NCT00176865

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Completed Phase 2 Results posted Last updated 3 December 2017

What this trial tests

Phase 2 trial testing Stem Cell Transplant in Hemophagocytic Lymphohistiocytosis in 19 participants. Completed in 1 August 2014.

Timeline

1 August 2002

Primary endpoint
1 August 2012

1 August 2014

Quick facts

Lead sponsor	Masonic Cancer Center, University of Minnesota
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	19
Start date	1 August 2002
Primary completion	1 August 2012
Estimated completion	1 August 2014
Sites	1 location across United States

Drugs / interventions tested

• Stem Cell Transplant
• Fludarabine (FLUDARABINE) — full drug profile →
• Melphalan (melphalan) — full drug profile →
• Anti-thymocyte globulin (ATG)
• Campath 1H
• Cyclosporin A — full drug profile →
• Mycophenolate mofetil (MYCOPHENOLATE MOFETIL) — full drug profile →
• Intravenous immunoglobulin (IVIG) — full drug profile →

Conditions studied

• Hemophagocytic Lymphohistiocytosis — all drugs for Hemophagocytic Lymphohistiocytosis →
• X-Linked Lymphoproliferative Disorders — all drugs for X-Linked Lymphoproliferative Disorders →
• Chediak-Higashi Syndrome — all drugs for Chediak-Higashi Syndrome →
• Griscelli Syndrome — all drugs for Griscelli Syndrome →

Sponsor

Masonic Cancer Center, University of Minnesota

Who can join

Under 35, any sex, with Hemophagocytic Lymphohistiocytosis or X-Linked Lymphoproliferative Disorders. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of Subjects With Mixed Chimerism
  Time frame: Day 100
  \>10% Donor Cells at Day 100

Sponsor's own description

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).

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Hemophagocytic Syndrome and Critical Illness: New Insights into Diagnosis and Management.
   Tothova Z, Berliner N. · · 2015 · cited 70× · PMID 24407034 · DOI 10.1177/0885066613517076
2. Hemophagocytic lymphohistiocytosis: current treatment advances, emerging targeted therapy and underlying mechanisms.
   Wu Y, Sun X, Kang K, Yang Y, et al · · 2024 · cited 47× · PMID 39511607 · DOI 10.1186/s13045-024-01621-x
3. Proliferation through activation: hemophagocytic lymphohistiocytosis in hematologic malignancy.
   Vick EJ, Patel K, Prouet P, Martin MG. · · 2017 · cited 31× · PMID 29296722 · DOI 10.1182/bloodadvances.2017005561