Ignite Creation Date:
2024-05-05 @ 5:11 PM
Last Modification Date:
2024-10-26 @ 9:29 AM
Study NCT ID:
NCT00408447
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-13
First Post:
2006-12-06
Brief Title:
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
Sponsor:
Columbia University
Organization:
Columbia University
Study Overview
Official Title:
Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients With Sickle Cell Disease and Thalassemia
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-06
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:
The primary purpose of this study is to see if giving lower doses of chemotherapy moderately ablative will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease Patients will receive a chemotherapy regimen with busulfan fludarabine and alemtuzumab followed by an infusion of stem cells either from a family-related or cord-blood matched donor
Detailed Description:
Sickle cell disease is a genetic disorder in which a mutation in the beta chain of human hemoglobin results in abnormal blood hemoglobin causing red blood cells to sickle under stress with resulting symptoms including severe pains and strokes Beta thalassemia is another genetic disorder in which there are abnormal beta hemoglobin chains causing anemia In both disorders frequent red blood cell transfusions may be required to sustain life but these often result in complications including multiple hospitalizations iron overload or bacterial or viral infections such as hepatitis Standard drugs and therapies used in the treatment of sickle cell disease andor beta thalassemia provide only supportive care and may result in long-term side effects and inadequate control of the disease process Bone marrow transplant has been increasingly used for the long-term treatment and cure of sickle cell disease and beta thalassemia Although not without acute and potential long term side effects this alternative offers long term control and potential cure of the disease Most of the side effects seen with bone marrow transplant are directly related to the high intensity of chemotherapy used ablative
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CHNY-01-503 | OTHER | CU | None |