Washington University in St. Louis

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

Arm	Description	Assigned 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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.