Clinical Trials Logo

Maroteaux-Lamy Syndrome clinical trials

View clinical trials related to Maroteaux-Lamy Syndrome.

Filter by:
  • Recruiting  
  • Page 1

NCT ID: NCT02171104 Recruiting - Hunter Syndrome Clinical Trials

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

Start date: July 10, 2014
Phase: Phase 2
Study type: Interventional

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).