Stem Cell Transplant for Immunologic or Histiocytic Disorders
NCT ID: NCT00176865
Last Updated: 2017-12-28
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
19 participants
INTERVENTIONAL
2002-08-31
2014-08-31
Brief Summary
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Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).
Detailed Description
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After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Arm 1 - Matched sibling donor
Stem Cell Transplant: human leukocyte antigen (HLA) genotypic matched sibling donor and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)
Stem Cell Transplant
IV on Day 0
Fludarabine
30mg/m\^2 IV Day -7 through Day -3
Melphalan
140 mg/m\^2 IV Day -1
Anti-thymocyte globulin (ATG)
30 mg/kg IV Day -5 through Day -1
Campath 1H
0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
Cyclosporin A
2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
Mycophenolate mofetil
15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
Intravenous immunoglobulin (IVIG)
500 mg/kg IV weekly beginning on Day +7 until Day +100
Arm 2 - Matched unrelated donor
Stem Cell Transplant: HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)
Stem Cell Transplant
IV on Day 0
Fludarabine
30mg/m\^2 IV Day -7 through Day -3
Melphalan
140 mg/m\^2 IV Day -1
Anti-thymocyte globulin (ATG)
30 mg/kg IV Day -5 through Day -1
Campath 1H
0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
Cyclosporin A
2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
Mycophenolate mofetil
15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
Intravenous immunoglobulin (IVIG)
500 mg/kg IV weekly beginning on Day +7 until Day +100
Arm 3 - Mismatched double cord donors
Stem Cell Transplant: two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord) and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)
Stem Cell Transplant
IV on Day 0
Fludarabine
30mg/m\^2 IV Day -7 through Day -3
Melphalan
140 mg/m\^2 IV Day -1
Anti-thymocyte globulin (ATG)
30 mg/kg IV Day -5 through Day -1
Campath 1H
0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
Cyclosporin A
2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
Mycophenolate mofetil
15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
Intravenous immunoglobulin (IVIG)
500 mg/kg IV weekly beginning on Day +7 until Day +100
Interventions
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Stem Cell Transplant
IV on Day 0
Fludarabine
30mg/m\^2 IV Day -7 through Day -3
Melphalan
140 mg/m\^2 IV Day -1
Anti-thymocyte globulin (ATG)
30 mg/kg IV Day -5 through Day -1
Campath 1H
0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
Cyclosporin A
2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
Mycophenolate mofetil
15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
Intravenous immunoglobulin (IVIG)
500 mg/kg IV weekly beginning on Day +7 until Day +100
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients with histocytic disorders (hemophagocytic lymphohistiocytosis of any etiology and refractory Langerhans cell histiocytosis) who do not meet eligibility criteria for a myeloablative transplant procedure
* Patients with immunodeficiency disorders in whom residual immune function may not require a fully myeloablative preparative regimen or patient is ineligible for standard myeloablative preparative regimen (any form of severe combined immunodeficiency \[SCID\], or other immunodeficiency with T cell defect)
* Patients with immunodeficiency disorders that have had poor outcome with myeloablative stem cell transplants (including, but not limited to, common variable immunodeficiency \[CVID\], Wiskott Aldrich Syndrome \[WAS\] if \> 5 years of age, ataxia telangiectasia)
* Patients with immunodeficiencies or histocytic disorders that require a second stem cell transplant (SCT) for any reason
Exclusion Criteria
* Glomerular filtration rate (GFR)\<30% predicted
* Cardiac function \<50% normal by echocardiogram
* Serum creatinine \> 2x normal for age/weight
* Pregnant or lactating females
* Active serious infection that has not had an adequate course of therapy pre-SCT. Any patient with acquired immune deficiency syndrome (AIDS) or AIDS-related complex (ARC) or human immunodeficiency virus (HIV) seropositivity
35 Years
ALL
No
Sponsors
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Masonic Cancer Center, University of Minnesota
OTHER
Responsible Party
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Principal Investigators
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Angela Smith, MD
Role: PRINCIPAL_INVESTIGATOR
Masonic Cancer Center, University of Minnesota
Locations
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Masonic Cancer Center University of Minnesota
Minneapolis, Minnesota, United States
Countries
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Other Identifiers
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0207M29448
Identifier Type: OTHER
Identifier Source: secondary_id
MT2002-12
Identifier Type: -
Identifier Source: org_study_id