NeuroblastomaPhase 1/2NCT06625190
Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors
A Phase 1/2 study testing Miltenyi CliniMACS Prodigy ® system and Zoledronic acid in people with Neuroblastoma and Rhabdomyosarcoma, run by University of Florida at 1 site in United States.
Trial summary
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for hematologic malignancies and non-malignant diseases additionally increasing the donor pool as to… Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma
- What is being tested?
- Miltenyi CliniMACS Prodigy ® system and Zoledronic acid
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Feb 2026to Feb 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients 6 months to ≤ 25 years old
- Relapsed/Refractory Solid Tumor whom failed or deemed ineligible to receive autologous transplant or if autologous transplant did not offer >20% chance of cure with the following diseases:
- neuroblastoma (high risk with relapsed or refractory disease),
- relapsed/refractory rhabdomyosarcoma,
- relapsed/refractory non-rhabdomyosarcoma soft tissue sarcoma (NRSTS): synovial sarcoma, malignant peripheral nerve sheath tumors (MPNST),
- High risk adult type NRSTS: clear cell sarcoma, alveolar soft part sarcoma,
Show all 19Show fewer
- Other high-risk extracranial solid tumors: desmoplastic small round cell tumors, chordoma, malignant rhabdoid tumor, epithelioid sarcoma, myoepithelial tumor
- relapsed/refractory bone tumors: osteosarcoma and Ewing sarcoma/PNET, or
- Wilm's tumor or other high-risk solid tumors with <10% expected survival with conventional treatment.
- Subjects must not have more than one active malignancy at the time of enrollment. (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen [as determined by the treating physician and approved by the PI] may be included.)
- Haplo-identical related donor (at least one full haplotype must be matched).
- Karnofsky or Lansky score ≥60% at the time of enrollment. Karnofsky scores must be used for patients >16 years of age and Lansky scores for patients ≤16 years of age
- Adequate organ function (within 4 weeks of initiation of preparative regimen), defined as:
- Pulmonary: FEV1, FVC, and corrected DLCO must all be ≥ 50% of predicted by pulmonary function tests (PFTs). For children who are unable to perform for PFTs due to age, the criteria are: no evidence of dyspnea at rest and no need for supplemental oxygen.
- Renal: Creatinine clearance or radioisotope GFR ≥60 mL/min/1.73 m2 or a serum creatinine based on age/gender
- Cardiac: Ejection fraction of ≥ 40% by echocardiogram or radionuclide scan (MUGA).
- Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures
- Individuals of childbearing potential (IOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for one year following transplantation to minimize the risk of pregnancy. Prior to study enrollment, individuals of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factor for an unintentional pregnancy.
- Subjects with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for one year following stem cell transplantation.
Who cannot join
- Patients with documented uncontrolled infection at the time of study entry are not eligible.
- Uncontrolled infection is patient without treatment antimicrobials and/or demonstrating progression despite antimicrobials
- Patients with progressive solid tumor disease after relapsed/refractory treatment.
- Demonstrated lack of compliance with medical care, as determined by the treating physician.
- Patients who have received an allogeneic HSCT within 6 months.
- Patients who do not have an eligible allogeneic donor available.
Show all 12Show fewer
- Patients with a life expectancy <3 months
- Patients not meeting inclusion criteria for organ function.
- Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least one year after transplantation.
- Females who are known to be pregnant or breastfeeding.
- History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.
- Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Florida
Gainesville, Florida, 32610
Giselle Moore-Higgs, PhD, APRN, 352-273-9050, mooregj@ufl.edu
Jordan Milner, MD
About this study
Official title: Allogeneic Stem Cell Transplantation Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion With Zoledronic Acid in Combination to Treat Pediatric, Adolescent, and Young Adult Patients With Relapsed/Refractory Solid Tumors
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for hematologic malignancies and non-malignant diseases additionally increasing the donor pool as to allow for haploidentical transplant to safely occur.
While solid tumors can be highly chemotherapy sensitive, many remain resistant and require multimodalities of treatment. Immunotherapy has been developed to harness the immune system in fighting solid tumors, though not all have targeted effects. Some solid tumors are treated with autologous transplants; however, they do not always demonstrate an improved event free survival or overall survival. There has been evidence of the use of allogeneic stem cell transplants to provide a graft versus tumor effect, though studies remain limited.
Read more
By utilizing αβ-T cell and B cell depletion for stem cell transplants and combining with zoledronic acid, the immune system may potentially be harnessed and enhanced to provide an improved graft versus tumor effect in relapsed/refractory solid tumors and promote an improved event-free survival and overall survival.
