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Immune Disorder HSCT Protocol

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ClinicalTrials.gov Identifier: NCT01821781
Recruitment Status : Recruiting
First Posted : April 1, 2013
Last Update Posted : April 27, 2022
Information provided by (Responsible Party):
Washington University School of Medicine

Brief Summary:
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 or disease Intervention/treatment 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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
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
Study Start Date : March 2013
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : March 2024

Arm Intervention/treatment
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

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

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

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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

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

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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         
Sponsors and Collaborators
Washington University School of Medicine
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01821781    
Other Study ID Numbers: 201301135
First Posted: April 1, 2013    Key Record Dates
Last Update Posted: April 27, 2022
Last Verified: April 2022
Keywords provided by Washington University School of Medicine:
Immune deficiency
Immune disorders
Immune dysregulatory
Reduced Intensity
Additional relevant MeSH terms:
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DiGeorge Syndrome
Chediak-Higashi Syndrome
Granulomatous Disease, Chronic
Lymphohistiocytosis, Hemophagocytic
Wiskott-Aldrich Syndrome
Autoimmune Lymphoproliferative Syndrome
Lymphoproliferative Disorders
Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Common Variable Immunodeficiency
Pathologic Processes
Immune System Diseases
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases
Primary Immunodeficiency Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hemorrhagic Disorders