NCT00670748

Brief Summary

Background:

  • This study uses an experimental cancer treatment that uses the patient s own lymphocytes (type of white blood cell), which are specially selected and genetically modified to target and destroy their tumor. Objectives:
  • To test the safety of the treatment and determine if it can cause the patient s tumor to shrink. Eligibility:
  • Patients greater than 18 years and less than or equal to 66 years of age whose cancer has spread beyond the original site and does not respond to standard treatment.
  • Patients have tissue type human leukocyte antigen (HLA)-A\*0201.
  • Patients cancer cells have the ESO-1 gene. Design:
  • Workup: Patients have scans, x-rays, laboratory tests, and other tests as needed.
  • Patients have leukapheresis to collect cells for laboratory treatment and later reinfusion. For this procedure, whole blood is collected thorough a tube in a vein, the desired cells are extracted from the blood, and the rest of the blood is returned to the patient.
  • Chemotherapy: Patients have low-dose chemotherapy for 1 week to prepare the immune system to receive the treated lymphocytes.
  • Cell infusion and aldesleukin (IL-2) treatment: Patients receive the lymphocytes by a 30-minute infusion through a vein. Starting within 24 hours of the infusion, they receive high-dose aldesleukin infusions every 8 hours for up to 5 days (maximum15 doses).
  • Recovery: Patients rest for 1 to 2 weeks to recover from the effects of chemotherapy and aldesleukin.
  • Tumor biopsy: Patients may be asked to undergo a biopsy (surgical removal of a small piece of tumor) after treatment to look at the effects of treatment on the immune cells in the tumor.
  • Follow-up: After treatment is completed, patients return to the clinic once a month for several months for physical examinations, a review of side effects, laboratory tests and scans. They may undergo leukapheresis at some visits to look at the effect of treatment on the immune system and check the viability of the infused cells. Patients then return to the National Institute of Health (NIH) clinic once a year for 5 years and then complete a follow-up questionnaire for another 10 years.
  • Retreatment: Patients whose tumor shrinks or disappears following treatment and then recurs may receive one additional treatment, using the same regimen of chemotherapy, lymphocyte infusion and IL-2 treatment.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
45

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started May 2008

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

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

First Submitted

Initial submission to the registry

May 1, 2008

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 2, 2008

Completed
27 days until next milestone

Study Start

First participant enrolled

May 29, 2008

Completed
8.1 years until next milestone

Results Posted

Study results publicly available

June 27, 2016

Completed
2 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 29, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 29, 2016

Completed
Last Updated

November 19, 2019

Status Verified

November 1, 2019

Enrollment Period

8.1 years

First QC Date

May 1, 2008

Results QC Date

May 19, 2016

Last Update Submit

November 6, 2019

Conditions

Keywords

Metastatic CancerGene TherapyImmunotherapyTumor RegressionMetastatic MelanomaMetastatic Renal Cell Cancer

Outcome Measures

Primary Outcomes (1)

  • Clinical Response Per the Response Evaluation Criteria in Solid Tumors (RECIST)

    Response was determined by the RECIST. Complete response (CR) is disappearance of all target lesions. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD. Progressive disease (PD) is at least a 20% increase in the sum of LD of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum LD.

    Approximately 3 years

Secondary Outcomes (2)

  • Number of Participants With In Vivo Survival of T-Cell Receptor (TCR)-Engineered Cells

    1 month post treatment

  • Number of Participants With Serious and Non-Serious Adverse Events

    Date treatment consent signed to date off study, approximately, 66 months and 10 days

Study Arms (4)

#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC

EXPERIMENTAL

Patients with melanoma or renal cell cancer (RCC) will receive non-myeloablative lymphodepleting regimen of cyclophosphamide and fludarabine followed by anti-NY ESO-1 T-cell receptor (TCR) peripheral blood lymphocytes (PBL) and high dose aldesleukin.

Biological: Anti-NY ESO-1 T-cell receptor PBLDrug: aldesleukinDrug: CyclophosphamideDrug: fludarabine phosphate

#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa

EXPERIMENTAL

Patients with cancers other than melanoma or RCC will receive non-myeloablative lymphodepleting regimen of cyclophosphamide and fludarabine followed by anti-NY ESO-1 TCR PBL and high dose (HD) aldesleukin

Biological: Anti-NY ESO-1 T-cell receptor PBLDrug: aldesleukinDrug: CyclophosphamideDrug: fludarabine phosphate

