NCT02722668

UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep

Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 75 Years
• Healthy volunteers: Not accepted

Summary

This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.

Conditions

• Acute Leukemia
• Acute Myeloid Leukemia
• Acute Lymphoblastic Leukemia/Lymphoma
• Burkitt's Lymphoma
• Natural Killer Cell Malignancies
• Chronic Myelogenous Leukemia
• Myelodysplastic Syndrome
• Large-cell Lymphoma
• Hodgkin Lymphoma
• Multiple Myeloma
• Relapsed Chronic Lymphocytic Leukemia
• Relapsed Small Lymphocytic Lymphoma
• Marginal Zone B-cell Lymphoma
• Follicular Lymphoma
• Lymphoplasmacytic Lymphoma
• Mantle-cell Lymphoma
• Prolymphocytic Leukemia
• Bone Marrow Failure Syndromes
• Myeloproliferative Neoplasms/Myelofibrosis
• Biphenotypic/Undifferentiated/Prolymphocytic Leukemias
• MRD Positive Leukemia
• Leukemia or MDS in Aplasia
• Relapsed T-Cell Lymphoma
• Relapsed Multiple Myeloma
• Plasma Cell Leukemia

Interventions

Type	Name	Description
DRUG	Fludarabine	Both Arms: 30 mg/m\^2 IV over 1 hour Day -6 to Day -2
DRUG	Cyclophosphamide	Arm 1: 50 mg/kg IV over 2 hours Day -6
DRUG	MMF	Both Arms: Mycophenolate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. In obese patients (\>125% IBW) 15 mg/kg every 12 hours may be considered. Pediatric patient (\<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning Day -3. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. Patients will be eligible for MMF dosing and pharmacokinetics studies. Stop MMF at Day +30 or 7 days after engraftment, whichever day is later, if no acute graft versus host disease (GVHD). (Definition of engraftment is 1st day of 3 consecutive days of absolute neutrophil count \[ANC) ≥ 0.5 x 109 /L\]). If no donor engraftment, do not stop MMF.
DRUG	Sirolimus	Both Arms: Adult Dosing: Sirolimus will be administered starting at Day -3 with 8-12 mg oral loading dose followed by single dose 4 mg/day with a target serum concentration of 3 to 12 mg/mL by high-performance liquid chromatography (HPLC) and will be monitored per institutional guidelines. In the absence of acute GVHD sirolimus may be tapered starting at Day +100 and eliminated by Day +180 post-transplantation. Pediatric Dosing: Sirolimus will be administered starting on Day -3 with an oral loading dose of 10 mg followed by maintenance dosing of 2.5 mg/m\^2/day (Maximum total daily dose of 4mg) as per institutional guidelines. Target serum concentration goals are 3 to 12 mg/mL by high-performance liquid chromatography (HPLC) and will be monitored per institutional guidelines.
RADIATION	TBI	Both Arms: 200 cGy on Day -1
BIOLOGICAL	Umbilical cord blood cell infusion	Both Arms: Day 0
BIOLOGICAL	ATG	Arm 2: 15 mg/kg IV every 12 hours Day -6 to Day -4

Timeline

• Start date: 2017-05-15
• Primary completion: 2023-02-22
• Completion: 2024-02-22
• First posted: 2016-03-30
• Last updated: 2025-07-04
• Results posted: 2025-07-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study