NeuroblastomaPhase 1CYCHE2NCT00048386
Neuroblastoma Vaccine for Treatment of High-Risk Neuroblastoma After Chemotherapy
A Phase 1 study testing autologous neuroblastoma vaccine in people with Neuroblastoma, run by Malcolm Brenner at 1 site in United States.
Trial summary
PRIMARY OBJECTIVE To determine the percentage of patients with high risk neuroblastoma in first or subsequent partial response or better, or with microscopic residual bone marrow disease, who demonstrate an immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with… Read more about this study
- Who is it for?
- Ages Up to 64 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- autologous neuroblastoma vaccine
- Type of treatment
- Vaccines, Injection or infusion
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 1999to Oct 2009
- 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 Up to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with high risk neuroblastoma defined below, who, following completion of front-line or salvage chemotherapy that may or may not have included high-dose chemotherapy followed by peripheral blood stem cell or bone marrow rescue with or without cis-retinoic acid, have achieved partial response or better, or who have microscopic residual bone marrow:
- INSS Stage 4 neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
- INSS Stage 3, N-myc amplified neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
- INSS Stage 3, N-myc non-amplified, Shimada unfavorable histology neuroblastoma, diagnosed between 1 and 21 years of age (inclusive)
- INSS Stages 2A or 2B, N-myc amplified, Shimada unfavorable histology, diagnosed between 1 and 21 years of age (inclusive)
- INSS Stage 4 neuroblastoma, with Shimada unfavorable histology, diagnosed under 365 days of age
Show all 15Show fewer
- Patients with intermediate or low risk neuroblastoma, who have achieved a second or subsequent partial response or better following chemotherapy treatment of first or subsequent relapse
- Patients must have recovered from the toxic effects of prior chemotherapy prior to treatment on this study, and must have both an absolute lymphocyte count and an absolute neutrophil count greater than 500/mm3.
- Patients with disease status outlined in the first bullet who have been treated with allogeneic tumor vaccine are eligible for treatment with autologous tumor vaccine when that product becomes available
- Patients may not concurrently receive any investigational agents or other tumor vaccines.
- Patients must be HIV-negative.
- Female patients must not be pregnant or lactating.
- Patients must have autologous transduced neuroblastoma cells available that are demonstrably producing > 150 pg IL-2/10e6 cells/24 hours.
- Patients or legal guardians must sign an informed consent according to institutional guidelines.
- Patients who are sexually active must be willing to utilize one of the more effective birth control methods during the study and for 3 months after the study is concluded. The male partner should use a condom.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study of Gene Modified Autologous Neuroblastoma Vaccine for the Post-Chemotherapy Treatment of High-Risk Neuroblastoma
PRIMARY OBJECTIVE To determine the percentage of patients with high risk neuroblastoma in first or subsequent partial response or better, or with microscopic residual bone marrow disease, who demonstrate an immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with subcutaneous injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete interleukin-2 (IL-2) (autologous neuroblastoma vaccine)
SECONDARY OBJECTIVES 1. To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule 2. To obtain preliminary data on the effect of vaccine administration on progression-free survival from high-risk neuroblastoma
Read the detailed description
Tumor cells from subjects with high-risk neuroblastoma will be obtained at the time of presentation and initial diagnosis, during routine surveillance of bone marrow disease status, or at the time of surgical resection of disease during primary chemotherapy. The tumor cells will be irradiated to prevent the possibility of tumor growth. Autologous neuroblastoma tumor cells will be used to treat neuroblastoma following the completion of primary or salvage chemotherapy when peripheral blood lymphocyte counts have recovered to > 500 CD3+ cells/mm3. This preceding chemotherapy may or may not have included bone marrow or peripheral stem cell rescue. Therefore, following the achievement of best response with chemotherapy, vaccine will be administered for 6 months. Vaccine modified for secretion of IL-2 will be used.
Vaccine therapy will commence after complete recovery from the side effects of primary or salvage chemotherapy, as long as the subject has an absolute CD3+ lymphocyte count and an absolute neutrophil count greater than 500/mm3. A vaccine consisting of 0.3 x 10 8 cells/patient will be given per injection, based on data from the Phase I studies. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months. The immune effects of the vaccine, toxicity of treatment, and anti-tumor effects will be periodically assessed. Further evaluation will be done annually.
The first and second vaccine injection sites will be "punch biopsied" one week after the injection. Several x-rays and various types of scans will also be taken to assist with monitoring the status of the neuroblastoma. Complete details of the evaluations required by this study are included in.
Subjects may receive supportive care for any acute or chronic toxicity from the injections, including blood product support, antibiotics, and other appropriate intervention. Pneumocystis carinii prophylaxis initiated during chemotherapy may be continued during vaccine therapy for as long as deemed appropriate by the investigator. As well, subjects may receive concurrent therapy with cis-retinoic acid at the discretion of the treating physician.
An adenoviral vector is being used to transduce the cells ex-vivo. Subjects will not be treated with the viral vector.
- Conditions
- Neuroblastoma
- Being tested
- autologous neuroblastoma vaccine
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous neuroblastoma vaccineBiological
A vaccine dose of 0.3 x 108 IL-2 secreting cells/patient/injection will be used. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months.
What is being measured
The primary outcome is the main question the study is designed to answer.