This study will investigate the safety of treatment with a stem cell graft depleted of αβ-T cell and CD19+ B cells in combination with zoledronic acid in pediatric and young adult patients with select solid tumors, as well as whether this treatment improves survival rates in these patients.
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors, Clear Cell Sarcoma, Alveolar Soft Part Sarcoma
- Being tested
- Miltenyi CliniMACS Prodigy ® system, Zoledronic acid
- Keywords
- pediatric solid tumors, stem cell transplantation, graft manipulation
- Medical subject headings
- Neuroblastoma, Rhabdomyosarcoma, Sarcoma, Synovial, Nerve Sheath Neoplasms, Sarcoma, Clear Cell, Sarcoma, Alveolar Soft Part, Desmoplastic Small Round Cell Tumor, Chordoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Miltenyi CliniMACS Prodigy ® systemDevice
Subjects will receive an allogeneic stem cell transplant that has been depleted of ⍺/β T-cells and CD19+ B-cells using the Miltenyi CliniMACS Prodigy® system.
Zoledronic acidDrug
All subjects will receive zoledronic acid intravenously on days +28, +56, +84, +112, and +140.
Dosing in the phase Ib portion of the study will follow a 3 + 3 design where the first 3 subjects will receive the expected phase II dose of 1.25 mg/m2 (dose level 1). If no dose-limiting toxicities (DLTs) occur in these subjects, dose level 1 will be the maximum tolerated dose. However, if at least 1 DLT is observed in the first 3 patients, 3 additional subjects will be enrolled at dose level 1. If more than 2 DLTs are observed in these 6 subjects, then dose de-escalation to 0.8 mg/m2 (dose level 0) will occur and 3-6 additional subjects may be enrolled.
All subjects in the phase II portion of the study will receive the maximum tolerated dose determined in the phase Ib portion.
| Study group | Role | What they receive |
|---|---|---|
| alpha/beta T cell and CD19+ B cell depleted stem cell graft with zoledronic acid | Experimental | Not described Receives: Device: Miltenyi CliniMACS Prodigy ® system; Drug: Zoledronic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease-free survival rate
Determine the disease-free survival rate at 1 year post-transplant
Measured: 1 year post-transplant
Incidence of aGVHD
Determine the incidence of grade II-IV acute graft versus host disease (aGVHD)
Measured: 2 years post-transplant
Secondary outcomes (5)
Disease-free survival rate
Determine the disease-free survival rate at 100 days post-transplant
Measured: 100 days post-transplant
Disease-free survival rate
Determine the disease-free survival rate at 2 years post-transplant
Measured: 2 years post-transplant
Overall survival rate
Determine the overall survival rate at 100 days post-transplant
Measured: 100 days post-transplant
Overall survival rate
Determine the overall survival rate at 1 year post-transplant
Measured: 1 year post-transplant
Overall survival rate
Determine the overall survival rate at 2 years post-transplant
Measured: 2 years post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2024
- Study start11 Feb 2026
- Primary completionFeb 2030 (estimated)
- Study completionFeb 2030 (estimated)
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 University of FloridaThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Ocala Royal Dames for Cancer Research, Florida Department of Health
- Responsible party
- Sponsor
- Investigators
- Jordan Milner, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06625190
- Sponsor's protocol ID
- UF-PED-006
- Other IDs
- IRB202500132
- Status last verified
- Jun 2026
- First submitted
- 1 Oct 2024
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Stem Cell Transplantation Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion With Zoledronic Acid in Combination to Treat Pediatric, Adolescent, and Young Adult Patients With Relapsed/Refractory Solid Tumors
- ClinicalTrials.gov ID
- NCT06625190
- Acronym / sponsor ID
- UF-PED-006
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors, Clear Cell Sarcoma, Alveolar Soft Part Sarcoma
- Keywords
- pediatric solid tumors, stem cell transplantation, graft manipulation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Ocala Royal Dames for Cancer Research, Florida Department of Health
- Study start
- 11 Feb 2026 (actual)
- Primary completion
- Feb 2030 (estimated)
- Study completion
- Feb 2030 (estimated)
- First posted
- 3 Oct 2024 (actual)
- 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.
Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for hematologic malignancies and non-malignant diseases additionally increasing the donor pool as to allow for haploidentical transplant to safely occur.
While solid tumors can be highly chemotherapy sensitive, many remain resistant and require multimodalities of treatment. Immunotherapy has been developed to harness the immune system in fighting solid tumors, though not all have targeted effects. Some solid tumors are treated with autologous transplants; however, they do not always demonstrate an improved event free survival or overall survival. There has been evidence of the use of allogeneic stem cell transplants to provide a graft versus tumor effect, though studies remain limited.
