Phase 2
N=19
Stem Cell Transplant for Immunologic or Histiocytic Disorders
Hemophagocytic Lymphohistiocytosis · X-Linked Lymphoproliferative Disorders · Chediak-Higashi Syndrome · Griscelli Syndrome · Immunologic Deficiency Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00176865 ↗Enrolled (actual)
19
Serious AEs
5.3%
Results posted
Apr 2017
Primary outcome: Primary: Number of Subjects With Mixed Chimerism — 3; 8; 4 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Stem Cell Transplant (Procedure); Fludarabine (Drug); Melphalan (Drug); Anti-thymocyte globulin (ATG) (Drug); Campath 1H (Drug); Cyclosporin A (Drug); Mycophenolate mofetil (Drug); Intravenous immunoglobulin (IVIG) (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Aug 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Mixed Chimerism |
3; 8; 4 | — |
| SECONDARY Percentage of Donor Chimerism at 100 Days |
96.5; 75.5; 100 | — |
| SECONDARY Percentage of Donor Chimerism at 180 Days |
88.9; 73.3; 90.5 | — |
| SECONDARY Percentage of Donor Chimerism at 365 Days |
81.9; 78.6; 91.7 | — |
| SECONDARY Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD) |
1; 3; 0 | — |
| SECONDARY Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD) |
1; 1; 0 | — |
| SECONDARY Incidence of Chronic Graft Versus Host Disease (cGVHD) |
1; 0; 0 | — |
| SECONDARY Number of Subjects Alive at 100 Days |
3; 8; 4 | — |
| SECONDARY Number of Subjects Alive at One Year |
3; 7; 3 | — |
| SECONDARY Compare Quality of Life (QOL) |
— | — |
Summary
This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD).
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).
Eligibility Criteria
Inclusion Criteria
Patients with immunodeficiencies or histiocytic disorders 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
- 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
- Karnofsky or Lansky performance score 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
Data sourced from ClinicalTrials.gov (NCT00176865). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.