Patients who demonstrate immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete IL-2
Measured: 12 months post injections
Secondary outcomes (2)
To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule
Measured: 15 years
To obtain preliminary data on progression-free survival from high-risk neuroblastoma following vaccine administration
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2002 (estimated)
- Study startNov 1999
- Primary completionDec 2004
- Study completionOct 2009
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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Malcolm Brenner
- Sponsor type
- University, hospital or other
- Collaborators
- Baylor College of Medicine, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Sponsor-investigator, Malcolm Brenner, Baylor College of Medicine
- Investigators
- Heidi V Russell, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00048386
- Sponsor's protocol ID
- H8354
- Other IDs
- HIGH RISK NEUROBLASTOMA
- Acronym
- CYCHE2
- Status last verified
- Jul 2012
- First submitted
- 30 Oct 2002
- Last updated
- 27 Jul 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Gene Modified Autologous Neuroblastoma Vaccine for the Post-Chemotherapy Treatment of High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT00048386
- Acronym / sponsor ID
- CYCHE2 / H8354
- Conditions
- Neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Malcolm BrennerUniversity, hospital or other
- Collaborators
- Baylor College of Medicine, Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Nov 1999
- Primary completion
- Dec 2004 (actual)
- Study completion
- Oct 2009 (actual)
- First posted
- 1 Nov 2002 (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.
PRIMARY OBJECTIVE To determine the percentage of patients with high risk neuroblastoma in first or subsequent partial response or better, or with microscopic residual bone marrow disease, who demonstrate an immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with subcutaneous injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete interleukin-2 (IL-2) (autologous neuroblastoma vaccine)
SECONDARY OBJECTIVES 1. To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule 2. To obtain preliminary data on the effect of vaccine administration on progression-free survival from high-risk neuroblastoma
Detailed description
Tumor cells from subjects with high-risk neuroblastoma will be obtained at the time of presentation and initial diagnosis, during routine surveillance of bone marrow disease status, or at the time of surgical resection of disease during primary chemotherapy. The tumor cells will be irradiated to prevent the possibility of tumor growth. Autologous neuroblastoma tumor cells will be used to treat neuroblastoma following the completion of primary or salvage chemotherapy when peripheral blood lymphocyte counts have recovered to > 500 CD3+ cells/mm3. This preceding chemotherapy may or may not have included bone marrow or peripheral stem cell rescue. Therefore, following the achievement of best response with chemotherapy, vaccine will be administered for 6 months. Vaccine modified for secretion of IL-2 will be used.
Vaccine therapy will commence after complete recovery from the side effects of primary or salvage chemotherapy, as long as the subject has an absolute CD3+ lymphocyte count and an absolute neutrophil count greater than 500/mm3. A vaccine consisting of 0.3 x 10 8 cells/patient will be given per injection, based on data from the Phase I studies. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months. The immune effects of the vaccine, toxicity of treatment, and anti-tumor effects will be periodically assessed. Further evaluation will be done annually.
The first and second vaccine injection sites will be "punch biopsied" one week after the injection. Several x-rays and various types of scans will also be taken to assist with monitoring the status of the neuroblastoma. Complete details of the evaluations required by this study are included in.
Subjects may receive supportive care for any acute or chronic toxicity from the injections, including blood product support, antibiotics, and other appropriate intervention. Pneumocystis carinii prophylaxis initiated during chemotherapy may be continued during vaccine therapy for as long as deemed appropriate by the investigator. As well, subjects may receive concurrent therapy with cis-retinoic acid at the discretion of the treating physician.
An adenoviral vector is being used to transduce the cells ex-vivo. Subjects will not be treated with the viral vector.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Patients with high risk neuroblastoma defined below, who, following completion of front-line or salvage chemotherapy that may or may not have included high-dose chemotherapy followed by peripheral blood stem cell or bone marrow rescue with or without cis-retinoic acid, have achieved partial response or better, or who have microscopic residual bone marrow:
- Patients with intermediate or low risk neuroblastoma, who have achieved a second or subsequent partial response or better following chemotherapy treatment of first or subsequent relapse
- Patients must have recovered from the toxic effects of prior chemotherapy prior to treatment on this study, and must have both an absolute lymphocyte count and an absolute neutrophil count greater than 500/mm3.
- Patients with disease status outlined in the first bullet who have been treated with allogeneic tumor vaccine are eligible for treatment with autologous tumor vaccine when that product becomes available
- Patients may not concurrently receive any investigational agents or other tumor vaccines.
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 |
|---|---|---|
| autologous neuroblastoma vaccineA vaccine dose of 0.3 x 108 IL-2 secreting cells/patient/injection will be used. Injections will be given twice monthly for two months, then monthly for four months, for a total of eight vaccine injections over six months. | Biological | – |
Primary outcome
Patients who demonstrate immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete IL-2
Time frame: 12 months post injections
2 secondary outcomes
To determine the toxicity of the autologous neuroblastoma vaccine given according to this schedule
Time frame: 15 years
To obtain preliminary data on progression-free survival from high-risk neuroblastoma following vaccine administration
Time frame: 15 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2012.
Related trials for Neuroblastoma
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
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.
Heidi V Russell, MD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing autologous neuroblastoma vaccine in people with Neuroblastoma, run by Malcolm Brenner at 1 site 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 15 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: finished, as of the sponsor's last update on 27 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Patients who demonstrate immunological anti-tumor response at any time during, and for up to 12 months from initiation of, treatment with injections of autologous neuroblastoma cells, genetically modified by adenoviral vectors to secrete IL-2 (time frame: 12 months post injections). 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 27 Jul 2012.