Immune Disorder HSCT Protocol
Primary Purpose
Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Sponsored by
About this trial
This is an interventional treatment trial for Immune Deficiency Disorders focused on measuring Immune deficiency, Immune disorders, Immune dysregulatory, Reduced Intensity, Alemtuzumab, Campath
Eligibility 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
Sites / Locations
- Washington UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Preparative
Arm Description
Outcomes
Primary Outcome Measures
Number of participants with donor engraftment
Secondary Outcome Measures
Major Transplant Related Toxicities
Time to neutrophil recovery
Number of patient with acute GVHD
Number of participants with infectious complications
Time to immune reconstitution
Overall survival
Time to platelet recovery
Number of patients with chronic GVHD
Disease free survival
Full Information
NCT ID
NCT01821781
First Posted
March 19, 2013
Last Updated
August 1, 2023
Sponsor
Washington University School of Medicine
1. Study Identification
Unique Protocol Identification Number
NCT01821781
Brief Title
Immune Disorder HSCT Protocol
Official Title
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 2013 (undefined)
Primary Completion Date
March 2027 (Anticipated)
Study Completion Date
March 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Washington University School of Medicine
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
Keywords
Immune deficiency, Immune disorders, Immune dysregulatory, Reduced Intensity, Alemtuzumab, Campath
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Preparative
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Intervention Description
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 Measure Information:
Title
Number of participants with donor engraftment
Time Frame
1 year post transplant
Secondary Outcome Measure Information:
Title
Major Transplant Related Toxicities
Time Frame
1 years post transplant
Title
Time to neutrophil recovery
Time Frame
within 100 days post transplant
Title
Number of patient with acute GVHD
Time Frame
180 days post transplant
Title
Number of participants with infectious complications
Time Frame
2 years post transplant
Title
Time to immune reconstitution
Time Frame
2 years post transplant
Title
Overall survival
Time Frame
2 years post transplant
Title
Time to platelet recovery
Time Frame
within 100 days post transplant
Title
Number of patients with chronic GVHD
Time Frame
2 years post transplant
Title
Disease free survival
Time Frame
2 years post transplant
10. Eligibility
Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jeffrey Bednarski, MD
Phone
314-454-6018
Email
Bednarski_J@kids.wustl.edu
First Name & Middle Initial & Last Name & Degree
Stephanie Hyde, CCRP
Phone
314-286-1180
Email
stephanie.day@wustl.edu
First Name & Middle Initial & Last Name & Degree
Jeffrey Bednarski, MD
12. IPD Sharing Statement
Citations:
PubMed Identifier
31599480
Citation
Bhatt ST, Schulz G, Hente M, Slater A, Murray L, Shenoy S, Bednarski JJ. A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease. Pediatr Blood Cancer. 2020 Jan;67(1):e28030. doi: 10.1002/pbc.28030. Epub 2019 Oct 10.
Results Reference
derived
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Immune Disorder HSCT Protocol
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