NCT01174121

Brief Summary

Background: The NCI Surgery Branch has developed an experimental therapy that involves taking white blood cells from patients' tumors, growing them in the laboratory in large numbers, and then giving the cells back to the patient. These cells are called Tumor Infiltrating Lymphocytes, or TIL and we have given this type of treatment to over 200 patients with melanoma. Researchers want to know if TIL shrink s tumors in people with digestive tract, urothelial, breast, or ovarian/endometrial cancers. In this study, we are selecting a specific subset of white blood cells from the tumor that we think are the most effective in fighting tumors and will use only these cells in making the tumor fighting cells. Objective: The purpose of this study is to see if these specifically selected tumor fighting cells can cause digestive tract, urothelial, breast, or ovarian/endometrial tumors to shrink and to see if this treatment is safe. Eligibility: \- Adults age 18-72 with upper or lower gastrointestinal, hepatobiliary, genitourinary, breast, ovarian/endometrial cancer, or glioblastoma refractory to standard chemotherapy. Design: Work up stage: Patients will be seen as an outpatient at the NIH clinical Center and undergo a history and physical examination, scans, x-rays, lab tests, and other tests as needed. Surgery: If the patients meet all of the requirements for the study they will undergo surgery to remove a tumor that can be used to grow the TIL product. Leukapheresis: Patients may undergo leukapheresis to obtain additional white blood cells. (Leukapheresis is a common procedure, which removes only the white blood cells from the patient.) Treatment: Once their cells have grown, the patients will be admitted to the hospital for the conditioning chemotherapy, the TIL cells and aldesleukin. They will stay in the hospital for about 4 weeks for the treatment. Follow up: Patients will return to the clinic for a physical exam, review of side effects, lab tests, and scans about every 1-3 months for the first year, and then every 6 months to 1 year as long as their tumors are shrinking. Follow up visits will take up to 2 days.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
332

participants targeted

Target at P75+ for phase_2

Timeline
43mo left

Started Aug 2010

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress82%
Aug 2010Dec 2029

First Submitted

Initial submission to the registry

July 31, 2010

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 3, 2010

Completed
23 days until next milestone

Study Start

First participant enrolled

August 26, 2010

Completed
18.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 27, 2028

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 27, 2029

Last Updated

June 11, 2026

Status Verified

May 6, 2026

Enrollment Period

18.4 years

First QC Date

July 31, 2010

Last Update Submit

June 10, 2026

Conditions

Keywords

Digestive Tract CancersBreast CancerEndocrine TumorsOvarian/Endometrial CancerGenitourinary Cancer

Outcome Measures

Primary Outcomes (1)

  • Response rate

    Percentage of patients who have a clinical response to treatment (objective tumor regression)

    6 and 12 weeks after cell infusion, then every 3 months x3, then every 6 months x 2 years, then per PI discretion

Secondary Outcomes (2)

  • Safety and efficacy of pembrolizumab in combination with TIL therapy

    Every 6 weeks (week 6, 12, 18, 24) within 24 hours prior to next scheduled pembrolizumab dose

  • Frequency and severity of treatment-related adverse events

    30 days after end of treatment

Study Arms (5)

1/CD8+ Enriched TIL (CLOSED)

EXPERIMENTAL

Non-myeloablative, lymphodepleting preparative regimen of cyclophosphamide and fludarabine + young CD8+ enriched TIL + high-dose aldesleukin (CLOSED)

Drug: FludarabineDrug: CyclophosphamideDrug: AldesleukinBiological: Young TIL

2/Unselected TIL (CLOSED)

EXPERIMENTAL

Non-myeloablative, lymphodepleting preparative regimen of cyclophosphamide and fludarabine + young unselected TIL + high-dose aldesleukin (CLOSED)

Drug: FludarabineDrug: CyclophosphamideDrug: AldesleukinBiological: Young TIL

3/Unselected TIL + Pembro Prior to Cells

EXPERIMENTAL

Non-myeloablative, lymphodepleting preparative regimen of cyclophosphamide and fludarabine + young unselected TIL + high-dose aldesleukin + pembrolizumab prior to cell administration and 3 additional doses every 3 weeks following cell infusion

Drug: Pembrolizumab (Keytruda)Drug: FludarabineDrug: CyclophosphamideDrug: AldesleukinBiological: Young TIL

