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Stem Cell Transplant for Bone Marrow Failure Syndromes

NCT ID: NCT00176878

Last Updated: 2017-12-28

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-06-30

Study Completion Date

2009-03-31

Brief Summary

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The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

Detailed Description

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Prior to transplantation, subjects will receive the drugs busulfan (orally or through the catheter), as well as fludarabine and anti-thymocyte globulin (ATG) via the catheter. Busulfan, fludarabine and ATG will be given with Total Lymphoid Irradiation (TLI) to help the new donor bone marrow take and grow after transplantation.

Those patients receiving donor marrow will have the T cells (a type of white blood cell in the donor marrow) removed to lower the risk that the new marrow will react to their body, a condition called Graft-Versus-Host-Disease (GVHD). After bone marrow transplantation, subjects will receive drugs to help prevent GVHD, including cyclosporin and mycophenolate mofetil (MMF).

Blood samples are taken at day 28, day 60, day 100, 1 year and as required by medical status yearly for five years after transplant to evaluate how well the new marrow is growing. A bone marrow biopsy is required at day 21, at day 100 and 1 year.

Conditions

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Diamond-Blackfan Anemia Kostmann's Neutropenia Shwachman-Diamond Syndrome

Keywords

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Stem cell transplant T-cell depletion TLI bone marrow failure disorders

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Bone Marrow Failure Disorders

Patients with Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome

Group Type EXPERIMENTAL

Stem cell transplant

Intervention Type PROCEDURE

Stem cell transplant on Day 0 - healthy marrow from an unrelated individual. A minimum of 1.0 x 10\^9/kg nucleated cells/kg ideal body weight will be collected with a goal of 2.0 x 10\^9/kg.

Fludarabine monophosphate

Intervention Type DRUG

fludarabine 175 mg/m\^2 (total) on Days -6 through -3.

Total lymphoid irradiation

Intervention Type PROCEDURE

Dose 500 cGy radiation therapy to specific areas of the body

Busulfan

Intervention Type DRUG

Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),

anti-thymocyte globulin

Intervention Type BIOLOGICAL

anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter

Interventions

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Stem cell transplant

Stem cell transplant on Day 0 - healthy marrow from an unrelated individual. A minimum of 1.0 x 10\^9/kg nucleated cells/kg ideal body weight will be collected with a goal of 2.0 x 10\^9/kg.

Intervention Type PROCEDURE

Fludarabine monophosphate

fludarabine 175 mg/m\^2 (total) on Days -6 through -3.

Intervention Type DRUG

Total lymphoid irradiation

Dose 500 cGy radiation therapy to specific areas of the body

Intervention Type PROCEDURE

Busulfan

Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),

Intervention Type DRUG

anti-thymocyte globulin

anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter

Intervention Type BIOLOGICAL

Other Intervention Names

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BMT Fludara TLI Busulfex ATG

Eligibility Criteria

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Inclusion Criteria

* Patients eligible for transplantation under this protocol will be \<35 years of age, and will be diagnosed with:

* a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia.
* Diamond Blackfan Anemia:

* Patients must show evidence of steroid resistance requiring equivalent of \>6 transfusions yearly despite steroid therapy.
* Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation.
* Kostmann's Neutropenia, Shwachman-Diamond syndrome:

* Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia.
* Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) \>750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (\<4 /year).

Exclusion Criteria

* Patients \>35 years of age
* Karnofsky score \<70%
* Hepatic dysfunction as determined by bilirubin \>3.0, ALT \>150, or active hepatitis
* Pulmonary function tests with forced volume vital capacity (FVC) and forced expiratory volume (FEV) \<70%; O2 saturation \<94%
* Renal dysfunction with glomerular filtration rate (GFR) \<30% of predicted.
* Cardiac compromise, with left ejection fraction \<45%.
* Severe, stable neurologic impairment.
* Human immunodeficiency virus (HIV) positivity.
* Pregnant or lactating females
Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Paul Orchard, MD

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota Medical Center

Locations

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University of Minnesota Medical Center

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Other Identifiers

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9504M09637

Identifier Type: OTHER

Identifier Source: secondary_id

MT2000-18

Identifier Type: -

Identifier Source: org_study_id

NCT00005895

Identifier Type: -

Identifier Source: nct_alias