Ignite Creation Date:
2024-05-05 @ 7:46 PM
Last Modification Date:
2024-10-26 @ 9:53 AM
Study NCT ID:
NCT00730314
Status:
COMPLETED
Last Update Posted:
2016-06-23
First Post:
2008-08-06
Brief Title:
Unrelated Hematopoietic Stem Cell TransplantationHSCT for Genetic Diseases of Blood Cells
Sponsor:
Childrens Hospital Los Angeles
Organization:
Childrens Hospital Los Angeles
Study Overview
Official Title:
Phase III Trial Of Hematopoietic Stem Cell Transplant HSCT For Children With A Genetic Disease Of Blood Cells Without An HLA-Matched Sibling Donor
Status:
COMPLETED
Status Verified Date:
2016-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor Genetic diseases of blood cell include Red blood cell defects eg hemoglobinopathies sickle cell disease and thalassemia Blackfan-Diamond anemia and congenital or chronic hemolytic anemias White blood cells defectsimmune deficiencies eg chronic granulomatous disease Wiskott-Aldrich syndromeOsteopetrosis Kostmanns syndrome congenital neutropenia Hereditary Lymphohistiocytosis HLH Platelets defects egCongenital amegakaryocytic thrombocytopenia Metabolicstorage disorders eg leukodystrophiesmucopolysaccharidoses as Hurler diseaseStem cell defects egreticular agenesis among many other rare similar conditions
The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome
The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood
The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome
The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood
Detailed Description:
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor The stem cells will be derived from a 1 matched unrelated donor MUD or 2 unrelated umbilical cord blood UCB Patients will receive a novel conditioning regimen with Busulfan Cytoxan and Fludarabine BuCyFlu and either Alemtuzumab Campath 1H for recipients of a MUD or rabbit Antithymocyte Globulin rATG for recipients of unrelated UCB prior to hematopoietic stem cell transplant HSCT
It is hypothesized that reduced dosages of Cytoxan will decrease the acute toxicities associated with the standard chemotherapies of Busulfan and Cytoxan ie sinusoidal obstructive syndrome SOS hemorrhagic cystitis and mucositis And the addition of fludarabine to a conditioning regimen with myeloablative doses of Busulfan and reduced dosages of Cytoxan prior to HSCT will overcome the engraftment barrier posed by an intact immune system which is seen in patients with a genetic disease
It is hypothesized that reduced dosages of Cytoxan will decrease the acute toxicities associated with the standard chemotherapies of Busulfan and Cytoxan ie sinusoidal obstructive syndrome SOS hemorrhagic cystitis and mucositis And the addition of fludarabine to a conditioning regimen with myeloablative doses of Busulfan and reduced dosages of Cytoxan prior to HSCT will overcome the engraftment barrier posed by an intact immune system which is seen in patients with a genetic disease
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CHLA-07-00119 | None | None | None |