Sponsor: Karam Khaddour, MD, MS (other)
Phase: 2
Start date: Feb. 10, 2026
Planned enrollment: 14
Melanoma (post–checkpoint inhibitor setting) - Pivotal single‑arm, multicenter study (C‑144‑01; cohorts within the recommended dose range): ORR 31.5% (95% CI 21.1–43.4); median time to response 1.5 months; median duration of response (DoR) not reached at data cutoff. (fda.gov) - Pooled analysis of consecutive cohorts (C‑144‑01 cohorts 2 and 4; n=153) by independent review committee: ORR 31.4% with complete responses in ~6%; median DoR not reached at analysis; median overall survival ~13.9 months in the heavily pretreated population. Longer‑term updates report durable responses with a substantial proportion ongoing beyond 18 months. (pmc.ncbi.nlm.nih.gov)
Non–small cell lung cancer (NSCLC; investigational) - Phase 2 multicenter study in metastatic NSCLC resistant to immune checkpoint inhibitors (n=28): ORR 21.4% (6/28) with responses observed even in PD‑L1–negative, low TMB, and STK11‑mutant tumors; two treatment‑emergent deaths were reported. (aacrjournals.org)
Cervical cancer (investigational) - Phase 2 C‑145‑04 (ASCO 2019): in 27 evaluable patients with recurrent/metastatic or persistent cervical cancer after prior therapy, ORR 44% (3 CR, 9 PR); median DoR not reached at 7.4‑month median follow‑up. Study uses the same lymphodepletion/IL‑2‑supported LN‑145 approach. (ascopubs.org)
Ongoing/confirmatory studies - A randomized phase 3 trial (TILVANCE‑301) is evaluating lifileucel plus pembrolizumab versus pembrolizumab alone in previously untreated advanced melanoma. (ascopubs.org)
Note: As of October 7, 2025, lifileucel is FDA‑approved for melanoma as above; use in other tumor types remains investigational pending results of ongoing trials. (fda.gov)
Last updated: Oct 2025
Goal: To assess feasibility of manufacturing and delivering autologous tumor-infiltrating lymphocyte (TIL) therapy (lifileucel, LN-145) with lymphodepleting chemotherapy and IL-2, and to characterize safety and preliminary antitumor activity in cutaneous squamous cell carcinoma (CSCC) and Merkel cell carcinoma (MCC) that have progressed after prior immune checkpoint inhibitor therapy.
Patients: Adults (≥18 years) with histologically confirmed CSCC or MCC (mixed histology allowed) with unresectable, recurrent, or metastatic disease and documented progression after prior anti–PD-1/anti–PD-L1 therapy (or recurrence within 6 months after adjuvant/neoadjuvant ICI). ECOG 0–2, adequate marrow/organ function, LVEF ≥45% (NYHA I–II), and adequate pulmonary function are required. Patients must have at least one resectable lesion suitable for TIL harvest (with at least one remaining measurable/evaluable lesion after harvest). Key exclusions include active uncontrolled infection, need for >10 mg/day prednisone equivalent, symptomatic untreated brain metastases, primary immunodeficiency, prior allogeneic organ or stem cell transplant, and recent live vaccination.
Design: Open-label, single-center, multi-cohort, non-randomized phase 2 feasibility study with two cohorts (CSCC and MCC). Patients undergo tumor harvest for centralized TIL manufacturing followed by inpatient-style lymphodepletion, TIL infusion, and IL-2, with imaging response assessments at approximately weeks 6 and 12 and long-term follow-up every 3 months for up to 3 years.
Treatments: Cohort A (CSCC) and Cohort B (MCC) receive nonmyeloablative lymphodepleting chemotherapy with cyclophosphamide (days −5 and −4) and fludarabine (days −5 to −1), followed by a single infusion of autologous TIL product lifileucel (day 0) and subsequent high-dose IL-2 (aldesleukin) for up to 6 doses administered shortly after infusion (days 0–4 and day 14 per protocol schedule). Lifileucel (LN-145) is an autologous, ex vivo expanded TIL cell therapy generated from resected tumor; the intent is to reinfuse tumor-reactive T cells after lymphodepletion to enhance engraftment and antitumor activity, with IL-2 supporting post-infusion T-cell expansion. In advanced melanoma previously treated with checkpoint inhibitors, lifileucel has demonstrated an objective response rate of about 31% with durable responses in a substantial proportion; expected toxicities in TIL programs largely reflect lymphodepleting chemotherapy and IL-2 (e.g., severe cytopenias, febrile neutropenia, capillary leak/hypotension, and organ-function derangements), requiring close monitoring and supportive care.
