NeuroblastomaPhase 1NCT00944580
A Vaccine Study for High Risk Cancers
A Phase 1 study testing MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine in people with Neuroblastoma and Rhabdomyosarcoma, run by Penn State University.
Trial summary
The purpose of this study is to determine the safety and immunological effects of a vaccine for people diagnosed with high risk neuroblastoma, osteogenic sarcoma, and rhabdomyosarcoma. It is hypothesized that this vaccine could reduce the incidence of relapse. Read more about this study
- Who is it for?
- Ages 1 year to 70 years
- Conditions the study is for
- Neuroblastoma, Rhabdomyosarcoma, Osteogenic Sarcoma
- What is being tested?
- MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2009to Aug 2010
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 1 year to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient 1 to 70 years of age with neuroblastoma, rhabdomyosarcoma, or osteogenic sarcoma, who have one or more of the following high risk features:
- Neuroblastoma:
- Stage IV disease
- Stage III disease with n-myc amplification
- Osteogenic sarcoma:
- Presence of metastases
Show all 29Show fewer
- Elevated alkaline phosphatase or LDH at diagnosis
- Primary tumor affecting the axial skeleton
- Poor histopathological response after completion of pre-surgical chemotherapy (≥10% viable tumor)
- Rhabdomyosarcoma:
- Stage IV disease
- Alveolar histology
- Positive tumor margins, with lymph node positivity
- Patient meets all screening criteria and tumor is positive for NY-ESO-1, MAGE- A1, or MAGE-A3 by immunohistochemistry or RT-PCR.
- Patients who are between 3 months and 2 years following the completion of therapy, and have achieved at least a very good partial response to primary therapy.
- No chemotherapy is planned for one month following the last vaccination.
- Bilirubin <2 mg/dL, and SGOT/SGPT <2.5 x normal
- Creatinine clearance > 50ml/min as estimated by patient's serum creatinine, weight, and age
- Room air pulse oximetry >94%
- Patient is not pregnant
- Male and female sexually active patients of reproductive who wish to participate must agree to use acceptable contraception
- Patient is not moribund and has a projected life expectancy >6 months
- Lansky performance scale > 70, ECOG < 2 (Appendix I)
- Potential subjects will be tested for HIV 1 and 2 antibodies, HTLV 1/2 antibodies, and for HIV 1, hepatitis C and hepatitis B virus by NAT testing. - -Subjects testing positive for any of these pathogens will be ineligible for vaccine.
- White blood cells ≥ 2.5 K/µL, Hemoglobin ≥ 8 g/dL, Hematocrit > 25%, and Platelets ≥ 70 K/µL
- Patient does not have central nervous system involvement.
- Patient does not a have a history of autoimmune disease, specifically inflammatory bowel disease, systemic lupus erythematosis, or rheumatoid arthritis
- Patient is not receiving concurrent systemic steroid therapy
- Patient does not have a known systemic hypersensitivity to imiquimod or any vaccine component
Who cannot join
None listed.
About this study
Official title: A Phase 1 Study to Determine the Immunologic Effects of a MAGE- A1, MAGE- A3, NY-ESO-1 Vaccine in Patients With High Risk Neuroblastoma, Osteogenic Sarcoma, and Rhabdomyosarcoma
The purpose of this study is to determine the safety and immunological effects of a vaccine for people diagnosed with high risk neuroblastoma, osteogenic sarcoma, and rhabdomyosarcoma. It is hypothesized that this vaccine could reduce the incidence of relapse.
Read the detailed description
MAGE -A1, MAGE- A3, and NY-ESO-1 are antigens that can be found with significant frequency on neuroblastoma, rhabdomyosarcoma, and osteogenic sarcoma, three relatively common solid tumors that in some cases can be associated with a high risk for relapse. In this study each subject will be screened for the presence of these antigens, and an individualized vaccine will be developed and administered using the subject's own dendritic cells (DC).
This study consists of two phases: a screening phase and a treatment/vaccine phase. First, eligible individuals will be consented into the screening phase. Tumor specimens will be tested by immunohistochemistry or RT-PCR for the presence of MAGE- A1, MAGE- A3, and NY-ESO-1. Those testing positive for one or more antigen can be consented for the treatment phase of the study. Blood will be drawn for DC culture, and approximately one month later a series of three vaccines will be administered at two week intervals. Subjects will receive a topical medication called imiquimod to the vaccine site prior to and following each injection, to help immune cells travel into the area. Study participation occurs over 18 months and also involves periodic physical examinations and blood draws.
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteogenic Sarcoma
- Being tested
- MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine
- Keywords
- MAGE-A1, MAGE-A3, NY-ESO-1, antigen, vaccine, dendritic cells, immunohistochemistry, immunology
- Medical subject headings
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MAGE-A1, MAGE-A3, and NY-ESO-1 VaccineBiological
A regimen of three vaccines every two weeks. Each vaccine will contain 3,000,000-5,000,000 peptide pulsed dendritic cells. Imiquimod, a topical cream, will be applied to the vaccination site before and after each vaccination.
| Study group | Role | What they receive |
|---|---|---|
| Vaccine Intervention | Experimental | MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine: A regimen of three vaccines every two weeks. Each vaccine will contain 3,000,000-5,000,000 peptide pulsed dendritic cells. Imiquimod, a topical cream, will be applied to the vaccination site before and after each vaccination. Receives: Biological: MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary objective is to determine if there is an amplification or new development of NY-ESO-1, MAGE-A1, or MAGE-A3 specific CD4+ or CD8+ T cells post-vaccination.
