Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00354419
• Date of registration: 19/07/2006
• Prospective Registration: No
• Primary sponsor: Fred Hutchinson Cancer Research Center
• Public title: Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant
• Scientific title: A Dose Finding Study of Total Body Irradiation for Conditioning Patients With Severe Aplastic Anemia Transplanted With Umbilical Cord Blood
• Date of first enrolment: February 2006
• Target sample size: 30
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT00354419
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Ann Woolfrey
• Affiliation: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Key inclusion & exclusion criteria

Inclusion Criteria:

- Life-threatening marrow failure of nonmalignant etiology meeting two of the three
following criteria: granulocytes < 500/mm^3; a corrected reticulocyte count < 1%;
platelet count < 20,000/mm^3

- Failure to respond to the best available immunosuppressive treatment protocol by 75
days after initiation of therapy

- Lack of an HLA-identical family member or closely matched (9 or 10 of 10 HLA-locus
match) unrelated marrow donor

- DONOR: Unrelated UCB unit matched for at least 4 of 6 loci

- DONOR: Related UCB unit matched for at least 3 of 6 lock

- Selection of the UCB unit(s) will be based upon matching or mismatching at HLA-A, B
antigen level and DRB1 allele level typing; while HLA-C antigen/allele and HLA-DQB1
antigen/allele level typing are not considered in the matching criteria, if available
each may be used to optimize unit selection

- Multiple UCB units are allowed to provide sufficient cell dose; when multiple units
are selected, the following rules apply: a) the UCB unit with the least HLA disparity
will be selected first (i.e., selection priority is 6/6 match > 5/6 match > 4/6
match), additional UCB units may be selected to increase cell dose; b) UCB units must
be matched to each other for at least 4 of 6 loci; c) each unit must contain at least
1.5 x 10^7 Total Nucleated Cells per kg recipient weight; d) the total cell dose of
the combined units must be at least 3.0 x 10^7 Total Nucleated Cells per kg recipient
weight

Exclusion Criteria:

- Severe disease other than aplastic anemia that would severely limit the probability
of survival during the graft procedure; patients who present with active fungal
infections must be treated to resolve this problem before beginning the conditioning
regimen

- HIV seropositive patients

- Patients who have developed clonal cytogenetic abnormalities or a myelodysplastic
syndrome (these patients will be considered in separate protocols for myelodysplastic
syndrome, etc.)

- Patients with paroxysmal nocturnal hemoglobinuria or Fanconi anemia

- Patients > 40 years of age

- Related or unrelated cord blood units with < 1.5 x 10^7 Total Nucleated Cells per kg
recipient weight

- Related or unrelated cord blood units without full testing and negative results for
hepatitis A, B, C, HIV, HTLV-1, CMV viruses



Age minimum: N/A
Age maximum: 40 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: mycophenolate mofetil

Drug: cyclosporine

Drug: cyclophosphamide

Procedure: umbilical cord blood transplantation

Biological: filgrastim

Procedure: bone marrow aspiration

Radiation: total-body irradiation

Biological: anti-thymocyte globulin

Genetic: DNA analysis

Primary Outcome(s)

Toxicity [Time Frame: Yes]

Secondary Outcome(s)

Engraftment [Time Frame: No]

Secondary ID(s)

2030.00

NCI-2010-00191

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Cancer Institute (NCI)