Stem Cell Transplantation for Hurler

This study is ongoing, but not recruiting participants.

Study NCT00176917 Information provided by Masonic Cancer Center, University of Minnesota

First Received: September 12, 2005 Last Updated: July 28, 2009 History of Changes

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Single Group Assignment
Conditions:	Mucopolysaccharidosis I Mucopolysaccharidosis VI Mannosidosis Mucolipidosis Type II (I-cell Disease)
Interventions:	Procedure: Stem Cell Transplant Drug: Busulfan, Cyclophosphamide, ATG

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups

	Description
Transplant Patients	Patients that received hematopoietic stem cell transplant.

Participant Flow: Overall Study

Baseline Characteristics

Reporting Groups

	Description
Transplant Patients	Patients that received hematopoietic stem cell transplant.

Baseline Measures

	Transplant Patients
Number of Participants [units: participants]	41
Age [units: participants]
<=18 years	41
Between 18 and 65 years	0
>=65 years	0
Age [units: years] Mean ± Standard Deviation	1.8 ± 1.4
Gender [units: participants]
Female	23
Male	18
Region of Enrollment [units: participants]
United States	41

Outcome Measures

1. Primary:

Mean Percentage of Donor Cells in Study Population (Chimerism).

Show Outcome Measure 1

2. Secondary:

Number of Patients Surviving on Study

Show Outcome Measure 2

3. Secondary:

Number of Patients Who Failed Engraftment.

Show Outcome Measure 3

4. Secondary:

Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).

Show Outcome Measure 4