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Stem Cell Transplantation

Stem Cell Transplantation for Leukemia

Phase 3

Recruiting

Led By Heather J Symons

Research Sponsored by Children's Oncology Group

Eligibility Criteria Checklist

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

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up-to 1-year post-hct

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial compares stem cell transplants from related or unrelated donors to treat leukemia or MDS in children, teens, & young adults.

Who is the study for?

This trial is for children, adolescents, and young adults aged 6 months to under 22 years with acute leukemia or myelodysplastic syndrome (MDS) who need a stem cell transplant but don't have a matched sibling donor. Participants must be in good health with proper kidney, liver, heart, and lung function. They can't join if they're pregnant, unwilling to use contraception during the study, have uncontrolled infections or certain genetic disorders.

What is being tested?

The trial compares two types of stem cell transplants: one from mismatched related donors (family members not fully matching) versus another from matched unrelated donors found through registries. It aims to determine which source is better for treating high-risk leukemia or MDS after receiving intensive chemotherapy or radiation therapy.

What are the potential side effects?

Potential side effects include reactions to immune globulin infusions; complications from chemotherapy like nausea and low blood counts; risks associated with lumbar punctures; organ damage due to intense treatment regimens; and graft-versus-host disease where donated cells attack the patient's body.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1-year post-hct

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1-year post-hct

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

Treatment Details

Study Objectives

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

Primary study objectives

Disease free survival (DFS) (where an event is the occurrence of death from any cause or relapse)

Severe GVHD (Grade III-IV acute GVHD or chronic GVHD requiring systemic immunosuppressive therapy)

Secondary study objectives

Overall survival (OS)

Summary score from the Generic Pediatric Quality of Life Inventory (PedsQL) (excluding School Functioning)

Summary score from the PedsQL Stem Cell Transplant module

Other study objectives

Acute graft versus host disease (aGVHD) Grades II-IV and III-IV

Graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) (using the standard definition and two landmark definitions)

Mild, moderate, and severe chronic graft versus host disease (cGVHD)

+13 more

Awards & Highlights

No Placebo-Only Group

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

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Arm C (haploHCT)Experimental Treatment20 Interventions

Patients who only have a haplo donor receive a myeloablative conditioning regimen with PTCy or alpha beta T cell depletion at the discretion of the treating provider. Patients then undergo haploHCT on day 0. Patients undergoing myeloablative conditioning regimen with PTCy also receive GVHD prophylaxis on days 3-5. Patients undergo lumbar puncture, bone marrow aspiration, and ECHO or MUGA during screening. Patients also undergo collection of blood throughout the trial.

Group II: Arm B (MUD-HCT)Experimental Treatment17 Interventions

Patients receive a TBI-based or chemotherapy-based myeloablative conditioning regimen between days -9 and -2, followed by MUD-HCT on day 0. Patients then receive GVHD prophylaxis regimen on days 1-11. Patients undergo lumbar puncture, bone marrow aspiration, and ECHO or MUGA during screening. Patients also undergo collection of blood throughout the trial.

Group III: Arm A (halploHCT)Experimental Treatment20 Interventions

Patients receive a myeloablative conditioning regimen with PTCy or alpha beta T cell depletion at the discretion of the treating provider. Patients then undergo haploHCT on day 0. Patients undergoing myeloablative conditioning regimen with PTCy also receive GVHD prophylaxis on days 3-5. Patients undergo lumbar puncture, bone marrow aspiration, and ECHO or MUGA during screening. Patients also undergo collection of blood throughout the trial.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

2010

Completed Phase 4

~2310

Multigated Acquisition Scan

2015

Completed Phase 3

~270