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.
Histologically or cytologically confirmed biliary tract malignancy (intrahepatic, perihilar, or distal extrahepatic cholangiocarcinoma, or gallbladder cancer).
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.
At least one measurable lesion per RECIST v1.1 at baseline imaging.
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).
Single group
BTC-Ag-T (ACH-AgT001)
Biological/VaccineCD160-enhanced autologous antigen-specific T cells. IV infusion.
Cyclophosphamide and Fludarabine
DrugCombination of cyclophosphamide and Fludarabine as part of lymphodepletion
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.
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.
Objective Response Rate (ORR)
The proportion of patients achieving Complete Response (CR) plus Partial Response (PR), as defined per RECIST v1.1 criteria.
Duration of Response (DoR)
Duration of response per RECIST v1.1 in responders.
Disease Control Rate (DCR)
Disease control rate (CR + PR + SD ≥ 12 weeks) per RECIST v1.1.
Progression-Free Survival (PFS)
Time from the date of Ag-T cell infusion to the first objective documentation of disease progression (per RECIST v1.1) or death due to any cause.
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