By utilizing αβ-T cell and B cell depletion for stem cell transplants and combining with zoledronic acid, the immune system may potentially be harnessed and enhanced to provide an improved graft versus tumor effect in relapsed/refractory solid tumors and promote an improved event-free survival and overall survival.
This study will investigate the safety of treatment with a stem cell graft depleted of αβ-T cell and CD19+ B cells in combination with zoledronic acid in pediatric and young adult patients with select solid tumors, as well as whether this treatment improves survival rates in these patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| alpha/beta T cell and CD19+ B cell depleted stem cell graft with zoledronic acid | Experimental | Device: Miltenyi CliniMACS Prodigy ® system, Drug: Zoledronic acid |
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Patients 6 months to ≤ 25 years old
- Relapsed/Refractory Solid Tumor whom failed or deemed ineligible to receive autologous transplant or if autologous transplant did not offer >20% chance of cure with the following diseases:
- Subjects must not have more than one active malignancy at the time of enrollment. (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen [as determined by the treating physician and approved by the PI] may be included.)
- Haplo-identical related donor (at least one full haplotype must be matched).
- Karnofsky or Lansky score ≥60% at the time of enrollment. Karnofsky scores must be used for patients >16 years of age and Lansky scores for patients ≤16 years of age
Key exclusion criteria (12 listed)
- Patients with documented uncontrolled infection at the time of study entry are not eligible.
- Patients with progressive solid tumor disease after relapsed/refractory treatment.
- Demonstrated lack of compliance with medical care, as determined by the treating physician.
- Patients who have received an allogeneic HSCT within 6 months.
- Patients who do not have an eligible allogeneic donor available.
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 |
|---|---|---|
| Miltenyi CliniMACS Prodigy ® systemSubjects will receive an allogeneic stem cell transplant that has been depleted of ⍺/β T-cells and CD19+ B-cells using the Miltenyi CliniMACS Prodigy® system. | Device | alpha/beta T cell and CD19+ B cell depleted stem cell graft with zoledronic acid |
| Zoledronic acidAll subjects will receive zoledronic acid intravenously on days +28, +56, +84, +112, and +140. Dosing in the phase Ib portion of the study will follow a 3 + 3 design where the first 3 subjects will receive the expected phase II dose of 1.25 mg/m2 (dose level 1). If no dose-limiting toxicities (DLTs) occur in these subjects, dose level 1 will be the maximum tolerated dose. However, if at least 1 DLT is observed in the first 3 patients, 3 additional subjects will be enrolled at dose level 1. If more than 2 DLTs are observed in these 6 subjects, then dose de-escalation to 0.8 mg/m2 (dose level 0) will occur and 3-6 additional subjects may be enrolled. All subjects in the phase II portion of the study will receive the maximum tolerated dose determined in the phase Ib portion. | Drug | alpha/beta T cell and CD19+ B cell depleted stem cell graft with zoledronic acid |
Primary outcome
Disease-free survival rate
Time frame: 1 year post-transplant
Determine the disease-free survival rate at 1 year post-transplant
Incidence of aGVHD
Time frame: 2 years post-transplant
Determine the incidence of grade II-IV acute graft versus host disease (aGVHD)
5 secondary outcomes
Disease-free survival rate
Time frame: 100 days post-transplant
Disease-free survival rate
Time frame: 2 years post-transplant
Overall survival rate
Time frame: 100 days post-transplant
Overall survival rate
Time frame: 1 year post-transplant
Overall survival rate
Time frame: 2 years post-transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jun 2026.
Related trials for Neuroblastoma
RecruitingPhase 3
Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
Ganglioneuroblastoma, Nodular, Neuroblastoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingObservational study
International PPB/DICER1 Registry
Pleuropulmonary Blastoma, Sertoli-Leydig Cell Tumor
RecruitingPhase 2/3
A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients
High-Risk Neuroblastoma
Active, not recruitingObservational study
A Patient Registry of Patients With High-risk Neuroblastoma Being Treated With the Monoclonal Antibody Dinutuximab Beta
Neuroblastoma
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.
Jordan Milner, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Miltenyi CliniMACS Prodigy ® system and Zoledronic acid in people with Neuroblastoma and Rhabdomyosarcoma, run by University of Florida 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 19 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 30 Jun 2026. 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 combined 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.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Giselle Moore-Higgs, PhD, APRN (352-273-9050, mooregj@ufl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease-free survival rate (time frame: 1 year post-transplant). The study lists 2 primary outcomes in all. 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 Jun 2026.