4/Unselected TIL + Pembro at POD

EXPERIMENTAL

Non-myeloablative, lymphodepleting preparative regimen of cyclophosphamide and fludarabine + young unselected TIL + high-dose aldesleukin + pembrolizumab within 4 weeks of progressive disease for up to 8 doses every 3 weeks

Drug: Pembrolizumab (Keytruda)Drug: FludarabineDrug: CyclophosphamideDrug: AldesleukinBiological: Young TIL

5/Unselected TIL + Pembro Prior to Cells

EXPERIMENTAL

Non-myeloablative, lymphodepleting preparative regimen of cyclophosphamide and fludarabine + young unselected TIL + high-dose aldesleukin + pembrolizumab prior to cell administration and 3 additional doses every 3 weeks following cell infusion

Drug: Pembrolizumab (Keytruda)Drug: FludarabineDrug: CyclophosphamideDrug: AldesleukinBiological: Young TIL

Interventions

Days -7 to -3: Fludarabine 25 mg/m2/day IVPB daily over 30 minutes for 5 days.

1/CD8+ Enriched TIL (CLOSED)2/Unselected TIL (CLOSED)3/Unselected TIL + Pembro Prior to Cells4/Unselected TIL + Pembro at POD5/Unselected TIL + Pembro Prior to Cells

Days -7 and -6: Cyclophosphamide 60 mg/kg/day X 2 days IV in 250 ml D5W with Mesna 15 mg/kg/day X2 days over 1 hr.

1/CD8+ Enriched TIL (CLOSED)2/Unselected TIL (CLOSED)3/Unselected TIL + Pembro Prior to Cells4/Unselected TIL + Pembro at POD5/Unselected TIL + Pembro Prior to Cells

Aldesleukin 720,000 IU/kg IV (based on total body weight) over 15 minutes every eight hours (+/- 1 hour) beginning within 24 hours of cell infusion and continuing for up to 4 days (maximum 12 doses.)

1/CD8+ Enriched TIL (CLOSED)2/Unselected TIL (CLOSED)3/Unselected TIL + Pembro Prior to Cells4/Unselected TIL + Pembro at POD5/Unselected TIL + Pembro Prior to Cells
Young TILBIOLOGICAL

Day 0: Cells will be infused intravenously (IV) on the Patient Care Unit over 20-30 minutes (one to four days after the last dose of fludarabine).

1/CD8+ Enriched TIL (CLOSED)2/Unselected TIL (CLOSED)3/Unselected TIL + Pembro Prior to Cells4/Unselected TIL + Pembro at POD5/Unselected TIL + Pembro Prior to Cells

Arm 3 or 5: Pembrolizumab 2mg/kg IV over approximately 30 minutes on Days -2, 21, 42, and 63 Arm 4: Pembrolizumab 2mg/kg IV over approximately 30 minutes (for patients who meet progressive disease per RECIST criteria and have resolved major toxicities after cell infusion or anytime during the post-treatment evaluation period; starting within 4 weeks of progression; may receive up to 8 doses every 3 weeks).

3/Unselected TIL + Pembro Prior to Cells4/Unselected TIL + Pembro at POD5/Unselected TIL + Pembro Prior to Cells

