Chronic Granulomatous Disease Clinical Trial
Official title:
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
NCT number | NCT01821781 |
Other study ID # | 201301135 |
Secondary ID | |
Status | Recruiting |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | March 2013 |
Est. completion date | March 2027 |
Verified date | August 2023 |
Source | Washington University School of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Recruiting |
Enrollment | 20 |
Est. completion date | March 2027 |
Est. primary completion date | March 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 21 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Washington University | Saint Louis | Missouri |
Lead Sponsor | Collaborator |
---|---|
Washington University School of Medicine |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of participants with donor engraftment | 1 year post transplant | ||
Secondary | Major Transplant Related Toxicities | 1 years post transplant | ||
Secondary | Time to neutrophil recovery | within 100 days post transplant | ||
Secondary | Number of patient with acute GVHD | 180 days post transplant | ||
Secondary | Number of participants with infectious complications | 2 years post transplant | ||
Secondary | Time to immune reconstitution | 2 years post transplant | ||
Secondary | Overall survival | 2 years post transplant | ||
Secondary | Time to platelet recovery | within 100 days post transplant | ||
Secondary | Number of patients with chronic GVHD | 2 years post transplant | ||
Secondary | Disease free survival | 2 years post transplant |
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