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

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Baseline Characteristics

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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

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1. Primary:

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

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2. Secondary:

Number of Patients Surviving on Study

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3. Secondary:

Number of Patients Who Failed Engraftment.

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4. Secondary:

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

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

All Principal Investigators ARE employed by the organization sponsoring the study.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Based on variability in age, diagnosis and condition of these patients, the data on enzyme levels and neuropsych testing is extremely difficult to report. For adverse events, see Secondary Outcome Measures #3 and #4.

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data

Based on variability in age, diagnosis and condition of these patients, the data on enzyme levels and neuropsych testing is extremely difficult to report.

For adverse events, see Secondary Outcome Measures #3 and #4.

Results Point of Contact:

Name/Title: Paul J. Orchard, M.D.
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2313
e-mail: orcha001@umn.edu

No publications provided

Responsible Party:	Masonic Cancer Center at the University of Minnesota ( Orchard, Paul J., MD )
Study ID Numbers:	0104M93821, MT1999-07
Study First Received:	September 12, 2005
Results First Received:	July 28, 2009
Last Updated:	July 28, 2009
ClinicalTrials.gov Identifier:	NCT00176917 History of Changes
Health Authority:	United States: Institutional Review Board