NCT01273181

Brief Summary

Background: \- MAGE-A3/12 is a type of protein commonly found on certain types of cancer cells, particularly in metastatic cancer. Researchers have developed a process to take lymphocytes (white blood cells) from cancer patients, modify them in the laboratory to target cancer cells that contain MAGE-A3/12, and return them to the patient to help attack and kill the cancer cells. These modified white blood cells are an experimental treatment, but researchers are interested in determining their safety and effectiveness as a possible treatment for cancers that involve MAGE-A3/12. Objectives: \- To evaluate the safety and effectiveness of anti-MAGE-A3/12 lymphocytes as a treatment for metastatic cancers that have not responded to standard treatment. Eligibility: \- Individuals at least 18 years of age who have been diagnosed with metastatic melanoma, renal cell cancer, or another type of metastatic cancer that has not responded to standard treatment. Design:

  • Participants will be screened with a full medical history and physical examination, as well as blood and urine tests, tumor samples, and imaging studies.
  • Participants will have leukapheresis to collect enough white blood cells for modification in the laboratory.
  • Seven days before the start of anti-MAGE-A3/12 treatment, participants will have chemotherapy with cyclophosphamide and fludarabine to suppress the immune system in preparation for the treatment.
  • After the last dose of chemotherapy, participants will receive the anti-MAGE-A3/12 cells as an infusion for 20 to 30 minutes, followed by a dose of interleukin-2 to keep the anti-MAGE-A3/12 cells alive and active as long as possible. Participants will also receive filgrastim to encourage the production of blood cells.
  • Participants will remain in the hospital to be monitored for possible side effects, and after release from the hospital will have regular followup exams with blood samples and imaging studies to evaluate the effectiveness of the treatment....

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Dec 2010

Typical duration for phase_1

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

Study Start

First participant enrolled

December 1, 2010

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

January 7, 2011

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 10, 2011

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2012

Completed
4 months until next milestone

Results Posted

Study results publicly available

March 27, 2013

Completed
Last Updated

October 28, 2015

Status Verified

October 1, 2015

Enrollment Period

2 years

First QC Date

January 7, 2011

Results QC Date

February 14, 2013

Last Update Submit

October 6, 2015

Conditions

Keywords

ImmunotherapyGene TherapyMetastatic CancerClinical ResponseMetastatic MelanomaMetastatic Renal Cell Cancer

Outcome Measures

Primary Outcomes (2)

  • Toxicity Profile

    Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module.

    2 years

  • Clinical Tumor Regression (Complete Response (CR) + Partial Response (PR)) in Patients With Metastatic Cancer

    Tumor regression response is assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response (CR) is a 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.

    2 years

Study Arms (4)

Ph I:Anti-MAGE A3/12 TCR PBL 5x10e9

EXPERIMENTAL

Cyclophosphamide : 60 mg/kg/day x 2 days intravenous (IV) Aldesleukin : 720,000 IU/kg every 8 hours for a maximum of 15 doses PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes : Fludarabine : 25 mg/m\^2/day intravenous piggy back (IVPB) daily over 30 minutes for 5 days. Note for phase I: The study will begin by evaluating the safety of two ranges of cells, 5x10\^9-3x10\^10, and greater than 3x10\^10-1x10\^11 in a standard phase I dose escalation fashion using a 3+3 design.

Biological: PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood LymphocytesDrug: AldesleukinDrug: CyclophosphamideDrug: Fludarabine

Ph I:Anti-MAGE A3/12 TCR PBL 3x10e10

EXPERIMENTAL

Cyclophosphamide : 60 mg/kg/day x 2 days intravenous (IV) Aldesleukin : 720,000 IU/kg every 8 hours for a maximum of 15 doses PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes : Fludarabine : 25 mg/m\^2/day intravenous piggy back (IVPB) daily over 30 minutes for 5 days. Note for phase I: The study will begin by evaluating the safety of two ranges of cells, 5x10\^9-3x10\^10, and greater than 3x10\^10-1x10\^11 in a standard phase I dose escalation fashion using a 3+3 design.

