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Immune Disorder HSCT Protocol

This study is currently recruiting participants.
Verified November 2017 by Washington University School of Medicine
Sponsor:
ClinicalTrials.gov Identifier:
NCT01821781
First Posted: April 1, 2013
Last Update Posted: November 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Washington University School of Medicine
  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.

Condition Intervention Phase
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 Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (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 Study Completion Date: March 2021
Estimated Primary Completion Date: March 2019 (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

  Eligibility

Information from the National Library of Medicine

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

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01821781


Locations
United States, Missouri
Washington University Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Jeffrey Bednarski, MD    314-454-6018    Bednarski_J@kids.wustl.edu   
Contact: Lisa Murray, CCRP    314-454-4240    Murray_L@kids.wustl.edu   
Principal Investigator: Jeffrey Bednarski, MD         
United States, Texas
Methodist Heathcare Recruiting
San Antonio, Texas, United States, 78229
Contact: Troy Quigg, DO, MS       Troy.Quigg@mhshealth.com   
Sponsors and Collaborators
Washington University School of Medicine
  More Information

Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01821781     History of Changes
Other Study ID Numbers: 201301135
First Submitted: March 19, 2013
First Posted: April 1, 2013
Last Update Posted: November 21, 2017
Last Verified: November 2017

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

Additional relevant MeSH terms:
Autoimmune Lymphoproliferative Syndrome
Disease
Syndrome
Immunologic Deficiency Syndromes
Granulomatous Disease, Chronic
Severe Combined Immunodeficiency
Lymphohistiocytosis, Hemophagocytic
Wiskott-Aldrich Syndrome
DiGeorge Syndrome
Agammaglobulinemia
Chediak-Higashi Syndrome
Genetic Diseases, X-Linked
Common Variable Immunodeficiency
Lymphoproliferative Disorders
Pathologic Processes
Immune System Diseases
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hemorrhagic Disorders
Lymphopenia