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Trial record 14 of 17 for:    Sandhoff Disease

Stem Cell Transplant for Inborn Errors of Metabolism

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00176904
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : June 10, 2011
Last Update Posted : December 28, 2017
Information provided by (Responsible Party):

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Adrenoleukodystrophy
Metachromatic Leukodystrophy
Globoid Cell Leukodystrophy
Gaucher's Disease
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

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
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 

  Baseline Characteristics

  Outcome Measures

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

2.  Secondary:   Overall Donor Engraftment   [ Time Frame: Day 100 ]

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

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

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

  Serious Adverse Events

  Other Adverse Events

  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.

  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.

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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176904     History of Changes
Obsolete Identifiers: NCT00005894
Other Study ID Numbers: MT1995-01
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: May 17, 2011
Results First Posted: June 10, 2011
Last Update Posted: December 28, 2017