UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT02722668

UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

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.

Status: 
Active, not recruiting
Study Date: 
Fri, 04/01/2016 to Wed, 12/01/2027
Bone Marrow Disease(s): 
myelodysplastic syndromes (MDS)
Associated Drug(s): 
Intervention: 
Drug: Fludarabine Both Arms: 30 mg/m^2 IV over 1 hour Day -6 to Day -2 Other Name: Fludara Drug: Cyclophosphamide Arm 1: 50 mg/kg IV over 2 hours Day -6 Other Name: Cytoxan 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. Other Name: Mycophenolate Mofetil 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. Other Name: Rapamycin Radiation: TBI Both Arms: 200 cGy on Day -1 Other Name: Total body irradiation Biological: Umbilical cord blood cell infusion Both Arms: Day 0 Other Name: UCB Biological: ATG Arm 2: 15 mg/kg IV every 12 hours Day -6 to Day -4 Other Name: Anti-thymocyte Globulin