Stem Cell Transplantation for Hurler
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|ClinicalTrials.gov Identifier: NCT00176917|
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : September 4, 2009
Last Update Posted : December 28, 2017
|Study Design||Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment|
Mucolipidosis Type II (I-cell Disease)
Procedure: Stem Cell Transplant
Drug: Busulfan, Cyclophosphamide, ATG
Based on variability in age, diagnosis and condition of these patients, the data on enzyme levels and neuropsych testing is extremely difficult to report.
See Secondary Outcome Measures #3 and #4; no other adverse events were collected.