Eligibility Criteria

Age18 Years - 72 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Measurable (per RECIST v1.0 criteria), metastatic cancer of one of the following types: upper or lower gastrointestinal, hepatobiliary, genitourinary, breast, ovarian/endometrial, or endocrine tumors including neuroendocrine tumors. Patients must have at least one lesion that is resectable for TIL generation with minimal morbidity, preferentially using minimal invasive laparoscopic or thoracoscopic surgery for removal of superficial tumor deposit.
  • Confirmation of diagnosis of metastatic cancer by the NCI Laboratory of Pathology.
  • Refractory to approved standard systemic therapy. Specifically:
  • Patients with metastatic colorectal cancer must have received oxaliplatin or irinotecan.
  • Patients with hepatocellular carcinoma must have received sorafenib (Nexavar(R)), since level 1 data support a survival benefit with this agent.
  • Patients with breast and ovarian cancer must be refractory to both first- and second-line treatments and must have received at least one second-line chemotherapy regimen.
  • Patients with 3 or fewer brain metastases that are \< 1 cm in diameter and asymptomatic are eligible. Lesions that have been treated with stereotactic radiosurgery must be clinically stable for one month after treatment for the patient to be eligible. Patients with surgically resected brain metastases are eligible.
  • Age greater than or equal to 18 years and less than or equal to 72 years.
  • Clinical performance status of ECOG 0 or 1.
  • Patients of both sexes must be willing to practice birth control from the time of enrollment on this study and 12 months after the last dose of combined chemotherapy for individuals of child-bearing potential (IOCBP) and for four months after treatment for individuals that can father children.
  • IOCBP must have a negative pregnancy test be a pregnancy test prior to the start of treatment because of the potentially dangerous effects of the treatment on the fetus.
  • Serology
  • Seronegative for HIV antibody. (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who are HIV seropositive may have decreased immune-competence and thus may be less responsive to the experimental treatment and more susceptible to its toxicities.)
  • Seronegative for hepatitis B antigen, and seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then the patient must be tested for the presence of antigen by RT-PCR and be HCV RNA negative.
  • Hematology
  • +13 more criteria

You may not qualify if:

  • Participants who are pregnant or nursing because of the potentially dangerous effects of the treatment on the fetus or infant.
  • Concurrent systemic steroid therapy.
  • Active systemic infections requiring anti-infective treatment, coagulation disorders, or any other active or uncompensated major medical illnesses.
  • Advanced primary with impeding occlusion, perforation or bleeding, dependent on transfusion.
  • Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease and AIDS).
  • History of major organ autoimmune disease.
  • Grade 3 or 4 major organ irAEs clinically attributed to anti-PD-1/PD-L1 therapy.
  • Concurrent opportunistic infections (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who have decreased immunecompetence may be less responsive to the experimental treatment and more susceptible to its toxicities.)
  • History of severe immediate hypersensitivity reaction to cyclophosphamide, fludarabine, or aldesleukin.
  • History of coronary revascularization or ischemic symptoms.
  • For select patients with a clinical history prompting cardiac evaluation: last known LVEF less than or equal to 45%.
  • Documented Child-Pugh score of B or C for hepatocellular carcinoma patients with known underlying liver dysfunction.
  • For select patients with a clinical history prompting pulmonary evaluation: known FEV1 less than or equal to 50%.
  • Patients who are receiving any other investigational agents.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (8)

  • Chiba T, Ohtani H, Mizoi T, Naito Y, Sato E, Nagura H, Ohuchi A, Ohuchi K, Shiiba K, Kurokawa Y, Satomi S. Intraepithelial CD8+ T-cell-count becomes a prognostic factor after a longer follow-up period in human colorectal carcinoma: possible association with suppression of micrometastasis. Br J Cancer. 2004 Nov 1;91(9):1711-7. doi: 10.1038/sj.bjc.6602201.

    PMID: 15494715BACKGROUND
  • Fong Y, Fortner J, Sun RL, Brennan MF, Blumgart LH. Clinical score for predicting recurrence after hepatic resection for metastatic colorectal cancer: analysis of 1001 consecutive cases. Ann Surg. 1999 Sep;230(3):309-18; discussion 318-21. doi: 10.1097/00000658-199909000-00004.

    PMID: 10493478BACKGROUND
  • Tomlinson JS, Jarnagin WR, DeMatteo RP, Fong Y, Kornprat P, Gonen M, Kemeny N, Brennan MF, Blumgart LH, D'Angelica M. Actual 10-year survival after resection of colorectal liver metastases defines cure. J Clin Oncol. 2007 Oct 10;25(29):4575-80. doi: 10.1200/JCO.2007.11.0833.

    PMID: 17925551BACKGROUND
  • Lowery FJ, Goff SL, Gasmi B, Parkhurst MR, Ratnam NM, Halas HK, Shelton TE, Langhan MM, Bhasin A, Dinerman AJ, Dulemba V, Goldlust IS, Gustafson AM, Hakim AA, Hitscherich KJ, Kenney LM, Levy L, Rault-Wang JG, Bera A, Ray S, Seavey CD, Hoang CD, Hernandez JM, Gartner JJ, Sindiri S, Prickett TD, McIntyre LS, Krishna S, Robbins PF, Klemen ND, Kwong MLM, Yang JC, Rosenberg SA. Neoantigen-specific tumor-infiltrating lymphocytes in gastrointestinal cancers: a phase 2 trial. Nat Med. 2025 Jun;31(6):1994-2003. doi: 10.1038/s41591-025-03627-5. Epub 2025 Apr 1.

