Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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16 December 2017 |
Main ID: |
NCT00301834 |
Date of registration:
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09/03/2006 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
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Scientific title:
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Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia |
Date of first enrolment:
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January 2005 |
Target sample size:
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35 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT00301834 |
Study type:
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Interventional |
Study design:
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Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Morton J. Cowan, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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University of California, San Francisco |
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Key inclusion & exclusion criteria
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DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following hematologic conditions:
- Aplastic anemia with marrow aplasia, meeting all of the following criteria:
- Absolute neutrophil count < 500/mm^3
- Platelet and/or red cell transfusion dependent
- Chronic aplastic anemia, meeting all of the following criteria:
- Transfusion dependent
- Unresponsive to immunosuppressive therapy
- Alternative matched unrelated donor has been identified
- Congenital marrow failure syndrome, including any of the following (with closely
matched related or unrelated donor):
- Primary red cell aplasia (Diamond-Blackfan syndrome)
- Congenital neutropenia (Kostmann's syndrome)
- Amegakaryocytic thrombocytopenia
- Congenital dyserythropoietic anemias
- Other severe acquired cytopenias in which a transplantation using a combined
busulfan/cyclophosphamide conditioning regimen is indicated
- Hemoglobinopathy (with closely matched related or unrelated donor)
- ß-thalassemia major
- Sickle cell anemia
- Hemoglobin E/ß-thalassemia
- Severe immunodeficiency disease
- Chediak-Higashi disease
- Wiskott-Aldrich syndrome
- Combined immunodeficiency disease (Nezelof's)
- Hyper immunoglobulin M (IgM) syndrome
- Bare lymphocyte syndrome
- Chronic granulomatous disease
- Familial erythrohemophagocytic lymphohistiocytosis
- Other stem cell defects (e.g., osteopetrosis)
- Severe immune dysregulation/autoimmune disorders
- Achieved a transient response to prior immunosuppressive therapy
- Chronic myelogenous leukemia
- Disease in first chronic phase
- Acute myeloid leukemia
- Disease in first remission
- Myelodysplastic syndromes
- Inborn errors of metabolism
- Histiocytosis
- No severe combined immunodeficiency disease
- Matched related or unrelated donor available by high resolution DNA typing
- Related donor, meeting both of the following criteria:
- Matched at both human leukocyte antigen (HLA)-Drß1 alleles
- No more than 1 mismatch at the 4 HLA-A and -B alleles
- Unrelated donor, meeting 1 of the following criteria:
- Marrow matched at both HLA-Drß1 alleles AND no more than 1 mismatch at the 4
HLA-A and -B alleles
- Umbilical cord blood matched at 5/6 HLA-A, -B, and -DRß1 alleles with at
least 1 -DRß1 match AND there are = 3x10^5 CD34+ (Cluster of differentiation
34-positive) cells per kg body weight of recipient available at the time of
cryopreservation
PATIENT CHARACTERISTICS:
- Cardiac ejection fraction = 27%
- Creatinine clearance = 50 mL/min by 24-hour urine collection or glomerular filtration
rate
- DLCO (diffusion capacity of lung for carbon monoxide) = 50% of predicted (corrected
for anemia/lung volume)
PRIOR CONCURRENT THERAPY:
- No prior transplantation for leukemia from which patient remains engrafted and
alemtuzumab is not needed as part of the conditioning regimen
Age minimum:
N/A
Age maximum:
21 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Myelodysplastic Syndromes
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Diamond-blackfan Anemia
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Severe Congenital Neutropenia
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Leukemia
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Congenital Amegakaryocytic Thrombocytopenia
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Intervention(s)
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Drug: methylprednisolone
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Drug: fludarabine phosphate
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Procedure: allogeneic hematopoietic stem cell transplantation
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Biological: alemtuzumab
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Drug: busulfan
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Drug: cyclosporine
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Drug: methotrexate
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Procedure: allogeneic bone marrow transplantation
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Procedure: peripheral blood stem cell transplantation
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Procedure: umbilical cord blood transplantation
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Primary Outcome(s)
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Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation
[Time Frame: 6 weeks post-transplant]
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Secondary Outcome(s)
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Cytomegalovirus (CMV) Viral Infection and Disease Symptoms
[Time Frame: Up to one year post-transplant]
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Toxicity Grade = 3 From Start of Conditioning Through the First Year Post Transplantation
[Time Frame: 1 year post-transplantation]
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Disease-free Survival With Correction of Disease at One Year Post Transplantation
[Time Frame: 1 year post-transplantation]
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Treatment-related Mortality at 100 Days and 1 Year Post Transplantation
[Time Frame: 100 days and 1 year]
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Secondary ID(s)
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UCSF-00452
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CDR0000462406
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UCSF-H411-25738-02
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UCSF-04152
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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