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SarcomaPhase 2NCT01141491

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

A Phase 2 study testing Trivalent ganglioside vaccine and OPT-821 in people with Sarcoma, run by MabVax Therapeutics, Inc. at 13 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 16 years and older
Conditions the study is for
Sarcoma
What is being tested?
Trivalent ganglioside vaccine and OPT-821
Could I receive a placebo?
No, everyone receives an active treatment
How many people
136 participants
Where
13 sitesUnited States
Study dates
Jun 2010to 10 Mar 2017
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 16 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

United States13 sites

  • University of Michigan Hospital

    Ann Arbor, Michigan, 48109

  • Winship Cancer Institute at Emory Midtown

    Atlanta, Georgia, 30308

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Northwestern University - Robert H. Lurie Comprehensive Cancer Center

    Chicago, Illinois, 60611

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • University of Colorado (Denver)

    Denver, Colorado, 80045

  • University of Iowa Hospitals and Clinic

    Iowa City, Iowa, 52242

  • UCLA Medical Center

    Los Angeles, California, 90025

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10065

  • University of Pittsburg Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Sciences University

    Portland, Oregon, 97239

  • Fred Hutchinson Cancer Research Center/Seattle Cancer Care

    Seattle, Washington, 98109

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: A Randomized, Multicenter, Double-Blind, Phase II Trial of a KLH Conjugated Trivalent Ganglioside Vaccine Containing GM2, GD2 Lactone, and GD3 Lactone With the Immunological Adjuvant OPT-821 Versus OPT-821 Alone in Metastatic Sarcoma Patients Who Are Rendered Disease Free

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.

Read the detailed description

This study is a Phase II randomized, double-blind, multi-center study of a trivalent ganglioside vaccine plus the immunological adjuvant OPT-821 (Arm A) versus OPT-821 alone (Arm B) for patients with metastatic sarcoma at initial presentation or with relapsed disease who have been rendered disease-free following either surgical resection or multi-modality therapy. The primary aim of this study is to demonstrate the efficacy of vaccine therapy over non-specific immune therapy. Another aim of this study is to obtain sufficient data to further the development of this specific vaccine therapy as well as guide future study designs for therapeutic cancer vaccines in general.

To be eligible, patients must have histologically confirmed sarcoma, must be clinically free of disease after surgery or multimodality therapy, and must be within 8 weeks of completion of such therapy. Given the limited data regarding ganglioside expression in Ewing sarcoma, rhabdomyosarcoma, and gastrointestinal stromal tumors, patients with these sarcoma subtypes with the exception of pleomorphic/anaplastic rhabdomyosarcoma will be excluded. Patients must have a history of distant metastatic disease; patients with locally recurrent disease only will not be eligible, as these patients demonstrate a different natural history from those with metastatic disease.

All treatment will be performed in the outpatient setting. Patients will be randomized in a 1:1 ratio to receive a total of 10 treatments of either the vaccine plus OPT-821 (Arm A) or OPT-821 alone (Arm B). Treatment will be administered on Visit Weeks 1, 2, 3, 8, 16, 28, 40, 52, 68, and 84. All patients will receive 150 mcg of OPT-821.

Conditions
Sarcoma
Being tested
Trivalent ganglioside vaccine, OPT-821
Keywords
Sarcoma, Metastatic, Vaccine, Adjuvant
Medical subject headings
Sarcoma, Neoplasm Metastasis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Trivalent ganglioside vaccineBiological

    Also called Trivalent ganglioside vaccine plus adjuvant OPT-821

    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

  • OPT-821Biological

    Also called Immunologic adjuvant OPT-821

    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

Study groups in NCT01141491
Study groupRoleWhat they receive
Arm AExperimentalVaccine plus OPT-821
Receives: Biological: Trivalent ganglioside vaccine
Arm B - OPT-821 immunologic adjuvantActive comparatorPatients 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
Receives: Biological: OPT-821

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 3-years

Secondary outcomes (1)

  1. Overall Survival

    To compare the overall survival over time, to estimate the median and 3-year progression-free survival.

    Measured: Measured over time

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jun 2010 (estimated)
  2. Study startJun 2010
  3. Primary completionSep 2013
  4. Study completion10 Mar 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
136 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01141491
Sponsor's protocol ID
MV-0109DP001
Status last verified
Dec 2016
First submitted
9 Jun 2010
Last updated
12 Apr 2017
Data monitoring committee
No

Publications

  • 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.

    PubMed 36215947

  • 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.

    PubMed 33326254

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Trivalent ganglioside vaccine and OPT-821 in people with Sarcoma, run by MabVax Therapeutics, Inc. at 13 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Dec 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (time frame: 3-years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01141491, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2017.

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