NeuroblastomaPhase 1NCT02745756
A Combined Cell Therapy Approach to the Treatment of Neuroblastoma
A Phase 1 study testing Autologous Hematopoietic Progenitor Cell Transplant and KLH and Tumor Lysate Pulsed DC Vaccine in people with Neuroblastoma and Neoplasms, Nerve Tissue, run by H. Lee Moffitt Cancer Center and Research Institute.
Trial summary
This study adds an experimental treatment with another type of cells, called dendritic cells. It is hoped that these cells may stimulate the immune system to react against neuroblastoma in much the same way that vaccines cause the immune system to react to certain viruses and bacteria. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma, Neoplasms, Nerve Tissue
- What is being tested?
- Autologous Hematopoietic Progenitor Cell Transplant and KLH and Tumor Lysate Pulsed DC Vaccine
- Type of treatment
- Vaccines, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 14 Apr 2016to 21 Aug 2017
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Must have a histological diagnosis of neuroblastoma or ganglioneuroblastoma and be either newly diagnosed with high risk disease or have failed previous treatment: Patients who have failed previous treatment may have had no more than one earlier autologous HPC transplant.
- Participant is expected to undergo autologous HPC transplantation that is consistent with standard of care.
- Must have the presence of residual resectable disease for which surgery is clinically indicated, and will be performed at Johns Hopkins All Children's Hospital.
Who cannot join
- Not an eligible candidate for collection by apheresis or HPC transplant.
- History of autoimmune disorder or immune deficiency disorder.
About this study
This study adds an experimental treatment with another type of cells, called dendritic cells. It is hoped that these cells may stimulate the immune system to react against neuroblastoma in much the same way that vaccines cause the immune system to react to certain viruses and bacteria. The physicians conducting this study have observed from previous research that neuroblastoma cells can be recognized by the immune system, and that they can be destroyed by immune cells.The main goal of this study is to see if giving participants this additional anti-Neuroblastoma vaccine reduces the risk of relapse following the Hematopoietic Stem Cell Transplant.
Read the detailed description
All patients who are enrolled in this study will receive all treatment at All Children's Hospital which is located at 501 6th Ave South, St. Petersburg, FL 33701. This includes autologous cell donation by apheresis, high dose cytotoxic therapy conditioning for autologous HPC transplant, post-transplant follow-up care, and all administration of dendritic cell vaccines and blood draws for post therapy immunological monitoring.
All preparation of cellular products, including hematopoietic progenitor cell products for autologous transplantation, and dendritic cell vaccine products, will be carried out in the Cell Therapy Facility located within the Moffitt Cancer Center, which is located at 12902 Magnolia Drive, Tampa, FL 33612.
- Conditions
- Neuroblastoma, Neoplasms, Nerve Tissue
- Being tested
- Autologous Hematopoietic Progenitor Cell Transplant, KLH and Tumor Lysate Pulsed DC Vaccine
- Keywords
- Children, Cancer, High-risk neuroblastoma
- Medical subject headings
- Neuroblastoma, Neoplasms, Nerve Tissue, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Progenitor Cell TransplantProcedure or surgery
Also called stem cell transplant
Autologous Hematopoietic Progenitor Cell (HPC) Transplant (HPCT). Blood stem cells will be collected by apheresis during the induction phase as part of standard treatment. During apheresis, the participant's blood is collected into a machine that filters out the stem cells and the filtered blood is returned to their body. The stem cells will be separated by the type of protein within the cells. Only the stem cells with a protein called CD34 will be used for the stem cell transplant.
KLH and Tumor Lysate Pulsed DC VaccineBiological
Also called Dendritic Cell (DC) Vaccine
Keyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine. Post-transplant vaccine days: +14, +28, +56 (± 10 days), +84 (± 10 days). Vaccines will be administered by intradermal injection of 0.5 mL at two nodal basins.
| Study group | Role | What they receive |
|---|---|---|
| Combined Cell Therapy | Experimental | Hematopoietic progenitor cell (HPC) transplant (HPCT) with autologous tumor cell lysate and keyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine. Receives: Procedure: Autologous Hematopoietic Progenitor Cell Transplant; Biological: KLH and Tumor Lysate Pulsed DC Vaccine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Sufficient Tumor Cell Lysate and Dendritic Cells
The feasibility of manufacturing both a hematopoietic progenitor cell graft and multiple tumor lysate pulsed dendritic cell vaccine treatments from the same starting apheresis product, culminating in delivery of the vaccines in the immediate period following myeloablative therapy and autologous hematopoietic progenitor cell transplant period (autoHPCT). For what fraction of eligible patients can sufficient tumor cell lysate and dendritic cells, necessary for the production of the dendritic cell vaccines, be obtained? From what fraction would it be possible to make additional vaccines?
