Stem Cell Transplant for Inborn Errors of Metabolism

This study has been completed.

Study NCT00176904 Information provided by Masonic Cancer Center, University of Minnesota

First Received on September 12, 2005. Last Updated on June 14, 2011 History of Changes

Results First Received: May 17, 2011

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Cell Leukodystrophy Gaucher's Disease Fucosidosis Wolman Disease Niemann-Pick Disease Batten Disease GM1 Gangliosidosis Tay Sachs Disease Sandhoff Disease
Interventions:	Procedure: Stem Cell Transplant Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Patients Treated With Stem Cell Transplant	All patients treated with protocol regimen (chemotherapy and stem cell transplant).

Participant Flow: Overall Study

	Patients Treated With Stem Cell Transplant
STARTED	135
COMPLETED	135
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Patients Treated With Stem Cell Transplant	All patients treated with protocol regimen (chemotherapy and stem cell transplant).

Baseline Measures

	Patients Treated With Stem Cell Transplant
Number of Participants [units: participants]	135
Age [units: participants]
<=18 years	117
Between 18 and 65 years	18
>=65 years	0
Age [units: years] Mean ± Standard Deviation	8.7 ± 8.9
Gender [units: participants]
Female	44
Male	91
Region of Enrollment [units: participants]
United States	135

Outcome Measures

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1. Primary:

Overall Survival [ Time Frame: 100 Days, 1 Year and 3 Years ]

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2. Secondary:

Overall Donor Engraftment [ Time Frame: Day 100 ]

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3. Secondary:

Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease [ Time Frame: Day 100 ]

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4. Secondary:

Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease [ Time Frame: Day 100 ]

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5. Secondary:

Number of Patients With Chronic Graft-Versus-Host Disease [ Time Frame: 1 Year Post Transplant ]

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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
Change in Neuropsychometric function data is not available. Data would have been noted as descriptive and patient specific, as well as disease specific; not table format. No formal analysis was intended.

Results Point of Contact:

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

No publications provided by Masonic Cancer Center, University of Minnesota

Publications automatically indexed to this study:

Miller WP, Rothman SM, Nascene D, Kivisto T, DeFor TE, Ziegler RS, Eisengart J, Leiser K, Raymond G, Lund TC, Tolar J, Orchard PJ. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood. 2011 Aug 18;118(7):1971-8. Epub 2011 May 17.

Responsible Party:	Orchard, Paul J., MD, Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:	NCT00176904 History of Changes
Obsolete Identifiers:	NCT00005894
Other Study ID Numbers:	MT1995-01
Study First Received:	September 12, 2005
Results First Received:	May 17, 2011
Last Updated:	June 14, 2011
Health Authority:	United States: Institutional Review Board