NCT00706992

Brief Summary

Background:

  • Melanoma antigen recognized by T cells (MART-1) is a gene that is present in melanoma cells.
  • This study tests an experimental treatment that uses the patient's own lymphocytes (type of white blood cell), which are specially selected and genetically modified with a gene called anti-MART-1 transduced cells (F5) to target and destroy their tumor. Some of the cells are given as an infusion and others are given as a vaccine.
  • The anti-MART-1 F5 cells are currently being studied in other patients in combination with chemotherapy and IL-2 (aldesleukin) therapy. Objectives:
  • To determine if the anti-MART-1 F5 treatment can improve the immune system's ability to shrink tumors and to prevent melanoma from recurring. Eligibility:
  • Patients 18 years of age and older whose melanoma has been removed and are currently disease-free, but who are at risk for recurrence.
  • Patients who do not have ocular or mucosal melanoma.
  • Patients with tissue type human leukocyte antigens (HLA-A)\*0201). Design:
  • Workup: Patients have scans, x-rays, laboratory tests, other tests as needed and leukapheresis, a procedure for collecting white cells to modify in the laboratory and later reinfuse into the patient.
  • Patients are assigned to one of four study groups:
  • Group 1 receives anti-MART-1 F5 cells by 30-minute infusion through a vein on day 0.
  • Group 2 receives anti-MART-1 F5 cells on day 0 followed by injections of MART-1 vaccine, which contains MART-1 and an oil-based liquid called Montanide ISA-51 VG. The vaccine is repeated on day 30.
  • Group 3 receives anti-MART-1 F5 cells on day 0 followed by injections of low-dose IL-2 for 5 days (days 0-4).
  • Group 4 receives anti-MART-1 F5 cells on day 0 followed by MART-1 vaccine and low-dose IL-2 for 5 days. The vaccine is repeated on day 30.
  • Recovery: Patients are monitored closely and given medicines to prevent or treat any side effects of therapy.
  • Leukapheresis: Patients undergo leukapheresis at 1 and 3 months after therapy to collect cells to examine the effects of the treatment on the immune system.
  • Follow-up: Patients return to National Institutes of Health (NIH) 35 days after completing treatment and then at 3 months and every 6 months thereafter for evaluation with a physical examination, review of side effects, laboratory tests and scans. They have blood tests at 3, 6 and 12 months after treatment and then once a year after that. A biopsy may be requested after treatment ends to examine the effects of treatment on the immune system. All patients return to NIH for a physical examination once a year for 5 years and then complete a follow-up questionnaire for another 10 years.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2008

Typical duration 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

Study Start

First participant enrolled

June 1, 2008

Completed
26 days until next milestone

First Submitted

Initial submission to the registry

June 27, 2008

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 30, 2008

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2012

Completed
5 months until next milestone

Results Posted

Study results publicly available

March 19, 2013

Completed
Last Updated

October 28, 2015

Status Verified

October 1, 2015

Enrollment Period

4.4 years

First QC Date

June 27, 2008

Results QC Date

December 18, 2012

Last Update Submit

October 6, 2015

Conditions

Keywords

MART-1 PeptideMelanomaAdjuvant TherapyGene TherapyT Cell Persistence

Outcome Measures

Primary Outcomes (2)

  • Percentage of Participants With Immunologic Response

    Percentage of participants with an immunologic response of \>20 spots/100,000 cells measured by IFN gamma secretion using enzyme linked immunosorbent spot (ELISPOT) assay. This was done using ELISPOT assay which measures immune response at the single cell level.

    9/24/08-10/9/12

  • Number of Participants With Adverse Events

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

    4 years

Study Arms (7)

Arm I - Adj-4 A2 F5 cells

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced peripheral blood lymphocytes (PBLs) intravenously (IV) over 20-30 minutes on day 0. 1 x 10e9 to 5 x 10e10 IV.

Biological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytes

Arm II-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced PBLs as in arm I and MART-1:26-35(27L) peptide vaccine emulsified in Montanide ISA-51 subcutaneously (SC) on days 0 and 30. 1 x 10e9 to 5 x 10e10 IV + 1.0 mg peptide subcutaneously.

Biological: MART-1:26-35(27L) peptide vaccineBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytesBiological: incomplete Freund's adjuvant

Arm III-Adj-4 A2 F5 cells + SQ IL-2

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced PBLs as in arm I and aldesleukin SC on days 0-4. 1 x 10e9 to 5 x 10e10 IV + IL-2 (based on body weight) 125,000 IU/kg/day subcutaneously.

