138
Participants
Start Date
March 16, 2017
Primary Completion Date
May 2, 2023
Study Completion Date
August 2, 2023
Busulfan
"A:~Busulfan dose will be 3.2 mg/kg administered as a single daily dose IV on days -8 through -5 with dosing adjusted using targeted pharmacokinetics."
Fludarabine
"A:~Fludarabine dose will be 35 mg/m\^2/day administered IV on days -7 through -3 (total fludarabine dose is 175 mg/m\^2).~C:~Fludarabine 30mg/m2 IV dose will be given on Days -8, -7, -6, -5, -4"
r-ATG
"A:~r-ATG will be administered IV on day -6 at 0.5mg/kg, on day -5 at 1 mg/kg and on days -4, -3 and -2 at 1.5mg/kg (total r-ATG dose is 6 mg/kg)."
Hematopoietic Cell Transplant
"A,B,C:~Day 0 is the day of transplantation."
Tacrolimus
"A:~Tacrolimus commences on day -3 and extends through day +180 after transplantation with doses adjusted to maintain appropriate levels according to institutional guidelines.~C:~Tacrolimus at therapeutic doses through Day 180, then taper per institutional guidelines"
Methotrexate
"A:~Methotrexate will be administered intravenously on day+1 at 15mg/m\^2, day+3 at 10mg/m\^2, day+6 at 10mg/m\^2, and day+11 at 10mg/m\^2.~C:~Methotrexate IV 7.5 mg/m2 dose will be given on Days +1, 3, +6 following transplant"
Standard of Care
Continue to receive standard of care treatment per patient's SCD physician.
Alemtuzumab
"B:~Alemtuzumab 0.03 mg/kg IV dose will be given on Day -7, Alemtuzumab 0.1 mg/kg IV dose will be given on Day -6, Alemtuzumab 0.3 mg/kg IV dose will be given on Day -5,-4,-3~C:~Alemtuzumab test dose 3 mg IV once 24 hours prior to 1st dose of Alemtuzumab Alemtuzumab 10 mg IV, 15 mg IV, 20 mg IV given on Days -22 through Day -18. Alemtuzumab doses may be administered between Days -22 and -18 but are required to be on three consecutive days."
Total Body Irradiation (TBI)
Total Body Irradiation 300 cGY on Day -2
Sirolimus
Sirolimus at therapeutic doses through day 180, then taper per institutional guidelines if donor CD3+ \>50%
Melphalan
"C:~Melphalan 140 mg/m2 IV dose will be given on Day -3"
G-CSF
G-CSF 5 μg/kg/day continue until neutrophil engraftment.
Icahn School of Medicine at Mount Sinai, New York
Weill Cornell Medical College, New York
Montefiore Medical Center/Albert Einstein School of Medicine, The Bronx
Cohen Children's Medical Center, New Hyde Park
New York Presbyterian Brooklyn Methodist Hospital, Brooklyn
Children's Hospital of Philadelphia, Philadelphia
Children's National Medical Center, Washington D.C.
University of Virginia, Charlottesville
Virginia Commonwealth University, Richmond
University of North Carolina Hospital at Chapel Hill, Chapel Hill
Duke University Medical Center, Durham
Medical University of South Carolina, Charleston
Grady Memorial Hospital, Atlanta
Children's Healthcare of Atlanta, Atlanta
Emory University, Atlanta
Augusta University Medical Center, Augusta
University of Florida Gainsville, Gainesville
Foundation for Sickle Cell Research/Florida Sickle Inc., Hollywood
University of Miami, Miami
Ohio State University, Columbus
Barbara Ann Karmanos Cancer Institute, Detroit
University of Iowa, Iowa City
Washington University/St. Louis Children's Hospital, St Louis
Children's Hospital of New Orleans, New Orleans
University of Oklahoma, Oklahoma City
University of Texas Health Sciences Center, Houston
Baylor College of Medicine/The Methodist Hospital, Houston
University of Texas/MD Anderson CRC, Houston
Benioff Children's Hospital at Oakland, Oakland
Oregon Health Sciences University, Portland
Dana Farber Cancer Institute/Brigham & Women's Hospital, Boston
Dana Farber Cancer Institute/Massachusetts General Hospital, Boston
Boston University, Boston
Hackensack University Medical Center, Hackensack
Newark Beth Israel Medical Center, Newark
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Blood and Marrow Transplant Clinical Trials Network
NETWORK
Dana-Farber Cancer Institute
OTHER
National Marrow Donor Program
OTHER
Emory University
OTHER
Medical College of Wisconsin
OTHER