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Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Sponsor: Washington University School of Medicine
Summary
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
Official title: A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders
Key Details
Gender
All
Age Range
1 Day - 21 Years
Study Type
INTERVENTIONAL
Enrollment
29
Start Date
2017-03-20
Completion Date
2033-04
Last Updated
2026-01-07
Healthy Volunteers
No
Conditions
Interventions
RIC regimen
Days -60 to -21: hydroxyurea (30mg/kg/day po) \>6hrs prior to 1st dose: alemtuzumab (3mg IV) Day -21: alemtuzumab (10mg IV or S/C) Day -20: alemtuzumab (15mg IV or S/C) (10mg if \< 10kg) Day -19: alemtuzumab (20mg IV or S/C) (10mg if \< 10kg) Days -8 to -4: fludarabine (30mg/m2/day IV) Day -4: thiotepa (8mg/kg IV) Day -3: melphalan (140mg/m2) Days -2 to -1: rest days/no therapy Day 0: bone marrow transplant
GVHD prophylaxis regimen
Day +3 to +4: cyclophosphamide (50mg/kg/day IV) Day +5: Start of tacrolimus \& Start of mycophenolate mofetil (MMF) Days +5, +14, +30, +60, +90: abatacept (IND) (10mg/kg/day IV) Day +90: rituximab (375mg/m2 IV once) Patients \>/= 12 yrs - Days +120 to +180: abatacept (IND) monthly (10mg/kg/day IV) Patients \>/= 12 yrs - Days +210 to +390: abatacept (IND) monthly (5mg/kg/day) Patients \<12 yrs - Days +120 to +390: abatacept (IND) monthly (5mg/kg/day IV)
Locations (4)
Yale School of Medicine
New Haven, Connecticut, United States
Nemours Children's Health
Wilmington, Delaware, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Washington University School of Medicine
St Louis, Missouri, United States