Biological: PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood LymphocytesDrug: AldesleukinDrug: CyclophosphamideDrug: Fludarabine

Ph II:Anti-MAGE TCR PBL MTD+HD IL-2

EXPERIMENTAL

Phase II:Anti-MAGE A3/12 TCR PBL MTD + HD IL-2, Melanoma, RCC Cyclophosphamide : 60 mg/kg/day x 2 days intravenous (IV) Aldesleukin : 720,000 IU/kg every 8 hours for a maximum of 15 doses PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes : Fludarabine : 25 mg/m\^2/day intravenous piggy back (IVPB) daily over 30 minutes for 5 days. Note for phase II:patients will be entered into two cohorts based on histology:cohort 1 will include patients with metastatic melanoma or renal cell cancer; cohort 2 will include patients with other types of metastatic cancer.

Biological: PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood LymphocytesDrug: AldesleukinDrug: CyclophosphamideDrug: Fludarabine

Ph II:Anti-MAGE A3/12 TCR PBL MTD

EXPERIMENTAL

Phase II: Anti-MAGE A3/12 TCR PBL MTD + HD-IL2 Other Cancer Cyclophosphamide : 60 mg/kg/day x 2 days intravenous (IV) Aldesleukin : 720,000 IU/kg every 8 hours for a maximum of 15 doses PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes : Fludarabine : 25 mg/m\^2/day intravenous piggy back (IVPB) daily over 30 minutes for 5 days. Note for phase II:patients will be entered into two cohorts based on histology:cohort 1 will include patients with metastatic melanoma or renal cell cancer; cohort 2 will include patients with other types of metastatic cancer.

Biological: PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood LymphocytesDrug: AldesleukinDrug: CyclophosphamideDrug: Fludarabine

Interventions

Ph I:Anti-MAGE A3/12 TCR PBL 3x10e10Ph I:Anti-MAGE A3/12 TCR PBL 5x10e9Ph II:Anti-MAGE A3/12 TCR PBL MTDPh II:Anti-MAGE TCR PBL MTD+HD IL-2

720,000 IU/kg every 8 hours for a maximum of 15 doses

Also known as: IL-2
Ph I:Anti-MAGE A3/12 TCR PBL 3x10e10Ph I:Anti-MAGE A3/12 TCR PBL 5x10e9Ph II:Anti-MAGE A3/12 TCR PBL MTDPh II:Anti-MAGE TCR PBL MTD+HD IL-2

60 mg/kg/day x 2 days intravenous (IV)over 1 hour.

Ph I:Anti-MAGE A3/12 TCR PBL 3x10e10Ph I:Anti-MAGE A3/12 TCR PBL 5x10e9Ph II:Anti-MAGE A3/12 TCR PBL MTDPh II:Anti-MAGE TCR PBL MTD+HD IL-2

25 mg/m\^2/day intravenous piggy back (IVPB) daily over 30 minutes for 5 days.

Ph I:Anti-MAGE A3/12 TCR PBL 3x10e10Ph I:Anti-MAGE A3/12 TCR PBL 5x10e9Ph II:Anti-MAGE A3/12 TCR PBL MTDPh II:Anti-MAGE TCR PBL MTD+HD IL-2

Eligibility Criteria

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

You may qualify if:

  • Metastatic cancer that expresses MAGE-A3/12 as assessed by one of the following methods: reverse transcription polymerase chain reaction (RT-PCR) on tumor tissue defined as 30,000 copies of MAGE-A3/12 per 106 GAPDH copies, or by immunohistochemistry of resected tissue defined as 10% or greater of cells being 2-3+, or serum antibody reactive with MAGE-A3/12. Metastatic cancer diagnosis will be confirmed by the Laboratory of Pathology at the National Cancer Institute (NCI).
  • Patients with melanoma or renal cell cancer must have previously received high dose aldesleukin and have been either non-responders (progressive disease) or have recurred. Patients with other histologies, must have previously received at least one 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.
  • 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.
  • Hematology:
  • Absolute neutrophil count greater than 1000/mm\^3 without the support of filgrastim.
  • White blood cell (WBC) (\> 3000/mm\^3).
  • +7 more criteria

