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Immune Disorder HSCT Protocol

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2016 by Washington University School of Medicine
Information provided by (Responsible Party):
Washington University School of Medicine Identifier:
First received: March 19, 2013
Last updated: November 1, 2016
Last verified: November 2016
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.

Condition Intervention Phase
Immune Deficiency Disorders
Severe Combined Immunodeficiency
Chronic Granulomatous Disease
X-linked Agammaglobulinemia
Wiskott-Aldrich Syndrome
DiGeorge Syndrome
Chediak-Higashi Syndrome
Common Variable Immune Deficiency
Immune Dysregulatory Disorders
Hemophagocytic Lymphohistiocytosis
Autoimmune Lymphoproliferative Syndrome
X-linked Lymphoproliferative Syndrome
Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

Resource links provided by NLM:

Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Number of participants with donor engraftment [ Time Frame: 1 year post transplant ]

Secondary Outcome Measures:
  • Major Transplant Related Toxicities [ Time Frame: 1 years post transplant ]
  • Time to neutrophil recovery [ Time Frame: within 100 days post transplant ]
  • Number of patient with acute GVHD [ Time Frame: 180 days post transplant ]
  • Number of participants with infectious complications [ Time Frame: 2 years post transplant ]
  • Time to immune reconstitution [ Time Frame: 2 years post transplant ]
  • Overall survival [ Time Frame: 2 years post transplant ]
  • Time to platelet recovery [ Time Frame: within 100 days post transplant ]
  • Number of patients with chronic GVHD [ Time Frame: 2 years post transplant ]
  • Disease free survival [ Time Frame: 2 years post transplant ]

Estimated Enrollment: 20
Study Start Date: March 2013
Estimated Primary Completion Date: March 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • </= 21 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01821781

United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 63110
Contact: Jeffrey Bednarski, MD    314-454-6018   
Contact: Lisa Murray, CCRP    314-454-4240   
Principal Investigator: Jeffrey Bednarski, MD         
United States, Texas
Methodist Heathcare Recruiting
San Antonio, Texas, United States, 78229
Contact: Troy Quigg, DO, MS   
Sponsors and Collaborators
Washington University School of Medicine
  More Information

Responsible Party: Washington University School of Medicine Identifier: NCT01821781     History of Changes
Other Study ID Numbers: 201301135
Study First Received: March 19, 2013
Last Updated: November 1, 2016

Keywords provided by Washington University School of Medicine:
Immune deficiency
Immune disorders
Immune dysregulatory
Reduced Intensity

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Immunologic Deficiency Syndromes
Lymphohistiocytosis, Hemophagocytic
Wiskott-Aldrich Syndrome
Autoimmune Lymphoproliferative Syndrome
Chediak-Higashi Syndrome
DiGeorge Syndrome
Severe Combined Immunodeficiency
Lymphoproliferative Disorders
Common Variable Immunodeficiency
Genetic Diseases, X-Linked
Pathologic Processes
Immune System Diseases
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Leukocyte Disorders
Genetic Diseases, Inborn
Phagocyte Bactericidal Dysfunction
Autoimmune Diseases
Immunoproliferative Disorders processed this record on May 25, 2017