Ignite Creation Date:
2024-05-05 @ 9:26 PM
Last Modification Date:
2024-10-26 @ 10:04 AM
Study NCT ID:
NCT00881556
Status:
TERMINATED
Last Update Posted:
2021-08-17
First Post:
2009-04-14
Brief Title:
Allogeneic Stem Cell Transplantation ALLOSCT in Recessive Dystrophic Epidermolysis Bullosa RDEB
Sponsor:
Columbia University
Organization:
Columbia University
Study Overview
Official Title:
A Pilot Study of Reduced Intensity Conditioning RIC and Allogeneic Stem Cell Transplantation ALLOSCT In Children With Recessive Dystrophic Epidermolysis Bullosa RDEB
Status:
TERMINATED
Status Verified Date:
2021-08
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:
RDEB
Brief Summary:
Reduced Intensity Conditioning RIC and Allogeneic Stem Cell Transplantation AlloSCT from family-related donors and unrelated cord blood UCB donors will be safe and well tolerated in selected patients with RDEB
To determine the event-free survival EFS and overall survival OS following RIC consisting of busulfanfludarabinealemtuzumab BFA and AlloSCT in selected patients with RDEB
To determine the event-free survival EFS and overall survival OS following RIC consisting of busulfanfludarabinealemtuzumab BFA and AlloSCT in selected patients with RDEB
Detailed Description:
Epidermolysis bullosa EB is a diverse group of genodermatoses which is considered a rare and orphan disease and affects approximately 1 in 20000 people in the United States for a cumulative total of close to 200001-4 There are three major subtypes of inherited EB including EB simplex EBS junctional EB JEB and dystrophic EB1-4 RDEB is among the most severe and represents approximately 10 of all forms of EB1-4 A rough estimate would then project that there are several thousand patients with RDEB in the US at the current time Up to 30 different clinical phenotypes and mutations in at least 10 structural genes in different sub-types of EB have been reported4-8 In addition to heritable subtypes of EB there is an acquired autoimmune form in which the patients develop auto-antibodies directed against similar proteins of the inherited dystrophic forms of EB including EB acquisita EBA
We have previously reported our experience with RIC with BFA 48 in pediatric AlloSCT recipients mean age 95 yrs 14-21 114 MF 10 non-malignant 5 malignant disease 6 sibling 5 UCB 5 matched unrelated donor median time to ANC 500mm3 and platelet count 20Kmm3 was 22 and 30 days respectively Probability of day 180 and 365 donor chimerism was 90 Figure 7 and OS was 95 Figure 8 This conditioning regimen therefore results in a high degree of donor chimerism and survival with minimal regimen related mortality
We have previously reported our experience with RIC with BFA 48 in pediatric AlloSCT recipients mean age 95 yrs 14-21 114 MF 10 non-malignant 5 malignant disease 6 sibling 5 UCB 5 matched unrelated donor median time to ANC 500mm3 and platelet count 20Kmm3 was 22 and 30 days respectively Probability of day 180 and 365 donor chimerism was 90 Figure 7 and OS was 95 Figure 8 This conditioning regimen therefore results in a high degree of donor chimerism and survival with minimal regimen related mortality
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?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CHNY-08-536 | OTHER | CUMC | None |