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Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia (AACD34)

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ClinicalTrials.gov Identifier: NCT00186797
Recruitment Status : Completed
First Posted : September 16, 2005
Last Update Posted : April 9, 2010
Sponsor:
Information provided by:
St. Jude Children's Research Hospital

Study Description
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Brief Summary:

This study is for patients with Severe Aplastic Anemia (SAA). A stem cell transplant from a genetically matched sibling donor can help or cure this disease in 85 to 100 percent of patients. Stem cells are immature blood cells that grow to become red blood cells, white blood cells or platelets. A genetic "match" means a brother or sister has same immune type (HLA type) as the patient. Unfortunately, few patients have a matched sibling donor. The chance of negative outcomes is much higher with other types of donors.

This study will test the success of a new approach to stem cell transplant for SAA. Patients in this study will receive drugs and radiation treatment to destroy their diseased bone marrow and to prepare them for stem cell transplant. Bone marrow is the tissue inside the bones where stem cells are made.Stem cells will be harvested from the blood or bone marrow of genetically matched unrelated donors or partially matched family donors. The stem cells will be filtered using a new device that is currently under study. The patients will receive large doses of the filtered stem cells (stem cell graft). Researchers want to find out how the study treatment affects patients, the disease, and the chances for survival.


Condition or disease Intervention/treatment Phase
Aplastic Anemia Procedure: Allogeneic stem cell transplant Drug: Fludarabine, Cyclophosphamide Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 28 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : December 2002
Actual Primary Completion Date : September 2005
Actual Study Completion Date : May 2007

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Arms and Interventions
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Outcome Measures
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Primary Outcome Measures :
  1. To learn the safety of stem cell transplantation in patients with unrelated donors or partially matched family donors
  2. To study the effects (good and bad) of this treatment on the patients, the aplastic anemia, and survival
  3. To learn how well the donor bone marrow grows in patients who receive the research treatment
  4. To learn how many patients need extra T-cells or extra stem cells from the donor to help the donor's blood stem cells grow

Eligibility Criteria
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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age < 21 years
  • Diagnosis of severe aplastic anemia. (As defined by at least 2 of the following: ANC < 500/μl, platelet count < 20,000/μl, and a reticulocyte count < 1% after correction for the hematocrit. In addition, the diagnostic bone marrow biopsy must contain less than 25% of the normal cellularity).
  • Patient must have failed one or more courses of immunosuppressive therapy that included ATG. As immunosuppression may take up to 6 months to demonstrate a response, patients must have been observed to have failed immunosuppression for a minimum of six months.
  • Absence of suitable HLA-matched sibling donor.
  • Negative serum pregnancy test for females with child bearing potential.
  • Patient/parent/guardian is able to provide informed consent.

Exclusion Criteria:

  • Patients with a life expectancy < 6 weeks.
  • Patients with severe renal disease (creatinine clearance < 40cc/min/1.73m2)
  • Patients with pre-existing severe restrictive pulmonary disease (FVC <40% of predicted)
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00186797


Locations
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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
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Principal Investigator: Paul Woodard, MD St. Jude Children's Research Hospital
More Information
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Additional Information:

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ClinicalTrials.gov Identifier: NCT00186797    
Other Study ID Numbers: AACD34
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: April 9, 2010
Last Verified: April 2010
Keywords provided by St. Jude Children's Research Hospital:
Aplastic Anemia
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Fludarabine
Immunosuppressive Agents
Immunologic Factors
 Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists