatic: total bilirubin ≤ 2.0 × ULN (≤ 3.0 × ULN allowed for documented Gilbert syndrome); AST and ALT ≤ 5.0 × ULN.
• Renal: serum creatinine ≤ 1.5 × ULN, or estimated creatinine clearance (e.g., Cockcroft-Gault) ≥ 40 mL/min.
• Adequate cardiopulmonary reserve to tolerate lymphodepleting conditioning and cell infusion in the investigator's judgment.
• \. Viral serology

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded:
• Mixed/combined hepatocellular-cholangiocarcinoma, ampullary carcinoma, and other histologies not consistent with BTC
• Prior allogeneic transplant or recent gene-modified cell therapy
• Active CNS metastases
• Patients with uncontrolled or high-risk active infection are excluded, including hepatitis B virus (HBV), hepatitis C virus (HCV), Epstein-Barr virus (EBV), and active tuberculosis (TB).
• Active autoimmune disease requiring systemic immunosuppression
• Significant cardiovascular disease
• Significant pulmonary disease
• Severe hepatic decompensation
• Active variceal bleeding, or recent life-threatening portal-hypertension complications that cannot be stably controlled.
• Another primary malignancy within the past 3 years, except: tumors treated with curative intent and at low risk of recurrence (e.g., adequately treated basal- or squamous-cell skin cancer, in-situ cervical cancer, or low-Gleason localized prostate cancer, occult thyroid carcinoma).
• Severe hypersensitivity.
• Pregnant or lactating women
• Concurrent participation in another interventional study
• Any other condition that, in the investigator's judgment, renders the patient unsuitable for enrollment.

Sponsors & Collaborators

• Shanghai Zhongshan Hospital: lead

Study Sites (1)

Shanghai Zhongshan Hospital, Fudan University

Shanghai, 200032, China

RECRUITING

MeSH Terms

Conditions

Biliary Tract Neoplasms; Cholangiocarcinoma; Klatskin Tumor; Gallbladder Neoplasms

Interventions

Cyclophosphamide; fludarabine

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Biliary Tract Diseases; Digestive System Diseases; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Gallbladder Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Guoming Shi, MD, PhD

  Department of Liver Surgery and Transplantation, Shanghai Zhongshan Hospital, Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Guoming Shi, MD, PhD

CONTACT

+86 021-64041990; shi.guoming@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Open-label, single-arm, sequential dose-escalation design with two operationally distinct modules. Module A uses a 3+3 design across three dose levels to identify dose-limiting toxicity (DLT), maximum tolerated dose (MTD), and the recommended phase 2 dose (RP2D). Module B evaluates the safety and feasibility of repeat lymphodepletion / re-induction at the selected dose. All participants receive the same class of investigational product.

Study Record Dates

• First Submitted: May 22, 2026
• First Posted: May 29, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: May 29, 2026

Record last verified: 2026-05

Locations

CN (1)