NCT00354419

Brief Summary

RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening. PURPOSE: This phase I trial is studying the side effects and best dose of total-body irradiation when given together with cyclophosphamide and antithymocyte globulin in treating patients with severe aplastic anemia undergoing umbilical cord blood transplant.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2006

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

July 19, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 20, 2006

Completed
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
Last Updated

January 5, 2011

Status Verified

January 1, 2011

Enrollment Period

4.8 years

First QC Date

July 19, 2006

Last Update Submit

January 3, 2011

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

  • Toxicity

Secondary Outcomes (1)

  • Engraftment

Study Arms (1)

Arm I

EXPERIMENTAL

See Detailed Description

Radiation: total-body irradiationDrug: cyclophosphamideBiological: anti-thymocyte globulinDrug: cyclosporineProcedure: umbilical cord blood transplantationDrug: mycophenolate mofetilProcedure: bone marrow aspirationGenetic: DNA analysisBiological: filgrastim

Interventions

total-body irradiationRADIATION

Undergo radiotherapy

Also known as: TBI
Arm I
cyclophosphamideDRUG

Given IV

Also known as: CPM, CTX, Cytoxan, Endoxan, Endoxana, Enduxan
Arm I
anti-thymocyte globulinBIOLOGICAL

Given IV

Also known as: ATG, ATGAM, lymphocyte immune globulin, Thymoglobulin
Arm I
cyclosporineDRUG

Given IV

Also known as: 27-400, ciclosporin, cyclosporin, cyclosporin A, CYSP, Sandimmune
Arm I
umbilical cord blood transplantationPROCEDURE

Undergo transplantation

Also known as: cord blood transplantation, transplantation, umbilical cord blood, UCB transplantation
Arm I
mycophenolate mofetilDRUG

Given IV or orally

Also known as: Cellcept, MMF
Arm I
bone marrow aspirationPROCEDURE

Correlative study

Arm I
DNA analysisGENETIC

Correlative study

Arm I
filgrastimBIOLOGICAL

Given IV or SC

Also known as: G-CSF, granulocyte colony-stimulating factor, Neupogen, r-metHuG-CSF, Recombinant Methionyl Human Granulocyte Colony Stimulating Factor
Arm I

Eligibility Criteria

AgeUp to 40 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Whole-Body IrradiationCyclophosphamideAntilymphocyte SerumthymoglobulinCyclosporineCyclosporinsCord Blood Stem Cell TransplantationTransplantationMycophenolic AcidFilgrastimGranulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Intervention Hierarchy (Ancestors)

RadiotherapyTherapeuticsInvestigative TechniquesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsImmune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex MixturesPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesStem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapySurgical Procedures, OperativeCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipidsColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsBiological Factors

Study Officials

  • Ann Woolfrey

    Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

July 19, 2006

First Posted

July 20, 2006

Study Start

February 1, 2006

Primary Completion

December 1, 2010

Last Updated

January 5, 2011

Record last verified: 2011-01

Locations

US(1)