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Immunotherapy

Stem Cell Transplant + Zometa for Pediatric Blood Cancers

Phase 1

Recruiting

Led By Mario Otto, MD, PhD

Research Sponsored by University of Wisconsin, Madison

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with hematologic malignancy must have no HLA identical sibling or suitable unrelated donor OR time needed to find an acceptable unrelated donor match would likely result in disease progression such that the patient may become ineligible for any type of potentially curative transplant

Relapsed or primary therapy-refractory AML with bone marrow blast < 20%

Must not have

Heart failure or uncontrolled cardiac rhythm disturbance

Prior organ allograft

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new way to transplant stem cells from a donor who is only a half-match to the patient, using a drug to remove TCRαβ+ cells and CD19+ cells.

Who is the study for?

This trial is for pediatric patients with high-risk or relapsed blood cancers and solid tumors who have a suitable haploidentical donor. It's not open to those pregnant, breastfeeding, with uncontrolled infections, prior organ transplants, or conditions that could affect study participation.

What is being tested?

The trial tests a transplant using stem cells from a half-matched donor that are filtered to remove certain immune cells (TCRαβ+/CD19+), combined with Zoledronate post-transplant. This Phase I study aims to assess the safety of this approach.

What are the potential side effects?

Potential side effects may include complications from the stem cell transplant like infection risk due to immune suppression and reactions related to Zoledronate such as fever, bone pain, and nausea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have blood cancer and either can't find a matching donor or can't wait for one without my disease getting worse.

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My AML is not responding to treatment and my bone marrow has less than 20% cancer cells.

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I have been diagnosed with a blood disorder affecting my bone marrow.

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My Hodgkin lymphoma has returned and I can't have a stem cell transplant.

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My non-Hodgkin lymphoma did not respond well to initial treatments and is not eligible for a specific stem cell transplant.

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My solid tumor cannot be treated with a stem cell transplant.

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My neuroblastoma is high risk and has come back or didn't respond to treatment.

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My leukemia has returned or didn't respond to treatment, and I need a transplant.

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My Hodgkin lymphoma has returned after a stem cell transplant.

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My non-Hodgkin lymphoma has returned after a stem cell transplant.

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My cancer is a high-risk type and has come back or didn't respond to initial treatment.

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I have a blood cancer or a solid tumor.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have heart failure or uncontrolled heart rhythm problems.

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I have received an organ transplant from another person.

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I am HIV positive.

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I do not have any serious, uncontrolled infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Immune reconstitution

Biotin

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: TCRαβ+/CD19+ depleted Haploidentical HSCT+ ZoledronateExperimental Treatment2 Interventions

Patients with high-risk leukemia (who are at least one year of age and who have not received TBI as conditioning for a previous HSCT) will receive myeloablative conditioning with ATG, Fludarabine, Thiotepa, and TBI. All other subjects will undergo a reduced-intensity conditioning regimen consisting of ATG, Fludarabine, Thiotepa, and Melphalan prior to transplant with a KIR/KIR ligand mismatched haploidentical donor peripheral blood stem cell graft depleted of TCR-αβ+ and CD19+ cells. Patients will receive 5 doses of zoledronate (at 28 day intervals) starting 28 days after stem cell transplant.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Zoledronate

2016

Completed Phase 4

~480

0 / 16Eligibility criteria met