Trivalent Ganglioside Vaccine With Immunological Adjuvant or Immunological Adjuvant Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free

NCT01141491 | Completed Phase 2 Results

• 1 other identifier: MV-0109DP001
• Study Type: interventional
• Target: 136
• Locations: 1 country
• Sites: 13

Brief 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.

Conditions

Sarcoma

Keywords

Sarcoma; Metastatic; Vaccine; Adjuvant

Outcome Measures

Primary Outcomes (1)

• Progression Free Survival

  The primary objective is to compare the progression-free survival (PFS) over time. Progression free survival is defined as the time from randomization until any evidence of tumor growth or appearance anywhere in the body or death from any cause as determined by the principal investigator at each site. The principal investigator will determine Progression-free survival by using CT scans to evaluate disease recurrence. For the purpose of this study, progression of disease is defined as the development of tumor growth or recurrence at any site of the body as determined by the principal investigator at each study site or death from disease

  3-years

Secondary Outcomes (1)

• Overall Survival

  Measured over time

Study Arms (2)

Arm A

EXPERIMENTAL

Vaccine plus OPT-821

Biological: Trivalent ganglioside vaccine

Arm B - OPT-821 immunologic adjuvant

ACTIVE COMPARATOR

Patients will be given 10 injections of OPT-821 alone 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

Biological: OPT-821

Interventions

Trivalent ganglioside vaccine BIOLOGICAL

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

Also known as: Trivalent ganglioside vaccine plus adjuvant OPT-821

Arm A

OPT-821 BIOLOGICAL

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

Also known as: Immunologic adjuvant OPT-821

Arm B - OPT-821 immunologic adjuvant

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, 16 years or older.
• 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.
• Histological confirmation of sarcoma, as performed by a pathologist at one of the participating study sites, prior to entry on study.
• Patients must have undergone surgical metastectomy within 8 weeks prior to initiation of treatment on this study.
• 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.
• 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.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 1.
• Weight ≥ 40 kg.
• 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
• Current use of an acceptable form of birth control.
• 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.

You may not qualify if:

• Patients with evidence of local or metastatic disease or who are not disease free at the time of the first vaccination.
• 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.
• Patients with brain or bone metastasis even if they are able to undergo complete surgical resection.
• Patients with Ewing sarcoma, rhabdomyosarcoma (except for pleomorphic/anaplastic rhabdomyosarcoma), or gastrointestinal stromal tumors. Patients with pleomorphic/anaplastic rhabdomyosarcoma are eligible.
• Patients previously treated with KLH or ganglioside containing vaccines or monoclonal antibodies (mAbs) against gangliosides.
• 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.
• 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.
• 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).
• 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.
• 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.
• 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.
• Inability or unwillingness to meet the attendance requirements of the study.
• 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.

Sponsors & Collaborators

• MabVax Therapeutics, Inc.: lead

Study Sites (13)

UCLA Medical Center

Los Angeles, California, 90025, United States

Location

University of Colorado (Denver)

Denver, Colorado, 80045, United States

Location

Winship Cancer Institute at Emory Midtown

Atlanta, Georgia, 30308, United States

Location

Northwestern University - Robert H. Lurie Comprehensive Cancer Center

Chicago, Illinois, 60611, United States

Location

University of Iowa Hospitals and Clinic

Iowa City, Iowa, 52242, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

University of Michigan Hospital

Ann Arbor, Michigan, 48109, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Sciences University

Portland, Oregon, 97239, United States

Location

University of Pittsburg Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

Location

Fred Hutchinson Cancer Research Center/Seattle Cancer Care

Seattle, Washington, 98109, United States

Location

Related Publications (2)

• Rosenbaum E, Chugh R, Ryan CW, Agulnik M, Milhem MM, George S, Jones RL, Chmielowski B, Van Tine BA, Tawbi H, Elias AD, Read WL, Budd GT, Qin LX, Rodler ET, Hirman J, Weiden P, Bennett CM, Livingston PO, Ragupathi G, Hansen D, D'Angelo SP, Tap WD, Schwartz GK, Maki RG, Carvajal RD. A randomised phase II trial of a trivalent ganglioside vaccine targeting GM2, GD2 and GD3 combined with immunological adjuvant OPT-821 versus OPT-821 alone in metastatic sarcoma patients rendered disease-free by surgery. Eur J Cancer. 2022 Nov;176:155-163. doi: 10.1016/j.ejca.2022.09.003. Epub 2022 Oct 8.

  PMID: 36215947 DERIVED

• Cheung IY, Cheung NV, Modak S, Mauguen A, Feng Y, Basu E, Roberts SS, Ragupathi G, Kushner BH. Survival Impact of Anti-GD2 Antibody Response in a Phase II Ganglioside Vaccine Trial Among Patients With High-Risk Neuroblastoma With Prior Disease Progression. J Clin Oncol. 2021 Jan 20;39(3):215-226. doi: 10.1200/JCO.20.01892. Epub 2020 Dec 16.

  PMID: 33326254 DERIVED

Related Links

• NCI sarcoma information
• Related Info

MeSH Terms

Conditions

Sarcoma; Neoplasm Metastasis

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Dave Hansen
• Organization: MabVax Therapeutics, Inc.

dhansen@mabvax.com

858.259.9405

Study Officials

• William Tap, M.D.

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2010
• First Posted: June 10, 2010
• Study Start: June 1, 2010
• Primary Completion: September 1, 2013
• Study Completion: March 10, 2017
• Last Updated: April 12, 2017
• Results First Posted: December 7, 2016

Record last verified: 2016-12

Locations

US (13)