Biological: AldesleukinBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytes

Arm IV-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide+SQ IL-2

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced PBLs as in arm I, MART-1:26-35(27L) peptide vaccine emulsified in Montanide ISA-51 as in arm II, and aldesleukin as in arm III. 1 x 10e9 to 5 x 10e10 IV + 1.0 mg peptide subcutaneously + IL-2 (based on body weight) 125,000 IU/kg/day subcutaneously.

Biological: MART-1:26-35(27L) peptide vaccineBiological: AldesleukinBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytesBiological: incomplete Freund's adjuvant

Arm V-Adj-4 A2 F5 cells + ALVAC MART-1:26-35(27L) Vaccine

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced PBLs IV over 20-30 minutes on day 0, and ALVAC-MART-1 vaccine SC on days 0 and 14. 1 x 10e9 to 5 x 10e10 IV + ALVAC vaccine 0.5 ml containing target dose of 10e7 CCID50 (with a range of approximately 10\^6.4 to 10\^7.9/mL subcutaneously (total of 4 x 10e7 CCID50/2 mL).

Biological: ALVAC-MART-1 vaccineBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytes

Arm VI-Adj-4 A2 F5 cells + ALVAC MART-1 VAccine + SQ IL-2

EXPERIMENTAL

Patients receive anti-MART-1 F5 TCR-transduced PBLs and ALVAC-MART-1 vaccine as in arm V, and low-dose aldesleukin SC on days 0-4. 1 x 10e9 to 5 x 10e10 IV + ALVAC vaccine 0.5 ml containing target dose of 10e7 CCID50 (with a range of approximately 10\^6.4 to 10\^7.9/mL subcutaneously (total of 4 x 10e7 CCID50/2 mL)+ 125,000 IU/kg/day subcutaneously.

Biological: ALVAC-MART-1 vaccineBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytes

Arm VII-Adj-4 A2 ALVAC MART-1:26-35(27L) Vaccine

EXPERIMENTAL

Patients receive ALVAC-MART-1 vaccine SC on days 0 and 14. ALVAC vaccine 0.5 ml containing target dose of 10e7 CCID50 (with a range of approximately 10\^6.4 to 10\^7.9/mL subcutaneously (total of 4 x 10e7 CCID50/2 mL).

Biological: ALVAC-MART-1 vaccineBiological: AldesleukinBiological: autologous anti-MART-1 F5 T-cell receptor gene-engineered peripheral blood lymphocytes

Interventions

Given subcutaneously

Arm V-Adj-4 A2 F5 cells + ALVAC MART-1:26-35(27L) VaccineArm VI-Adj-4 A2 F5 cells + ALVAC MART-1 VAccine + SQ IL-2Arm VII-Adj-4 A2 ALVAC MART-1:26-35(27L) Vaccine

Given subcutaneously

Arm II-Adj-4 A2 F5 cells + MART-1:26-35(27L) PeptideArm IV-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide+SQ IL-2
AldesleukinBIOLOGICAL

Given subcutaneously

Arm III-Adj-4 A2 F5 cells + SQ IL-2Arm IV-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide+SQ IL-2Arm VII-Adj-4 A2 ALVAC MART-1:26-35(27L) Vaccine

Given intravenously (IV)

Arm I - Adj-4 A2 F5 cellsArm II-Adj-4 A2 F5 cells + MART-1:26-35(27L) PeptideArm III-Adj-4 A2 F5 cells + SQ IL-2Arm IV-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide+SQ IL-2Arm V-Adj-4 A2 F5 cells + ALVAC MART-1:26-35(27L) VaccineArm VI-Adj-4 A2 F5 cells + ALVAC MART-1 VAccine + SQ IL-2Arm VII-Adj-4 A2 ALVAC MART-1:26-35(27L) Vaccine

Given subcutaneously

Arm II-Adj-4 A2 F5 cells + MART-1:26-35(27L) PeptideArm IV-Adj-4 A2 F5 cells + MART-1:26-35(27L) Peptide+SQ IL-2

Eligibility Criteria

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

You may qualify if:

