NeuroblastomaPhase 1STALLONCT01462396
Allogeneic Stem Cell Transplantation for Advanced Neuroblastoma Using MHC Mismatched Related Donors
A Phase 1 study testing CD34+ cells selected with the Miltenyi Clinimacs machine in people with Neuroblastoma, run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial summary
Allogeneic stem cell transplantation has been explored for patients with high risk neuroblastoma. Results have been mixed, with only small series and case reports. Recent reports, however, especially with haploidentical transplantation have been more encouraging. Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- CD34+ cells selected with the Miltenyi Clinimacs machine
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2011to Dec 2015
- 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 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 6 months - <18 years
- Measurable tumor by routine imaging or bone marrow biopsy
- Patient must have an 3/6, 4/6, or 5/6 human leukocyte antigen (HLA)-mismatched related donor who is Epstein-Barr virus (EBV) seropositive
- Karnofsky score 60% or greater if 10yrs old or older, Lansky score 60% or greater if under 10yrs old
- Pulse ox >90% on room air
- Recovered from toxic effects of prior chemotherapy
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- Patient must not be pregnant
- Patient must be HIV negative
- Patient or responsible person must be able to understand and sign an informed consent
- Available donor without contraindication for stem cell collection
Who cannot join
- Pregnant and lactating women.
- Human immunodeficiency virus (HIV) positive patient.
- Uncontrolled intercurrent infection.
- Renal failure (Creatine > 1.5 or Creatinine Clearance < 40 ml/min/1.73m2)
- Active hepatitis or cirrhosis with liver test values greater than 3 times normal
- NOTE: Patients who would be excluded from the protocol strictly for laboratory abnormalities can be included at the investigator's discretion, after review by the Children's Mercy Hospital ethics board
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- Donor Inclusion/Exclusion Criteria
- Donor must be in good health based on review of systems and results of physical examination, and routine testing per standards of good medical care.
- Female donors of childbearing age must have a negative pregnancy test and must not be lactating
- EBv seropositive
- Donor stem cells should be human leukocyte antigen (HLA) typed using molecular methods. See section 6.1.3 for HLA matching requirements.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
Official title: Phase I Study of Allogeneic Stem Cell Transplantation for Advanced Neuroblastoma Using Major Histocompatibility Complex (MHC) Mismatched Related Donors and Sub-Myeloablative Regimen
Allogeneic stem cell transplantation has been explored for patients with high risk neuroblastoma. Results have been mixed, with only small series and case reports. Recent reports, however, especially with haploidentical transplantation have been more encouraging. Eradication of neuroblastoma may be mediated by both components of the innate immune system (natural killer cells) and through the adaptive immune system via T-cell cytotoxicity and the development of a humoral response to tumor specific antigens and minor histocompatibility antigens. To overcome restrictions created by unavailability of Human leukocyte antigen (HLA) matched donors, stem cell grafts from haploidentical related donors have been explored. Historically, the use of full haplotype mismatched family member donors has been limited by the development of severe graft-versus-host disease and the high rate of graft failure. Graft failure can now be overcome by increasing immunosuppression and increasing the number of transplanted stem cells. The most effective means of graft versus host disease (GVHD) prophylaxis is T cell depletion of the donor marrow. A 3-4 log depletion will reduce the risk of developing significant GVHD to less than 10%. Methods to mobilize stem cells from the bone marrow into the peripheral blood and collect these stem cells by apheresis now increase the availability of stem cells by a magnitude. Selection devices have been developed that will prepare extremely pure populations of these CD34 cells with upwards of 5 logs depletion of contaminating T cells. The CliniMACS CD34 Reagent System is a medical device designed to select CD34+ hematopoietic cells from heterogeneous hematologic cell populations. The investigators intend to provide mismatched related hematopoietic stem cell transplantation to up to 10 patients with relapsed refractory neuroblastoma. Harnessing the potential for innate and adaptive immune responses through allogeneic Hematopoietic stem cell transplantation (HSCT) may provide cure for some patients with this tumor.
- Conditions
- Neuroblastoma
- Being tested
- CD34+ cells selected with the Miltenyi Clinimacs machine
- Keywords
- Neuroblastoma, Relapsed, Refractory
- Medical subject headings
- Neuroblastoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD34+ cells selected with the Miltenyi Clinimacs machineDevice
Haploidentical allogeneic stem cell transplant following sub-myeloablative conditioning and cell selection using the Miltenyi Clinimacs device
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Haploidentical allogeneic stem cell transplant following sub-myeloablative conditioning and cell selection using the Miltenyi Clinimacs device Receives: Device: CD34+ cells selected with the Miltenyi Clinimacs machine |
What is being measured
The primary outcome is the main question the study is designed to answer.
