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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Sponsor: Masonic Cancer Center, University of Minnesota
Summary
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Official title: MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Key Details
Gender
All
Age Range
Any - 55 Years
Study Type
INTERVENTIONAL
Enrollment
149
Start Date
2014-07-10
Completion Date
2029-07-14
Last Updated
2026-01-07
Healthy Volunteers
No
Conditions
Interventions
Stem Cell Transplantation
Infusion given on Day 0
IMD Preparative Regimen
* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan
Osteopetrosis Only Preparative Regimen
* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Thiotepa
Osteopetrosis Haploidentical Only Preparative Regimen
* Rituximab * Alemtuzumab * Busulfan * Fludarabine
cALD SR-A (Standard-Risk, Regimen A)
N-acetylcysteine start day +1 through day +28
cALD SR-B (Standard-Risk, Regimen B)
N-acetylcysteine start day +1through day +56
cALD HR-D (High-Risk, Regimen C)
N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100
cALD HR-D (High-Risk, Regimen D)
N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100
Locations (1)
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States