  • Primary melanomas with lesions that are ulcerated and greater than or equal to 2.0 mm, or any lesions that are greater than or equal to 4.0 mm in thickness, or greater than or equal to 1 positive lymph node, or local recurrence, or resected metastatic disease, within 6 months of surgical resection will be considered. Patients must be clinically disease free at the time of protocol entry as documented by radiologic studies within 6 weeks of patient entry. Patients must have pathologic confirmation of cutaneous melanoma, with slides reviewed at National Institutes of Health (NIH) (Department of Anatomic Pathology), and if the diagnosis is not confirmed, the patient will be excluded from the study.
  • Human leukocyte antigens (HLA-A) 0201 positive.
  • Age greater than or equal to18 years.
  • Clinical performance status of Eastern Cooperative Oncology Group (ECOG) 0 or 1.
  • Able to understand and sign the Informed Consent Document.
  • Patients of both genders must be willing to practice effective birth control during this trial because the potential for teratogenic effects are unknown. Effective birth control requires use of an effective method from the following list: Abstinence, Intrauterine device (IUD); Hormonal (Birth control pills, injections, implants); Tubal ligation; Cervical cap; or Partner's vasectomy
  • Patients may have had prior adjuvant treatment with immunotherapy, including interferon, as long as 3 weeks have elapsed since prior systemic therapy.
  • 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 (The experimental treatment being evaluated in this protocol depends upon an intact immune system and these conditions may have possible immune system effects).
  • Hematology:
  • Absolute neutrophil count greater than 1000/mm\^3 without the support of filgrastim.
  • White blood cell (WBC) (greater than 3000/mm\^3).
  • Platelet count greater than 90,000/mm\^3.
  • Hemoglobin greater than 8.0 g/dl.
  • +4 more criteria

You may not qualify if:

  • Ocular or mucosal melanoma.
  • Undergoing or have undergone in the past 3 weeks any systemic therapy except surgery for their cancer, and must have recovered to a grade 1 from any adverse effects of treatment prior to entry, other than those that do not have clinical implications, e.g. vitiligo, alopecia.
  • Have autoimmune disease (such as autoimmune colitis or Crohn's disease) or any known immunodeficiency disease, as evidenced by abnormal white blood count (WBC) count.
  • Concurrent systemic steroid therapy.
  • Known systemic hypersensitivity to any of the vaccine components, including egg products or Neomycin.
  • Women of child-bearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the treatment on the fetus or infant.
  • Have active systemic infections including 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).
  • Previous immunization with melanoma antigen recognized by T cells (MART-1).
  • Known hypersensitivity to any of the agents used in this study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Cancer Institute (NCI), 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

  • Schwartz RH. T cell clonal anergy. Curr Opin Immunol. 1997 Jun;9(3):351-7. doi: 10.1016/s0952-7915(97)80081-7.

    PMID: 9203408BACKGROUND
  • Rosenberg SA, Yang JC, Restifo NP. Cancer immunotherapy: moving beyond current vaccines. Nat Med. 2004 Sep;10(9):909-15. doi: 10.1038/nm1100.

    PMID: 15340416BACKGROUND
  • Dudley ME, Wunderlich JR, Robbins PF, Yang JC, Hwu P, Schwartzentruber DJ, Topalian SL, Sherry R, Restifo NP, Hubicki AM, Robinson MR, Raffeld M, Duray P, Seipp CA, Rogers-Freezer L, Morton KE, Mavroukakis SA, White DE, Rosenberg SA. Cancer regression and autoimmunity in patients after clonal repopulation with antitumor lymphocytes. Science. 2002 Oct 25;298(5594):850-4. doi: 10.1126/science.1076514. Epub 2002 Sep 19.

    PMID: 12242449BACKGROUND

MeSH Terms

Conditions

Melanoma

Interventions

Protein Subunit Vaccinesaldesleukinincomplete Freund's adjuvant

Condition Hierarchy (Ancestors)

Neuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Nerve TissueNevi and MelanomasSkin NeoplasmsNeoplasms by SiteSkin DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Vaccines, AcellularVaccines, SubunitVaccinesBiological ProductsComplex Mixtures

Results Point of Contact

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

Study Officials

  • Steven 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
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NIH
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

June 27, 2008

First Posted

June 30, 2008

Study Start

June 1, 2008

Primary Completion

November 1, 2012

Study Completion

November 1, 2012

Last Updated

October 28, 2015

Results First Posted

March 19, 2013

Record last verified: 2015-10

Locations