#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC

EXPERIMENTAL

Patients with melanoma or RCC will receive non-myeloablative lymphodepleting regimen of cyclophosphamide and fludarabine followed by replication-defective recombinant canarypox virus (ALVAC) NY-ESO-1 vaccine, anti-NY ESO-1 TCR PBL and high dose aldesleukin

Biological: Anti-NY ESO-1 T-cell receptor PBLDrug: aldesleukinDrug: CyclophosphamideDrug: fludarabine phosphateBiological: ALVAC NY ESO-1 vaccine

#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

EXPERIMENTAL

Patients with cancers other than melanoma or RCC will receive non-myeloablative lymphodepleting regimen of cyclophosphamide and fludarabine followed by ALVAC NY-ESO-1 vaccine, anti-NY ESO-1 TCR PBL and high dose aldesleukin

Biological: Anti-NY ESO-1 T-cell receptor PBLDrug: aldesleukinDrug: CyclophosphamideDrug: fludarabine phosphateBiological: ALVAC NY ESO-1 vaccine

Interventions

Patients will receive non-myeloablative lymphodepleting preparative regimen consisting of cyclophosphamide and fludarabine followed by the administration of anti-NY ESO-1 T-cell receptor (TCR) peripheral blood lymphocytes (PBL) and high dose aldesleukin. On day 0,cells (1x10e8 to 1x10e11)will be infused intravenously on the Patient Care Unit over 20-30 minutes.

#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

Aldesleukin 720,000 IU/kg intravenous (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 5 days(maximum of 15 doses)

Also known as: Proleukin
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

Cyclophosphamide 60 mg/kg/day X 2 days IV in 250 ml dextrose 5% in water (D5W) with Mesna 15 mg/kg/day X 2 days over 1 hr.

Also known as: Cytoxan
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

Fludarabine 25 mg/m2/day intravenous piggyback (IVPB) daily over 30 minutes for 5 days.

Also known as: Fludara
#1 Anti-NY-ESO-1 TCR PBL+HD IL-2 Mel/RCC#2 Anti-NY-ESO-1 TCR PBL+HD IL-2 OtherCa#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

Approximately two hours prior to cell infusion, patients will receive 0.5 mL containing a target dose of 10e7 cell culture infectious dose 50 (CCID50) (with a range of approximately 10e6.4 to 10e7.9 / mL) of the ESO-1 ALVAC virus S.C. in each extremity (total of 4 x 10e7 CCID50/2 mL.

#3ESO1 TCR PBL+ALVAC ESO1+HD IL2 Mel/RCC#4ESO1 TCR PBL+ALVAC ESO1+HD IL2 OtherCa

Eligibility Criteria

Age18 Years - 66 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Metastatic cancer that expresses ESO as assessed by one of the following methods: reverse transcription-polymerase chain reaction (RT-PCR) on tumor tissue, or by immunohistochemistry of resected tissue, or serum antibody reactive with ESO. Metastatic cancer diagnosis will be confirmed by the Laboratory of Pathology at the National Cancer Institute (NCI).
  • Patients with histologies other than metastatic melanoma, must have previously received systemic standard care (or effective salvage chemotherapy regimens) for metastatic disease, if known to be effective for that disease, and have been either non-responders (progressive disease) or have recurred.
  • Greater than or equal to 18 years of age. and less than or equal to 66 years of age.
  • Willing to sign a durable power of attorney.
  • Able to understand and sign the Informed Consent Document.
  • Clinical performance status of Eastern Cooperative Oncology Group (ECOG) 0 or 1.
  • Life expectancy of greater than three months.
  • Patients of both genders must be willing to practice birth control for four months after receiving the preparative regimen.
  • Patients must be human leukocyte antigen (HLA)-A\*0201 positive
  • Serology:
  • Seronegative for human immunodeficiency virus (HIV) antibody. (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who are HIV seropositive can have decreased immune-competence and thus be less responsive to the experimental treatment and more susceptible to its toxicities.)
  • Seronegative for hepatitis B antigen and hepatitis C antibody unless antigen negative. If hepatitis C antibody test is positive, then patients must be tested for the presence of antigen by RT-PCR and be hepatitis C virus ribonucleic acid (HCV RNA) negative.
  • Hematology:
  • Absolute neutrophil count greater than 1000/mm(3) without the support of filgrastim.
  • White blood cell (WBC) (greater than 3000/mm(3)).
  • +9 more criteria

You may not qualify if:

