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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: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT00301834
Date of registration: 09/03/2006
Prospective Registration: No
Primary sponsor: University of California, San Francisco
Public title: Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
Scientific title: 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: January 2005
Target sample size: 35
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT00301834
Study type:  Interventional
Study design:  Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Morton J. Cowan, MD
Address: 
Telephone:
Email:
Affiliation:  University of California, San Francisco
Key inclusion & exclusion criteria

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
Health Condition(s) or Problem(s) studied
Myelodysplastic Syndromes
Diamond-blackfan Anemia
Severe Congenital Neutropenia
Leukemia
Congenital Amegakaryocytic Thrombocytopenia
Intervention(s)
Drug: methylprednisolone
Drug: fludarabine phosphate
Procedure: allogeneic hematopoietic stem cell transplantation
Biological: alemtuzumab
Drug: busulfan
Drug: cyclosporine
Drug: methotrexate
Procedure: allogeneic bone marrow transplantation
Procedure: peripheral blood stem cell transplantation
Procedure: umbilical cord blood transplantation
Primary Outcome(s)
Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation [Time Frame: 6 weeks post-transplant]
Secondary Outcome(s)
Cytomegalovirus (CMV) Viral Infection and Disease Symptoms [Time Frame: Up to one year post-transplant]
Toxicity Grade = 3 From Start of Conditioning Through the First Year Post Transplantation [Time Frame: 1 year post-transplantation]
Disease-free Survival With Correction of Disease at One Year Post Transplantation [Time Frame: 1 year post-transplantation]
Treatment-related Mortality at 100 Days and 1 Year Post Transplantation [Time Frame: 100 days and 1 year]
Secondary ID(s)
UCSF-00452
CDR0000462406
UCSF-H411-25738-02
UCSF-04152
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
National Cancer Institute (NCI)
Ethics review
Results
Results available: Yes
Date Posted: 16/05/2013
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00301834
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