Immune Disorder HSCT Protocol

Purpose

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Conditions

  • Immune Deficiency Disorders
  • Severe Combined Immunodeficiency
  • Chronic Granulomatous Disease
  • X-linked Agammaglobulinemia
  • Wiskott-Aldrich Syndrome
  • Hyper-IgM
  • DiGeorge Syndrome
  • Chediak-Higashi Syndrome
  • Common Variable Immune Deficiency
  • Immune Dysregulatory Disorders
  • Hemophagocytic Lymphohistiocytosis
  • IPEX
  • Autoimmune Lymphoproliferative Syndrome
  • X-linked Lymphoproliferative Syndrome

Eligibility

Eligible Ages
Under 21 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • </= 28 years of age - Performance status >/= 40 - DLCO >/= 40% - LVEF >/=40% or LVSF >/=26% - Serum creatinine < 2x ULN - Liver enzymes </= 5x ULN - Negative pregnancy test - Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord)

Exclusion Criteria

  • Known diagnosis of HIV I/II - Pregnant or breastfeeding - Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab - Uncontrolled viral infection within 1 week prior to starting alemtuzumab

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Preparative
  • Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
    Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF

Recruiting Locations

Washington University in St. Louis and nearby locations

Washington University
St Louis 4407066, Missouri 4398678 63110
Contact:
Jeffrey Bednarski, MD
314-454-6018
Bednarski_J@kids.wustl.edu

More Details

NCT ID
NCT01821781
Status
Recruiting
Sponsor
Washington University School of Medicine