Outcomes: Primary endpoints are feasibility measures: successful TIL production (manufactured product meeting a minimum cell yield threshold) and successful TIL administration (completion of lymphodepletion, full TIL infusion, and receipt of at least one IL-2 dose), along with incidence of grade ≥3 treatment-emergent adverse events (CTCAE v5.0). Secondary efficacy endpoints include objective response rate by RECIST v1.1, progression-free survival, duration of response, and overall survival, with follow-up for up to 3 years.
Burden on patient: High. Participation requires a surgical tumor harvest, extensive pre-treatment screening (including cardiac and pulmonary testing and potentially bone marrow biopsy/aspirate), then receipt of multi-day lymphodepleting chemotherapy followed by TIL infusion and IL-2 that typically necessitate inpatient admission or prolonged monitored care due to predictable severe cytopenias and IL-2–related cardiopulmonary and hemodynamic toxicities. Imaging is mandated early (around weeks 6 and 12) and then ongoing long-term follow-up visits approximately every 3 months for up to 3 years, adding travel and time commitments beyond routine palliative care in this setting.
Last updated: Feb 2026
Inclusion Criteria:
* Provide written informed consent, which includes understanding that there may be a need for intensive supportive care measures during the study and assessing willingness to undergo such measures, and written authorization for use and disclosure of protected health information.
* Patients must be ≥ than 18 years of age at the time of signing the informed consent form.
* Patients must have histologically or pathologically confirmed diagnosis of CSCC or MCC. Note: Mixed histology is allowed. Note: Neuroendocrine cancer that is clinically considered to be related to a cutaneous primary (MCC) or induced by sun damage (per investigator assessment) is allowed.
* Patients must have unresectable, recurrent, or metastatic disease.
* Patients must have a documented radiographic or clinical disease progression after treatment with ICI (including anti-PD-1 and anti-PD-L1) if it is used in the palliative setting. In patients who received ICI in the neoadjuvant or adjuvant setting, recurrence should have occurred within 6 months from the last treatment with ICI.
* Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 in the investigator's opinion (Appendix B).
* Patients must have at least 1 resectable lesion (or aggregate lesions) with an expected minimum of 1.5 cm diameter in the short axis for TIL production. Note: If a lesion that is considered for TIL harvest is within a previously irradiated field, the lesion must have demonstrated radiographic or clinical progression prior to harvest, and the irradiation must have been completed at least 6 months prior to enrollment.
* Patients must be expected to have at least 1 remaining measurable lesion as defined by RECIST v1.1 or evaluable (radiographically or on clinical examination) following tumor harvest for TIL manufacturing and production that is documented at screening with the following considerations:
* Lesions in a previously irradiated areas should not be selected as target lesions unless progression has been demonstrated in those lesions and the irradiation has been completed at least 6 months prior to enrollment.
* Patients who have only one site of disease may be enrolled if they have a lesion th can be partially resected for TIL harvest, and the remaining portion of the lesion is measurable or evaluable.
* Patients must have the following hematologic parameters:
* Absolute neutrophil count (ANC) ≥ 1000/mm3
* Hemoglobin ≥ 8.0 g/dL and have not received transfusion of packed red blood cells within 7 days.
* Platelet count ≥ 100,000/mm3
* Patients must have an adequate organ function with the following laboratory test values:
* Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 times the upper limit of normal (ULN); and for patients with liver metastases ≤ to 5 times ULN.
* Total bilirubin ≤ 2 mg/dL; patients with Gilbert's Syndrome ≤ to 3 mg/dL.
* Estimated creatinine clearance (eCrCl) ≥ 40 mL/min using the Cockcroft-Gault formula at Screening.
* Patients must have a left ventricular ejection fraction (LVEF) ≥ 45% and be New York Heart Association (NYHA) Class 1 or 2. A cardiac stress test is required for patients who have significant ischemic heart disease, or clinically significant unstable arrythmias; the cardiac stress test must demonstrate no irreversible wall movement abnormality. Patients with an abnormal cardiac stress test may be enrolled if they have adequate ejection fraction and cardiology clearance.
* Patients must have adequate pulmonary function within 2 months from enrollment.
Patients require pulmonary function testing (PFT) if they have any of the following:
* History of cigarette smoking of ≥ 20 pack-years
* Ceased smoking within the past 2 years or still smoking.
* History of chronic obstructive pulmonary disease (COPD)
* Any signs or symptoms of significant respiratory dysfunction.