The immediate safety of a fludarabine based reduced intensity conditioning regimen and CD34+ stem cell selected mis-matched, related, allogeneic transplant will be assess in patients with relapsed/refractory neuroblastoma
Monitoring of mortality, toxicity (NCI Common Criteria), acute and chronic graft versus host disease, engraftment rate will contribute to safety assessment
Measured: 6 weeks
Secondary outcomes (1)
Infusional and long term safety and persistence of tumor redirected, genetically modified, donor derived, allogeneic multi-virus specific cytotoxic T-cells (tV-CTL) after allogeneic hematopoietic stem cell transplant in patients with neuroblastoma
Tumor evaluation will occur 4-8 weeks after transplant
Measured: 4-8 weeks post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2011 (estimated)
- Study startOct 2011
- Primary completionDec 2015
- Study completionDec 2015
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
- Device feasibility
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Collaborators
- University of Kansas
- Responsible party
- Principal investigator, Doug Myers, Children's Mercy Hospital Kansas City
- Investigators
- Gary D Myers, MD, Principal investigator, Children's Mercy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01462396
- Sponsor's protocol ID
- STALLO
- Acronym
- STALLO
- Status last verified
- Jul 2018
- First submitted
- 25 Oct 2011
- Last updated
- 18 Jul 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Allogeneic Stem Cell Transplantation for Advanced Neuroblastoma Using Major Histocompatibility Complex (MHC) Mismatched Related Donors and Sub-Myeloablative Regimen
- ClinicalTrials.gov ID
- NCT01462396
- Acronym / sponsor ID
- STALLO / STALLO
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma, Relapsed, Refractory
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Collaborators
- University of Kansas
- Study start
- Oct 2011
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 31 Oct 2011 (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.
Allogeneic stem cell transplantation has been explored for patients with high risk neuroblastoma. Results have been mixed, with only small series and case reports. Recent reports, however, especially with haploidentical transplantation have been more encouraging. Eradication of neuroblastoma may be mediated by both components of the innate immune system (natural killer cells) and through the adaptive immune system via T-cell cytotoxicity and the development of a humoral response to tumor specific antigens and minor histocompatibility antigens. To overcome restrictions created by unavailability of Human leukocyte antigen (HLA) matched donors, stem cell grafts from haploidentical related donors have been explored. Historically, the use of full haplotype mismatched family member donors has been limited by the development of severe graft-versus-host disease and the high rate of graft failure. Graft failure can now be overcome by increasing immunosuppression and increasing the number of transplanted stem cells. The most effective means of graft versus host disease (GVHD) prophylaxis is T cell depletion of the donor marrow. A 3-4 log depletion will reduce the risk of developing significant GVHD to less than 10%. Methods to mobilize stem cells from the bone marrow into the peripheral blood and collect these stem cells by apheresis now increase the availability of stem cells by a magnitude. Selection devices have been developed that will prepare extremely pure populations of these CD34 cells with upwards of 5 logs depletion of contaminating T cells. The CliniMACS CD34 Reagent System is a medical device designed to select CD34+ hematopoietic cells from heterogeneous hematologic cell populations. The investigators intend to provide mismatched related hematopoietic stem cell transplantation to up to 10 patients with relapsed refractory neuroblastoma. Harnessing the potential for innate and adaptive immune responses through allogeneic Hematopoietic stem cell transplantation (HSCT) may provide cure for some patients with this tumor.
Study design
- Primary purpose
- Device feasibility
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Device: CD34+ cells selected with the Miltenyi Clinimacs machineHaploidentical allogeneic stem cell transplant following sub-myeloablative conditioning and cell selection using the Miltenyi Clinimacs device |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Age 6 months - <18 years
- Measurable tumor by routine imaging or bone marrow biopsy
- Patient must have an 3/6, 4/6, or 5/6 human leukocyte antigen (HLA)-mismatched related donor who is Epstein-Barr virus (EBV) seropositive
- Karnofsky score 60% or greater if 10yrs old or older, Lansky score 60% or greater if under 10yrs old
- Pulse ox >90% on room air
Key exclusion criteria (11 listed)
- Pregnant and lactating women.
- Human immunodeficiency virus (HIV) positive patient.
- Uncontrolled intercurrent infection.
- Renal failure (Creatine > 1.5 or Creatinine Clearance < 40 ml/min/1.73m2)
- Active hepatitis or cirrhosis with liver test values greater than 3 times normal
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 |
|---|---|---|
| CD34+ cells selected with the Miltenyi Clinimacs machineHaploidentical allogeneic stem cell transplant following sub-myeloablative conditioning and cell selection using the Miltenyi Clinimacs device | Device | Single Arm |
Primary outcome
The immediate safety of a fludarabine based reduced intensity conditioning regimen and CD34+ stem cell selected mis-matched, related, allogeneic transplant will be assess in patients with relapsed/refractory neuroblastoma
Time frame: 6 weeks
Monitoring of mortality, toxicity (NCI Common Criteria), acute and chronic graft versus host disease, engraftment rate will contribute to safety assessment
1 secondary outcome
Infusional and long term safety and persistence of tumor redirected, genetically modified, donor derived, allogeneic multi-virus specific cytotoxic T-cells (tV-CTL) after allogeneic hematopoietic stem cell transplant in patients with neuroblastoma
Time frame: 4-8 weeks post transplant
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 18 Jul 2018.
- Children's Mercy HospitalLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
Related trials for Neuroblastoma
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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.
Gary D Myers, MD principal investigator
Children's Mercy
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 CD34+ cells selected with the Miltenyi Clinimacs machine in people with Neuroblastoma, run by Children's Mercy Hospital Kansas City 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 10 inclusion and 11 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 18 Jul 2018. 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?
The immediate safety of a fludarabine based reduced intensity conditioning regimen and CD34+ stem cell selected mis-matched, related, allogeneic transplant will be assess in patients with relapsed/refractory neuroblastoma (time frame: 6 weeks). 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 18 Jul 2018.