Clinical Trial

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Active, Not Recruiting Phase 2
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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).
Protocol Amendment History 23 amendments
This ClinicalTrials.gov record has been amended 23 times since 2014-06-20; most recent amendment 2026-01-05.
Status change: Recruiting → Active, Not Recruiting 2026-01-05
Status change: Not Yet Recruiting → Recruiting 2014-07-10
Trial Details
NCT Number NCT02171104
Lead Sponsor Masonic Cancer Center, University of Minnesota
Conditions Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria +21 more
Enrollment 149 participants
Start Date 2014-07-10
Primary Completion 2026-01-05 (estimated)
Study Completion 2029-07-14 (estimated)
Updated on ClinicalTrials.gov 2026-01-07