Stem Cell Transplant for Bone Marrow Failure Syndromes

This study has been completed.

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

First Received on September 12, 2005. Last Updated on September 30, 2010 History of Changes

Results First Received: June 18, 2009

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Diamond-Blackfan Anemia Kostmann's Neutropenia Shwachman-Diamond Syndrome
Interventions:	Procedure: Stem cell transplant Drug: Fludarabine monophosphate Procedure: Total lymphoid irradiation Drug: Busulfan Biological: anti-thymocyte 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
Bone Marrow Failure Patients	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.

Participant Flow: Overall Study

	Bone Marrow Failure Patients
STARTED	10
COMPLETED	10
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Bone Marrow Failure Patients	All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.

Baseline Measures

	Bone Marrow Failure Patients
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 [ Time Frame: 2 years ]

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2. Secondary:

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

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3. Secondary:

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

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4. Secondary:

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

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5. Secondary:

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

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6. Secondary:

Number of Patients With Disease Recurrence [ Time Frame: 2 years ]

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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
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:	Paul Orchard, M.D., Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:	NCT00176878 History of Changes
Obsolete Identifiers:	NCT00005895
Other Study ID Numbers:	MT2000-18, 9504M09637
Study First Received:	September 12, 2005
Results First Received:	June 18, 2009
Last Updated:	September 30, 2010
Health Authority:	United States: Institutional Review Board