  • Prior vaccination with an replication-defective recombinant canarypox virus (ALVAC) containing vaccine for patients who will receive the ALVAC ESO-1 vaccine (cohorts 3 or 4).
  • Women of child-bearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the preparative chemotherapy on the fetus or infant.
  • Active systemic infections, coagulation disorders or other major medical illnesses of the cardiovascular, respiratory or immune system, myocardial infarction, cardiac arrhythmias, obstructive or restrictive pulmonary disease.
  • Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease).
  • Concurrent opportunistic infections (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who have decreased immune competence may be less responsive to the experimental treatment and more susceptible to its toxicities).
  • Concurrent Systemic steroid therapy.
  • Known systemic hypersensitivity to any of the vaccine components, including egg products or Neomycin for patients who will receive the ALVAC ESO-1 vaccine (cohorts 3 or 4).
  • History of severe immediate hypersensitivity reaction to any of the agents used in this study.
  • History of coronary revascularization or ischemic symptoms.
  • Any patient known to have an left ventricular ejection fraction (LVEF) less than or equal to 45 percent.
  • Documented forced expiratory volume (LVEF) of less than or equal to 45 percent tested in patients with:
  • History of ischemic heart disease, chest pain, or clinically significant atrial and/or ventricular arrhythmias including but not limited to: atrial fibrillation, ventricular tachycardia, second or third degree heart block.
  • Age greater than or equal to 60 years old.
  • Documented forced expiratory volume in 1 second (FEV1) less than or equal to 60 percent predicted tested in patients with:
  • A prolonged history of cigarette smoking (20 pk/year of smoking within the past 2 years).
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (4)

  • Zeng G, Touloukian CE, Wang X, Restifo NP, Rosenberg SA, Wang RF. Identification of CD4+ T cell epitopes from NY-ESO-1 presented by HLA-DR molecules. J Immunol. 2000 Jul 15;165(2):1153-9. doi: 10.4049/jimmunol.165.2.1153.

    PMID: 10878395BACKGROUND
  • Zhao Y, Bennett AD, Zheng Z, Wang QJ, Robbins PF, Yu LY, Li Y, Molloy PE, Dunn SM, Jakobsen BK, Rosenberg SA, Morgan RA. High-affinity TCRs generated by phage display provide CD4+ T cells with the ability to recognize and kill tumor cell lines. J Immunol. 2007 Nov 1;179(9):5845-54. doi: 10.4049/jimmunol.179.9.5845.

    PMID: 17947658BACKGROUND
  • Valmori D, Dutoit V, Lienard D, Rimoldi D, Pittet MJ, Champagne P, Ellefsen K, Sahin U, Speiser D, Lejeune F, Cerottini JC, Romero P. Naturally occurring human lymphocyte antigen-A2 restricted CD8+ T-cell response to the cancer testis antigen NY-ESO-1 in melanoma patients. Cancer Res. 2000 Aug 15;60(16):4499-506.

    PMID: 10969798BACKGROUND
  • Crompton JG, Sukumar M, Roychoudhuri R, Clever D, Gros A, Eil RL, Tran E, Hanada K, Yu Z, Palmer DC, Kerkar SP, Michalek RD, Upham T, Leonardi A, Acquavella N, Wang E, Marincola FM, Gattinoni L, Muranski P, Sundrud MS, Klebanoff CA, Rosenberg SA, Fearon DT, Restifo NP. Akt inhibition enhances expansion of potent tumor-specific lymphocytes with memory cell characteristics. Cancer Res. 2015 Jan 15;75(2):296-305. doi: 10.1158/0008-5472.CAN-14-2277. Epub 2014 Nov 28.

Related Links

MeSH Terms

Conditions

MelanomaCarcinoma, Renal CellNeoplasm Metastasis

Interventions

aldesleukinCyclophosphamidefludarabine phosphate

Condition Hierarchy (Ancestors)

Neuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Nerve TissueNevi and MelanomasSkin NeoplasmsNeoplasms by SiteSkin DiseasesSkin and Connective Tissue DiseasesAdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialKidney NeoplasmsUrologic NeoplasmsUrogenital NeoplasmsFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesKidney DiseasesUrologic DiseasesMale Urogenital DiseasesNeoplastic ProcessesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Results Point of Contact

Title
Dr. Steven A. Rosenberg
Organization
National Cancer Institute

Study Officials

  • Steven A Rosenberg, M.D.

    National Cancer Institute (NCI)

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 1, 2008

First Posted

May 2, 2008

Study Start

May 29, 2008

Primary Completion

June 29, 2016

Study Completion

June 29, 2016

Last Updated

November 19, 2019

Results First Posted

June 27, 2016

Record last verified: 2019-11

Data Sharing

IPD Sharing
Will not share

Locations