Stem Cell Transplant for Bone Marrow Failures

This study has been completed.

Study NCT00176878 Information provided by Masonic Cancer Center, University of Minnesota

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

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Parallel Assignment
Conditions:	Diamond-Blackfan Anemia Kostmann's Neutropenia Shwachman-Diamond Syndrome
Interventions:	Procedure: Stem cell transplant Drug: Busulfan, fludarabine and anti-thymocyte globulin (ATG) Procedure: Total lymphoid irradiation

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
Intent-To-Treat	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Participant Flow: Overall Study

	Intent-To-Treat
STARTED	10
COMPLETED	10
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Intent-To-Treat	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Baseline Measures

	Intent-To-Treat
Number of Participants [units: participants]	10
Age [units: participants]
<=18 years	10
Between 18 and 65 years	0
>=65 years	0
Age [units: years] Mean ± Standard Deviation	4.8 ± 7.6
Gender [units: participants]
Female	3
Male	7
Region of Enrollment [units: participants]
United States	10

Outcome Measures

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1. Primary:

Number of Patients Alive (Survival)at 2 Years [ 2 years ]

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2. Secondary:

Number of Patients Alive at Three Years (Survival) [ 3 years ]

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3. Secondary:

Number of Patients With Succcessful Engraftment After Transplantation [ 42 Days ]

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4. Secondary:

Number of Patients With Grade 2-4 Acute Graft Versus Host Disease [ 100 Days ]

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5. Secondary:

Number of Patients With Chronic Graft Versus Host Disease [ 2 years ]

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6. Secondary:

Number of Patients With Disease Recurrence [ 2 years ]

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Measure Type	Secondary
Measure Title	Number of Patients With Disease Recurrence
Measure Description	Number of patients who exhibited disease recurrence at 2 years.
Time Frame	2 years
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
Intent-To-Treat	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values

	Intent-To-Treat
Number of Participants Analyzed [units: participants]	10
Number of Patients With Disease Recurrence [units: Participants]	0

No statistical analysis provided for Number of Patients With Disease Recurrence

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
Pharmacokinetic parameters in patients receiving 2 mg/kg/dose of busulfan twice daily was not performed. No data is available.

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

Responsible Party:	Masonic Cancer Center, University of Minnesota ( Paul Orchard, M.D. )
Study ID Numbers:	9504M09637, MT2000-18
Study First Received:	September 12, 2005
Results First Received:	June 18, 2009
Last Updated:	August 28, 2009
ClinicalTrials.gov Identifier:	NCT00176878 History of Changes
Health Authority:	United States: Institutional Review Board