You may not qualify if:

  • 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
  • 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%.
  • Documented LVEF of less than or equal to 45% 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 1 (FEV1) less than or equal to 60% predicted tested in patients with:
  • A prolonged history of cigarette smoking (20 pk/year of smoking within the past 2 years).
  • Symptoms of respiratory dysfunction

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 (5)

  • Rosenberg SA. Progress in human tumour immunology and immunotherapy. Nature. 2001 May 17;411(6835):380-4. doi: 10.1038/35077246.

    PMID: 11357146BACKGROUND
  • Berendt MJ, North RJ. T-cell-mediated suppression of anti-tumor immunity. An explanation for progressive growth of an immunogenic tumor. J Exp Med. 1980 Jan 1;151(1):69-80. doi: 10.1084/jem.151.1.69.

    PMID: 6444236BACKGROUND
  • Gattinoni L, Powell DJ Jr, Rosenberg SA, Restifo NP. Adoptive immunotherapy for cancer: building on success. Nat Rev Immunol. 2006 May;6(5):383-93. doi: 10.1038/nri1842.

    PMID: 16622476BACKGROUND
  • Abate-Daga D, Hanada K, Davis JL, Yang JC, Rosenberg SA, Morgan RA. Expression profiling of TCR-engineered T cells demonstrates overexpression of multiple inhibitory receptors in persisting lymphocytes. Blood. 2013 Aug 22;122(8):1399-410. doi: 10.1182/blood-2013-04-495531. Epub 2013 Jul 16.

  • Morgan RA, Chinnasamy N, Abate-Daga D, Gros A, Robbins PF, Zheng Z, Dudley ME, Feldman SA, Yang JC, Sherry RM, Phan GQ, Hughes MS, Kammula US, Miller AD, Hessman CJ, Stewart AA, Restifo NP, Quezado MM, Alimchandani M, Rosenberg AZ, Nath A, Wang T, Bielekova B, Wuest SC, Akula N, McMahon FJ, Wilde S, Mosetter B, Schendel DJ, Laurencot CM, Rosenberg SA. Cancer regression and neurological toxicity following anti-MAGE-A3 TCR gene therapy. J Immunother. 2013 Feb;36(2):133-51. doi: 10.1097/CJI.0b013e3182829903.

Related Links

MeSH Terms

Conditions

Neoplasm MetastasisKidney NeoplasmsMelanomaCarcinoma, Renal Cell

Interventions

aldesleukinInterleukin-2Cyclophosphamidefludarabine

Condition Hierarchy (Ancestors)

Neoplastic ProcessesNeoplasmsPathologic ProcessesPathological Conditions, Signs and SymptomsUrologic NeoplasmsUrogenital NeoplasmsNeoplasms by SiteFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesKidney DiseasesUrologic DiseasesMale Urogenital DiseasesNeuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Nerve TissueNevi and MelanomasSkin NeoplasmsSkin DiseasesSkin and Connective Tissue DiseasesAdenocarcinomaCarcinomaNeoplasms, Glandular and Epithelial

Intervention Hierarchy (Ancestors)

InterleukinsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsLymphokinesProteinsBiological FactorsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Results Point of Contact

Title
Dr. Steven Rosenberg
Organization
National Cancer Institute, National Institutes of Health

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 1
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

January 7, 2011

First Posted

January 10, 2011

Study Start

December 1, 2010

Primary Completion

December 1, 2012

Study Completion

December 1, 2012

Last Updated

October 28, 2015

Results First Posted

March 27, 2013

Record last verified: 2015-10

Locations