Post-bronchodilator required pulmonary test results:
* Forced expiratory volume (FEV1)/ forced vital capacity (FVC) \> 70%. Or
* FEV1 \> 50% of predicted normal value. Note: If a patient is unable to perform reliable spirometry due to abnormal upper airway anatomy (i.e., tracheostomy), a 6-minute walk test may be used to assess pulmonary function. Patients must be able to walk a distance at least 80% of predicted for age and sex with no evidence of hypoxia at any point during the test (i.e., saturation of peripheral oxygen \[SpO2\] must remain ≥ 89%).
* Patients must have completed or discontinued systemic therapy ≥ 21 days prior to tumor harvest. Note: Patients are allowed to have palliative radiation or systemic therapy after tumor harvest and before NMA-LD but there should be at least 7 days between discontinuation of palliative treatment and start of NMA-LD.
* Patients must have recovered from all prior anticancer TRAEs to Grade ≤ 1 (per CTCAE v5.0) with the exceptions of vitiligo, alopecia or neuropathy. Patients with irreversible toxicity that are properly managed (such as with endocrinopathy treatment with hormone replacement therapy) may qualify for the study regardless of grade of TRAEs.
* Patients of childbearing potential or those with partners of childbearing potential must be willing to practice an approved method of highly effective birth control during treatment and for 12 months after receiving all protocol-related therapy (Appendix C). Additionally, males may not donate sperm and females may not donate eggs during the required contraception period.
Approved methods of birth control include:
* Combined (estrogen- and progesterone- containing) hormonal birth control associated with inhibition of ovulation: oral, intravaginal, transdermal.
* Progesterone-only hormonal birth control associated with inhibition of ovulation:
oral, injectable, implantable.
* Intrauterine device (IUD)
* Intrauterine hormone-releasing system (IUS)
* Bilateral tubal occlusion
* Vasectomy
* True absolute sexual abstinence when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar ovulation, symptothermal, post-ovulation methods) is not acceptable.
Exclusion Criteria:
* Have a history of allogenic organ transplant.
* Have symptomatic untreated brain metastases. Patients with brain metastases may be enrolled with the following considerations:
* Patients with asymptomatic brain metastases that are treated and have been stable for at least 7 days may be enrolled.
* Patients with historically or recently treated brain metastases will be considered for enrollment if the patient is clinically stable for ≥ 2 weeks, and the patient does not require ongoing corticosteroid treatment (\>10 mg/day prednisone or its equivalent).
* Patients who undergo tumor harvest prior to disease progression and develop symptomatic brain metastases after tumor harvest should have receive appropriate treatment for ≥ 2 weeks and not require corticosteroids (\>10 mg/day or its equivalent) at the start of NMA-LD (Day -5).
* Require systemic steroid therapy \>10 mg/day prednisone or its equivalent. Patient receiving steroids as replacement therapy for adrenocortical insufficiency are not excluded.
* Have evidence of any active viral, bacterial, or fungal infection requiring ongoing systemic treatment.
* Are pregnant or breastfeeding. Female patients of childbearing potential must have a negative beta human chorionic gonadotropin (B-HCG) test at Screening (Appendix C).
* Have active medical illness that in the opinion of the investigator would pose increased risk for study participation, such as systemic infections, coagulation disorders, or other active major medical illnesses of the cardiovascular, respiratory, or immune system.
* Have received a live or attenuated vaccination within 28 days prior to the start of NMALD.
* Have any form of primary immunodeficiency (e.g., severe combined immunodeficiency disease \[SCID\] or acquired immune deficiency syndrome \[AIDS\]).
* Have a history of allogenic stem cell transplant, or active hematological malignancy (such as chronic lymphocytic leukemia or lymphoma).
* Have a history of hypersensitivity to any component of the study drugs. TIL should not be administered to patients with a known hypersensitivity to any component of the autologous TIL product formulation including, but not limited to, any of the following:
* NMA-LD (cyclophosphamide, mesna, and fludarabine)
* Proleukin, aldesleukin, IL-2
* Antibiotics of the aminoglycoside group. These patients may be eligible if current hypersensitivity has been excluded.
* Any component of the TIL product formulation, including dimethyl sulfoxide (DMSO), human serum albumin (HSA), IL-2, or dextran-40
* Have had another primary malignancy within the previous 1 year (except for malignancies that do not require treatment or have been curatively treated, and do not pose a significant risk of recurrence including, but not limited to in situ carcinoma of the cervix, early stage skin cancer, including non-melanoma skin cancer; ductal carcinoma in situ (DCIS) or lobular carcinoma in the situ (LCIS) of the breast; intraductal carcinoma of the breast that has been treated with curative intent including patients who are on adjuvant hormonal treatment, prostate cancer with Gleason score ≤ to 6; or superficial bladder cancer).
Boston, Massachusetts, 02215, United States
[email protected] / 617-632-6571
Status: Recruiting