Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies (UCAML)
In this study, the investigators will use busulfan and cyclophosphamide (BuCy) backbone with the addition of fludarabine as the preparative Stem Cell Transplant (SCT) regimen. As an attempt to improve engraftment rate and reduce infections, the investigators are going to incorporate fludarabine in the conditioning regimen. The use of a BuCy backbone has been widely used and comparable to total body irradiation and cyclophosphamide (Cy/TBI) regimen.
Encouraging data on adding fludarabine to the SCT regimen have been reported. A fludarabine-based, conditioning regimen, with adequate immunosuppressive activity could conceivably allow engraftment of stem cells from alternative donors in hematologic malignancies patients with acceptable engraftment rates and low transplant-related mortality. Regimen-related toxicity is believed to be a major contributing factor to GVHD. Therefore this approach may also lead to reduced GVHD, as some investigators have suggested.
In an attempt to decrease the rate of viral infection and reactivation, the investigators will avoid ATG (Thymoglobulin) / Campath (anti-CD52), and instead administer Mycophenolate Mofetil (MMF). The addition of fludarabine should compensate any increase risk of graft failure with the removal of the ATG/Campath. The investigators anticipate that the removal of ATG/Campath will facilitate immune reconstitution more efficiently after receiving a UCBT.
Myeloid Hematological Malignancies
Procedure: Cord Blood Stem Cell Infusion
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies (UCAML)|
- Overall survival at 1 year after UCB transplant in pediatric patients. [ Time Frame: 1 year ] [ Designated as safety issue: No ]To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
- Number of participants with severe acute GVHD Grade III-IV as an assessment of safety. [ Time Frame: Day 100 ] [ Designated as safety issue: Yes ]To estimate the risk of severe Grade III-IV acute GvHD at Day 100.
- Number of participants with chronic GvHD as an assessment of safety. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]To estimate the risk of chronic GvHD at 1 year.
- Assess relapse rate after transplant. [ Time Frame: 1 and 3 years ] [ Designated as safety issue: Yes ]To assess relapse rate at 1 and 3 years after transplant.
- To evaluate donor engraftment after transplant. [ Time Frame: 100 days, 6, 9, 12, 24 and 36 months ] [ Designated as safety issue: No ]To evaluate donor engraftment at 100 days, 6, 9, 12, 24, and 36 months after transplant.
- Assessment of platelet count recovery. [ Time Frame: Day 42 ] [ Designated as safety issue: No ]To determine platelet count recovery at Day 42 post-transplant.
- Assessment of neutrophil count recovery. [ Time Frame: Day 42 ] [ Designated as safety issue: No ]To determine neutrophil count recovery at Day 42 post-transplant.
|Study Start Date:||November 2010|
|Estimated Study Completion Date:||November 2020|
|Estimated Primary Completion Date:||November 2018 (Final data collection date for primary outcome measure)|
Experimental: Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cyclophosphamide, Fludarabine, Cord Blood Stem Cell Infusion
Busulfan dosing will be as follows: Patients < 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients > 12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. It will be given on Days -9, -8, -7 and -6.
Other Name: BusulfexDrug: Cyclophosphamide
Cyclophosphamide (50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours. The total dose to be given over 4 days is 200 mg/kg.
Other Name: CTX, CytoxanDrug: Fludarabine
Fludarabine 40 mg/m^2 will be given IV daily over 1 hour on Days -3, -2 and -1.
Other Name: FluderaProcedure: Cord Blood Stem Cell Infusion
The cord blood stem cells will be infused on Day 0.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01247701
|Contact: Caridad Martinez, MDemail@example.com|
|United States, Texas|
|Texas Children's Hospital||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Caridad A Martinez, MD 832-824-4692 firstname.lastname@example.org|
|Principal Investigator:||Caridad A Martinez, MD||Baylor College of Medicine|
|Principal Investigator:||Robert A Krance, MD||Baylor College of Medicine|