Trivalent Ganglioside Vaccine With Immunological Adjuvant or Immunological Adjuvant Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free
Condition: Sarcoma
MeSH terms: Sarcoma
Intervention: Trivalent ganglioside vaccine; OPT-821
Intervention description: Patients will be given 10 injections of ganglioside vaccine plus adjuvant OPT-821 as a 1.0 ml subcutaneous injection in an outpatient setting at Visit Weeks 1, 2, 3, 8, 16, 28, 40, 52, 68 and 84; Patients will be given 10 injections of adjuvant OPT-821 as a 1.0 ml subcutaneous injection in an outpatient setting at Visit Weeks 1, 2, 3, 8, 16, 28, 40, 52, 68 and 84
Intervention MeSH terms: Vaccines; Adjuvants, Immunologic
Phase: Phase 2
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 136.0
Sex: All
Minimum age: 16 Years
Healthy volunteers: No
Keywords: Sarcoma; Metastatic; Vaccine; Adjuvant

Summary:

      Sarcoma patients are at high risk for their cancer to recur even when the sarcoma has been
      removed surgically or treated with radiation or chemotherapy. The patients in the study will
      be randomized (like flipping a coin) to receive either a vaccine that is combined with an
      immune system stimulant or the immune system stimulant alone. The immune system stimulant is
      called OPT-821 and is an immunological booster. The trivalent vaccine is being developed to
      teach the patient's immune system to recognize 3 types of sugars called GM2, GD2 and GD3 that
      are found primarily on the surface of sarcoma cells. If the trivalent vaccine can stimulate
      the patient's immune system to develop antibodies which recognize and target the GM2, GD2 and
      GM3 sugars, then the patient's antibodies could attack and kill any remaining sarcoma cells
      potentially preventing the recurrence of sarcoma.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Male or female, 16 years or older.

          2. American Joint Committee on Cancer (AJCC) Stage IV sarcoma with no current
             radiological evidence of residual disease following either surgery alone or
             multi-modality therapy for treatment of metastatic or relapsed disease. Patients must
             have presented with either newly diagnosed metastatic sarcoma or distant relapsed
             disease. Patients who present with more than one site of metastases are eligible as
             long as at least one new site is distant from the original site and the surgical
             resection(s) results in clear margins as assessed by the site pathologist.
             Non-surgical local ablative therapies such as SRS or cryotherapy cannot replace
             surgical resection of disease for the purpose of eligibility.

          3. Histological confirmation of sarcoma, as performed by a pathologist at one of the
             participating study sites, prior to entry on study.

          4. Patients must have undergone surgical metastectomy within 8 weeks prior to initiation
             of treatment on this study.

          5. Patients previously treated with neoadjuvant chemotherapy and/or radiotherapy as part
             of a multi-modality treatment for metastatic disease must have recovered from all
             adverse effects of treatment and have returned to baseline status.

          6. Imaging study performed within 4 weeks prior to administration of first vaccination
             documenting that patient has no evidence of disease. Study must include CT scan of
             chest, abdomen, and pelvis.

          7. Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 1.

          8. Weight ≥ 40 kg.

          9. Have organ and marrow function as defined below:

             WBC ≥ 3.0 cells/mm3 Platelets ≥ 100,000/mm3 Total bilirubin ≤ 2.0 mg/dL AST (SGOT)/ALT
             (SGPT) ≤ 1.5 x ULN

         10. Current use of an acceptable form of birth control.

         11. Ability to understand English and to provide written informed consent and
             authorization for protected health information disclosure whether by self or by
             legally authorized representative.

        Exclusion Criteria:

          1. Patients with evidence of local or metastatic disease or who are not disease free at
             the time of the first vaccination.

          2. Patients who develop locally recurrent disease only with no evidence of concurrent or
             previous distant metastatic disease. Patients with a primary retroperitoneal and/or
             uterine sarcoma that present with recurrence within the retroperitoneum or pelvis only
             are not eligible. Patients must have evidence of hematogenously disseminated distant
             disease.

          3. Patients with brain or bone metastasis even if they are able to undergo complete
             surgical resection.

          4. Patients with Ewing sarcoma, rhabdomyosarcoma (except for pleomorphic/anaplastic
             rhabdomyosarcoma), or gastrointestinal stromal tumors. Patients with
             pleomorphic/anaplastic rhabdomyosarcoma are eligible.

          5. Patients previously treated with KLH or ganglioside containing vaccines or monoclonal
             antibodies (mAbs) against gangliosides.

          6. Females of childbearing potential that are pregnant or intend to become pregnant or
             who are breastfeeding. Females must have negative βHCG test within two weeks of first
             vaccination.

          7. Current active malignancy or history of malignancy, other than sarcoma, within the
             past two years, except for cervical carcinoma in situ or superficial skin cancer that
             has been surgically removed.

          8. Any medical condition that may limit the ability of the patient to complete the full
             course of treatment or to respond immunologically to vaccination, (including
             autoimmune or neurodegenerative disorders such as multiple sclerosis and amyotrophic
             lateral sclerosis).

          9. Patients requiring continuous doses of anti-inflammatory medications (steroids
             including inhaled steroids, non-steroidal anti-inflammatory drugs, or full dose
             aspirin). Episodic use of steroids or non-steroidal anti-inflammatory drugs permitted
             as long as they are not given within one week prior to or following vaccine
             administration. Continuous dosing of low-dose aspirin (≤ 81 mg/day) is acceptable.

         10. Use of or treatment with a drug that has not received regulatory approval or
             participation in a drug or device study during the 28 days preceding the first
             vaccination.

         11. Known history of HIV-positivity OR serologic evidence of HIV at screening or any
             immunodeficiency disorders or illnesses. Serologic positivity for the Hepatitis B
             Virus (HBV) or the Hepatitis C Virus (HCV), unless explained by a documented
             vaccination.

         12. Inability or unwillingness to meet the attendance requirements of the study.

         13. Any clinically significant abnormal finding at Screening (as determined by the
             principal investigator, in consultation with the Medical Monitor and the Sponsor),
             that would interfere with study participation, that would interfere with the
             evaluation or quality of the data, or that would put the patient at increased risk of
             illness or injury.