A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies

Phase 2

Terminated

• Conditions: Myeloid Malignancy; Hematologic Malignancy
• Interventions: Drug: Ruxolitinib; Drug: Anti-thymocyte globulin (ATG); Drug: Mesna; Drug: Cyclosporine; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Methotrexate; Radiation: Total Body Irradiation (radiation treatment); Drug: Busulfan; Drug: Bone marrow infusion; Drug: Thiotepa

• Registration Number: NCT05579769
• Lead Sponsor: St. Jude Children's Research Hospital

Brief Summary

The participants are being asked to take part in this clinical trial because the participant have a lymphoid or myeloid based cancer diagnosis that requires a bone marrow transplant.

Primary Objectives
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Detailed Description

The investigator propose to employ two preparative regimens based on the underlying hematological malignancy. For hematological malignancies of the lymphoid lineage we will use a standard preparative regimen consisting of Total Body Irradiation and cyclophosphamide (TBI/Cy), unless TBI is contraindicated. For myeloid malignancies we will use thiotepa, busulf...

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2022-10-11
• Study First Submitted QC: 2022-10-11
• Study First Posted: 2022-10-14
• Study Start: 2023-03-14
• Primary Completion: 2024-05-08
• Study Completion: 2024-05-08
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-09
• Last Update Posted: 2024-07-10

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ArmA- Lymphoid	Bone marrow infusion	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmB-Myeloid	Anti-thymocyte globulin (ATG)	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmA- Lymphoid	Total Body Irradiation (radiation treatment)	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmA- Lymphoid	Ruxolitinib	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmA- Lymphoid	Anti-thymocyte globulin (ATG)	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmB-Myeloid	Ruxolitinib	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmB-Myeloid	Bone marrow infusion	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmA- Lymphoid	Mesna	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmA- Lymphoid	Cyclosporine	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmA- Lymphoid	Cyclophosphamide	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmA- Lymphoid	Methotrexate	Total Body Irradiation and cyclophosphamide (TBI/Cy)
ArmB-Myeloid	Cyclosporine	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmB-Myeloid	Fludarabine	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmB-Myeloid	Methotrexate	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmB-Myeloid	Busulfan	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)
ArmB-Myeloid	Thiotepa	TBF with comparable NRM in comparison to busulfan and cyclophosphamide (BuCy)

Primary Outcome Measures

Name	Time	Method
Proportion of severe acute GVHD (saGVHD) using a prophylaxis regimen with no calcineurin inhibitors after day 100 post first allogeneic Human Leukocyte antigen (HLA)-matched sibling or unrelated donor HCT for hematological malignancies.	100 days post transplant	Development of saGVHD at or before Day 100 post transplant is considered as an event.

Secondary Outcome Measures

Name	Time	Method
Incidence of Leukemic Relapse	One-year post-transplantation.	Bone marrow studies for disease status evaluation will be performed.
Non-relapse mortality	One-year post-transplantation	The one-year non-relapse mortality (NRM is defined by the patient who expire while in remission within one year. The probability of NRM is estimated by the cumulative incidence method.
Overall Survival	one year post-transplantation	The one-year survival is defined by the patient who has not died within one year after post transplantation. The probability is estimated by the Kaplan-Meier method.
Incidence of Chronic Graft Versus Host Disease (cGVHD)	1 year post transplant	Ongoing assessment of toxicity will be done using the NCI CTCAE version 3.0. Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The cumulative incidence of cGvHD will be estimated using Kalbfleisch-Prentice method.

Trial Locations

Locations (1)

St. Jude Children's Research Hospital; 🇺🇸 Memphis, Tennessee, United States