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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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21 August 2023 |
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Main ID: |
NCT01821781 |
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Date of registration:
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19/03/2013 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Immune Disorder HSCT Protocol
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Scientific title:
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A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime |
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Date of first enrolment:
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March 2013 |
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Target sample size:
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20 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT01821781 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Jeffrey Bednarski, MD |
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Address:
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Telephone:
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314-454-6018 |
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Email:
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Bednarski_J@kids.wustl.edu |
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Affiliation:
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Key inclusion & exclusion criteria
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Inclusion Criteria:
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- Performance status >/= 40
- DLCO >/= 40%
- LVEF >/=40% or LVSF >/=26%
- Serum creatinine < 2x ULN
- Liver enzymes
- 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
Age minimum:
N/A
Age maximum:
21 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Common Variable Immune Deficiency
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Hemophagocytic Lymphohistiocytosis
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Hyper-IgM
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X-linked Agammaglobulinemia
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Autoimmune Lymphoproliferative Syndrome
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IPEX
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Chronic Granulomatous Disease
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Severe Combined Immunodeficiency
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Immune Deficiency Disorders
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Immune Dysregulatory Disorders
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Chediak-Higashi Syndrome
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DiGeorge Syndrome
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X-linked Lymphoproliferative Syndrome
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Wiskott-Aldrich Syndrome
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Intervention(s)
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Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
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Primary Outcome(s)
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Number of participants with donor engraftment
[Time Frame: 1 year post transplant]
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Secondary Outcome(s)
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Number of participants with infectious complications
[Time Frame: 2 years post transplant]
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Overall survival
[Time Frame: 2 years post transplant]
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Time to platelet recovery
[Time Frame: within 100 days post transplant]
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Number of patients with chronic GVHD
[Time Frame: 2 years post transplant]
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Major Transplant Related Toxicities
[Time Frame: 1 years post transplant]
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Number of patient with acute GVHD
[Time Frame: 180 days post transplant]
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Disease free survival
[Time Frame: 2 years post transplant]
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Time to immune reconstitution
[Time Frame: 2 years post transplant]
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Time to neutrophil recovery
[Time Frame: within 100 days post transplant]
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Secondary ID(s)
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201301135
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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