Measured: 2 years
Secondary outcomes (1)
The investigators will determine the safety of vaccine and imiquimod administration in these patients.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2009 (estimated)
- Study startJun 2009
- Primary completionAug 2010
- Study completionAug 2010
Why it stopped: unexpectedly low screening results leading to poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Penn State University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kenneth G. Lucas, MD, Study chair, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00944580
- Sponsor's protocol ID
- IRB 30761
- Other IDs
- PSHCI 09-033
- Status last verified
- Nov 2017
- First submitted
- 21 Jul 2009
- Last updated
- 24 Nov 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Determine the Immunologic Effects of a MAGE- A1, MAGE- A3, NY-ESO-1 Vaccine in Patients With High Risk Neuroblastoma, Osteogenic Sarcoma, and Rhabdomyosarcoma
- ClinicalTrials.gov ID
- NCT00944580
- Acronym / sponsor ID
- IRB 30761
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteogenic Sarcoma
- Keywords
- MAGE-A1, MAGE-A3, NY-ESO-1, antigen, vaccine, dendritic cells, immunohistochemistry, immunology
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: unexpectedly low screening results leading to poor accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Penn State UniversityUniversity, hospital or other
- Study start
- Jun 2009
- Primary completion
- Aug 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 23 Jul 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety and immunological effects of a vaccine for people diagnosed with high risk neuroblastoma, osteogenic sarcoma, and rhabdomyosarcoma. It is hypothesized that this vaccine could reduce the incidence of relapse.
Detailed description
MAGE -A1, MAGE- A3, and NY-ESO-1 are antigens that can be found with significant frequency on neuroblastoma, rhabdomyosarcoma, and osteogenic sarcoma, three relatively common solid tumors that in some cases can be associated with a high risk for relapse. In this study each subject will be screened for the presence of these antigens, and an individualized vaccine will be developed and administered using the subject's own dendritic cells (DC).
This study consists of two phases: a screening phase and a treatment/vaccine phase. First, eligible individuals will be consented into the screening phase. Tumor specimens will be tested by immunohistochemistry or RT-PCR for the presence of MAGE- A1, MAGE- A3, and NY-ESO-1. Those testing positive for one or more antigen can be consented for the treatment phase of the study. Blood will be drawn for DC culture, and approximately one month later a series of three vaccines will be administered at two week intervals. Subjects will receive a topical medication called imiquimod to the vaccine site prior to and following each injection, to help immune cells travel into the area. Study participation occurs over 18 months and also involves periodic physical examinations and blood draws.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vaccine Intervention | Experimental | Biological: MAGE-A1, MAGE-A3, and NY-ESO-1 VaccineMAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine: A regimen of three vaccines every two weeks. Each vaccine will contain 3,000,000-5,000,000 peptide pulsed dendritic cells. Imiquimod, a topical cream, will be applied to the vaccination site before and after each vaccination. |
Who may qualify
- Ages
- 1 year to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (29 listed)
- Patient 1 to 70 years of age with neuroblastoma, rhabdomyosarcoma, or osteogenic sarcoma, who have one or more of the following high risk features:
- Neuroblastoma:
- Osteogenic sarcoma:
- Rhabdomyosarcoma:
- Patient meets all screening criteria and tumor is positive for NY-ESO-1, MAGE- A1, or MAGE-A3 by immunohistochemistry or RT-PCR.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MAGE-A1, MAGE-A3, and NY-ESO-1 VaccineA regimen of three vaccines every two weeks. Each vaccine will contain 3,000,000-5,000,000 peptide pulsed dendritic cells. Imiquimod, a topical cream, will be applied to the vaccination site before and after each vaccination. | Biological | Vaccine Intervention |
Primary outcome
The primary objective is to determine if there is an amplification or new development of NY-ESO-1, MAGE-A1, or MAGE-A3 specific CD4+ or CD8+ T cells post-vaccination.
Time frame: 2 years
1 secondary outcome
The investigators will determine the safety of vaccine and imiquimod administration in these patients.
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth G. Lucas, MD study chair
Milton S. Hershey Medical Center
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MAGE-A1, MAGE-A3, and NY-ESO-1 Vaccine in people with Neuroblastoma and Rhabdomyosarcoma, run by Penn State University.
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 29 inclusion and 0 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: withdrawn, as of the sponsor's last update on 24 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
The primary objective is to determine if there is an amplification or new development of NY-ESO-1, MAGE-A1, or MAGE-A3 specific CD4+ or CD8+ T cells post-vaccination. (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Nov 2017.