Measured: Up to 1 year
Secondary outcomes (1)
Occurrence of Dendritic Cell Related Adverse Events
Toxicities resulting from the administration of dendritic cell vaccines in the immediate post hematopoietic cell graft period.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2016 (estimated)
- Study start14 Apr 2016
- Primary completion21 Aug 2017
- Study completion21 Aug 2017
Why it stopped: no enrollment
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.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- 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
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins All Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Shari Pilon-Thomas, Ph.D., Principal investigator, H. Lee Moffitt Cancer Center and Research InstituteGregory Hale, M.D., Principal investigator, Johns Hopkins All Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02745756
- Sponsor's protocol ID
- MCC-17181
- Status last verified
- Aug 2017
- First submitted
- 18 Apr 2016
- Last updated
- 30 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Combined Cell Therapy Approach to the Treatment of Neuroblastoma
- ClinicalTrials.gov ID
- NCT02745756
- Acronym / sponsor ID
- MCC-17181
- Conditions
- Neuroblastoma, Neoplasms, Nerve Tissue
- Keywords
- Children, Cancer, High-risk neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no enrollment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Johns Hopkins All Children's Hospital
- Study start
- 14 Apr 2016 (actual)
- Primary completion
- 21 Aug 2017 (actual)
- Study completion
- 21 Aug 2017 (actual)
- First posted
- 20 Apr 2016 (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.
This study adds an experimental treatment with another type of cells, called dendritic cells. It is hoped that these cells may stimulate the immune system to react against neuroblastoma in much the same way that vaccines cause the immune system to react to certain viruses and bacteria. The physicians conducting this study have observed from previous research that neuroblastoma cells can be recognized by the immune system, and that they can be destroyed by immune cells.The main goal of this study is to see if giving participants this additional anti-Neuroblastoma vaccine reduces the risk of relapse following the Hematopoietic Stem Cell Transplant.
Detailed description
All patients who are enrolled in this study will receive all treatment at All Children's Hospital which is located at 501 6th Ave South, St. Petersburg, FL 33701. This includes autologous cell donation by apheresis, high dose cytotoxic therapy conditioning for autologous HPC transplant, post-transplant follow-up care, and all administration of dendritic cell vaccines and blood draws for post therapy immunological monitoring.
All preparation of cellular products, including hematopoietic progenitor cell products for autologous transplantation, and dendritic cell vaccine products, will be carried out in the Cell Therapy Facility located within the Moffitt Cancer Center, which is located at 12902 Magnolia Drive, Tampa, FL 33612.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combined Cell Therapy | Experimental | Procedure: Autologous Hematopoietic Progenitor Cell Transplant, Biological: KLH and Tumor Lysate Pulsed DC VaccineHematopoietic progenitor cell (HPC) transplant (HPCT) with autologous tumor cell lysate and keyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Must have a histological diagnosis of neuroblastoma or ganglioneuroblastoma and be either newly diagnosed with high risk disease or have failed previous treatment: Patients who have failed previous treatment may have had no more than one earlier autologous HPC transplant.
- Participant is expected to undergo autologous HPC transplantation that is consistent with standard of care.
- Must have the presence of residual resectable disease for which surgery is clinically indicated, and will be performed at Johns Hopkins All Children's Hospital.
Key exclusion criteria (2 listed)
- Not an eligible candidate for collection by apheresis or HPC transplant.
- History of autoimmune disorder or immune deficiency disorder.
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 Hematopoietic Progenitor Cell TransplantAlso: stem cell transplantAutologous Hematopoietic Progenitor Cell (HPC) Transplant (HPCT). Blood stem cells will be collected by apheresis during the induction phase as part of standard treatment. During apheresis, the participant's blood is collected into a machine that filters out the stem cells and the filtered blood is returned to their body. The stem cells will be separated by the type of protein within the cells. Only the stem cells with a protein called CD34 will be used for the stem cell transplant. | Procedure or surgery | Combined Cell Therapy |
| KLH and Tumor Lysate Pulsed DC VaccineAlso: Dendritic Cell (DC) VaccineKeyhole limpet hemocyanin (KLH) pulsed dendritic cell (DC) vaccine. Post-transplant vaccine days: +14, +28, +56 (± 10 days), +84 (± 10 days). Vaccines will be administered by intradermal injection of 0.5 mL at two nodal basins. | Biological | Combined Cell Therapy |
Primary outcome
Occurrence of Sufficient Tumor Cell Lysate and Dendritic Cells
Time frame: Up to 1 year
The feasibility of manufacturing both a hematopoietic progenitor cell graft and multiple tumor lysate pulsed dendritic cell vaccine treatments from the same starting apheresis product, culminating in delivery of the vaccines in the immediate period following myeloablative therapy and autologous hematopoietic progenitor cell transplant period (autoHPCT). For what fraction of eligible patients can sufficient tumor cell lysate and dendritic cells, necessary for the production of the dendritic cell vaccines, be obtained? From what fraction would it be possible to make additional vaccines?
1 secondary outcome
Occurrence of Dendritic Cell Related Adverse Events
Time frame: Up to 1 year
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 30 Aug 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.
Shari Pilon-Thomas, Ph.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
Gregory Hale, M.D. principal investigator
Johns Hopkins All Children's Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Hematopoietic Progenitor Cell Transplant and KLH and Tumor Lysate Pulsed DC Vaccine in people with Neuroblastoma and Neoplasms, Nerve Tissue, run by H. Lee Moffitt Cancer Center and Research Institute.
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 3 inclusion and 2 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 30 Aug 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?
Occurrence of Sufficient Tumor Cell Lysate and Dendritic Cells (time frame: Up to 1 year). 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 30 Aug 2017.