  • Levin N, Kim SP, Marquardt CA, Vale NR, Yu Z, Sindiri S, Gartner JJ, Parkhurst M, Krishna S, Lowery FJ, Zacharakis N, Levy L, Prickett TD, Benzine T, Ray S, Masi RV, Gasmi B, Li Y, Islam R, Bera A, Goff SL, Robbins PF, Rosenberg SA. Neoantigen-specific stimulation of tumor-infiltrating lymphocytes enables effective TCR isolation and expansion while preserving stem-like memory phenotypes. J Immunother Cancer. 2024 May 30;12(5):e008645. doi: 10.1136/jitc-2023-008645.

  • Zacharakis N, Huq LM, Seitter SJ, Kim SP, Gartner JJ, Sindiri S, Hill VK, Li YF, Paria BC, Ray S, Gasmi B, Lee CC, Prickett TD, Parkhurst MR, Robbins PF, Langhan MM, Shelton TE, Parikh AY, Levi ST, Hernandez JM, Hoang CD, Sherry RM, Yang JC, Feldman SA, Goff SL, Rosenberg SA. Breast Cancers Are Immunogenic: Immunologic Analyses and a Phase II Pilot Clinical Trial Using Mutation-Reactive Autologous Lymphocytes. J Clin Oncol. 2022 Jun 1;40(16):1741-1754. doi: 10.1200/JCO.21.02170. Epub 2022 Feb 1.

  • Malekzadeh P, Pasetto A, Robbins PF, Parkhurst MR, Paria BC, Jia L, Gartner JJ, Hill V, Yu Z, Restifo NP, Sachs A, Tran E, Lo W, Somerville RP, Rosenberg SA, Deniger DC. Neoantigen screening identifies broad TP53 mutant immunogenicity in patients with epithelial cancers. J Clin Invest. 2019 Mar 1;129(3):1109-1114. doi: 10.1172/JCI123791. Epub 2019 Feb 4. No abstract available.

  • Tran E, Turcotte S, Gros A, Robbins PF, Lu YC, Dudley ME, Wunderlich JR, Somerville RP, Hogan K, Hinrichs CS, Parkhurst MR, Yang JC, Rosenberg SA. Cancer immunotherapy based on mutation-specific CD4+ T cells in a patient with epithelial cancer. Science. 2014 May 9;344(6184):641-5. doi: 10.1126/science.1251102.

Related Links

MeSH Terms

Conditions

Colorectal NeoplasmsPancreatic NeoplasmsOvarian NeoplasmsBreast NeoplasmsEndometrial NeoplasmsUrogenital Neoplasms

Interventions

pembrolizumabfludarabineCyclophosphamidealdesleukin

Condition Hierarchy (Ancestors)

Intestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteNeoplasmsDigestive System DiseasesGastrointestinal DiseasesColonic DiseasesIntestinal DiseasesRectal DiseasesEndocrine Gland NeoplasmsPancreatic DiseasesEndocrine System DiseasesOvarian DiseasesAdnexal DiseasesGenital Diseases, FemaleFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesGenital Neoplasms, FemaleGenital DiseasesGonadal DisordersBreast DiseasesSkin DiseasesSkin and Connective Tissue DiseasesUterine NeoplasmsUterine DiseasesMale Urogenital Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Officials

  • Steven A Rosenberg, M.D.

    National Cancer Institute (NCI)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

NCI/Surgery Branch Recruitment Center

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 31, 2010

First Posted

August 3, 2010

Study Start

August 26, 2010

Primary Completion (Estimated)

December 27, 2028

Study Completion (Estimated)

December 27, 2029

Last Updated

June 11, 2026

Record last verified: 2026-05-06

Data Sharing

IPD Sharing
Will share

All IPD recorded in the medical record will be shared with intramural investigators upon request.

Shared Documents
STUDY PROTOCOL, SAP, ICF
Time Frame
Clinical data will be available during the study and indefinitely.
Access Criteria
Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

Locations