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A Pilot Study of Genetically Engineered NY-ESO-1 Specific (c259) T Cells in HLA-A2+ Patients With Synovial Sarcoma

Phase 1
4 Years
55 Years
Open (Enrolling)
Synovial Sarcoma

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Trial Information

A Pilot Study of Genetically Engineered NY-ESO-1 Specific (c259) T Cells in HLA-A2+ Patients With Synovial Sarcoma


- Patients will undergo apheresis at the enrolling institution. Fresh PBMC will be
shipped to University of Pennsylvania and shipped back to the enrolling institution.

- Patients will undergo lymphodepletion with denileukin diftitox, fludarabine and
cyclophosphamide, then infusion of NY-ESO-1 genetically engineered T cells on Day 0.

- Patients will be monitored for toxicity, antitumor effects and immune endpoints.

- Patients with a PR or SD may receive a 2nd cycle no earlier than 60 days following
completion of the first cycle if eligibility criteria are met. For patients with
progressive disease, a 2nd cycle that includes high dose aldesleukin administered
beginning on the day of T cell infusion may be administered no earlier than 60 days
following completion of the first cycle if eligibility criteria are met.

Inclusion Criteria:

- Synovial sarcoma that has been treated with standard chemotherapy containing
doxorubicin and remains: unresectable or metastatic or progressive/persistent or
recurrent disease

- Measurable disease

- Synovial sarcoma and NY-ESO-1+ expression by immunohistochemistry

- HLA-A2+

- Age 4 to less than or equal to 55. NIH 4 to less or equal to 35. Washington
University greater than or equal to 18.

- Weigh more than 18 kg

- All previous cytotoxic chemotherapy, monoclonal antibody therapy, immune, biologic or
molecularly targeted therapy must be completed at least 3 weeks prior to study entry.

- Any grade 3 or 4 non-hematologic toxicity of any previous therapy must have resolved
to grade 2 or less

- Performance Status. ECOG 0, 1 or 2 for age greater than 10 years. Lansky greater
than or equal to 60 for children less than or equal to 10 years of age.

- Life expectancy greater than 3 months

- Left ventricular ejection fraction greater than or equal to 40% or fractional
shortening greater than or equal to 28%

- T.bilirubin < 2 mg/dl (Patients with Gilbert Syndrome exempt)

- AST, ALT less than or equal to 2.5 x upper limit of normal

- ANC > 750/mm3

- Platelets > 75,000/mm3

- Age-adjusted normal serum creatinine or a creatinine clearance greater than or equal
to 60 ml/min/1.73m2

- Ability to give informed consent for patients greater than 18 years of age. For
patients less than 18 years of age the legal guardian must give informed consent.

- Female and male patients (and when relevant their partners) must be willing to
practice birth control (including abstinence) during and for two months after

Exclusion Criteria:

- Clinically significant systemic illness that in the judgment of the PI would
compromise the patient's ability to tolerate protocol therapy or significantly
increase the risk of complications.

- Untreated CNS metastasis

- Previous treatment with genetically engineered NY-ESO-1 specific T cells. Previous
vaccine therapy is not an exclusion criteria.

- Lactating or pregnant females

- Active HIV, HBV or HCV infection

- Patients who require systemic corticosteroid or other immunosuppressive therapy.
Immunosuppressive therapy must be stopped at least 14 days prior to cell infusion.

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Determine the response rate.

Outcome Description:

Disease Staging Evaluation: CT scan chest, head, abdomen, pelvis. MRI of tumro, FDG-PET, Bone Scan if indicated.

Outcome Time Frame:

Day 28, 60, 100, 180; Month 9, 12, then q6 months x 3 yrs

Safety Issue:


Principal Investigator

Crystal Mackall, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Institutes of Health (NIH)


United States: Food and Drug Administration

Study ID:

UPCC 04511



Start Date:

March 2011

Completion Date:

March 2029

Related Keywords:

  • Synovial Sarcoma
  • Sarcoma
  • Sarcoma, Synovial
  • Sarcoma



Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
NIHBethesda, Maryland  20892