Know Cancer

or
forgot password

A Dose Finding Study of Total Body Irradiation for Conditioning Patients With Severe Aplastic Anemia Transplanted With Umbilical Cord Blood


Phase 1
N/A
40 Years
Not Enrolling
Both
Aplastic Anemia

Thank you

Trial Information

A Dose Finding Study of Total Body Irradiation for Conditioning Patients With Severe Aplastic Anemia Transplanted With Umbilical Cord Blood


OBJECTIVES:

I. The objective of this study is to determine the lowest dose of total body irradiation
combined with cyclophosphamide and antithymocyte globulin that will achieve sustained
engraftment in patients with severe aplastic anemia transplanted with unrelated umbilical
cord blood.

OUTLINE: This is a dose-escalation study of total-body irradiation (TBI).

MYELOABLATIVE CONDITIONING REGIMEN: Patients receive cyclophosphamide IV on days -7 to -4,
-6 to -3, or -5 to -2 and antithymocyte globulin IV on days -6 to -4, -5 to -3, or -4 to -2.

TBI: Patients undergo TBI twice daily on days -3, -2, and/or -1.

UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT): Patients undergo UCBT on day 0. Patients
receive filgrastim (G-CSF) IV or subcutaneously beginning on day 1 and continuing until
blood counts recover.

GRAFT-VS-HOST DISEASE (GVHD) PROPHYLAXIS: Patients receive cyclosporine IV or orally (twice
daily for patients >= 6 years of age or 3 times daily for patients < 6 years of age) on days
-1 to +180 and mycophenolate mofetil IV or orally (twice daily for patients >= 50 kg or 3
times daily for patients < 50 kg) beginning 4 hours after UCBT and continuing until
approximately day +0.

After completion of study therapy, patients are followed periodically.


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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Toxicity

Safety Issue:

Yes

Principal Investigator

Ann Woolfrey

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Authority:

United States: Institutional Review Board

Study ID:

2030.00

NCT ID:

NCT00354419

Start Date:

February 2006

Completion Date:

Related Keywords:

  • Aplastic Anemia
  • Anemia
  • Anemia, Aplastic

Name

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumSeattle, Washington  98109