Ignite Creation Date:
2024-05-05 @ 5:31 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00478244
Status:
TERMINATED
Last Update Posted:
2017-12-28
First Post:
2007-05-23
Brief Title:
Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
Sponsor:
Masonic Cancer Center University of Minnesota
Organization:
Masonic Cancer Center University of Minnesota
Study Overview
Official Title:
Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
Status:
TERMINATED
Status Verified Date:
2017-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Competing studies
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
RATIONALE In animal models stem cells have been shown to home to the skin and repair the biochemical and structural abnormalities associated with recessive dystrophic epidermolysis bullosa RDEB collagen 7 deficiency
PURPOSE To determine the safety and effectiveness of stem cell infusion in the treatment of RDEB
PURPOSE To determine the safety and effectiveness of stem cell infusion in the treatment of RDEB
Detailed Description:
OBJECTIVES
Primary
Estimate the incidence of detectable donor-derived collagen type VII at day 100 in patients with epidermolysis bullosa by donor
Secondary
Determine the incidence of transplant-related mortality at day 180
Determine the incidence of blood chimerism at days 21 100 180 365 and 730
Determine the incidence of neutrophil recovery at day 42 and platelet recovery at day 180
Determine the incidence of acute graft-versus-host disease GVHD grade II-IV and grade III-IV at day 100
Determine the incidence of chronic GVHD at 1 year
Determine the probability of survival at 1 and 2 years
Determine the incidence of donor derived cells in the skin
Determine resistance to blister formation OUTLINE This is an open-label pilot study
Conditioning regimen Busulfan intravenously IV over 2 hours every 6 hours on days -9 to -4 fludarabine phosphate IV over 1 hour on days -5 to -3 and high-dose cyclophosphamide IV over 1 hour on days -5 to -2
Stem cell transplantation on day 0
After completion of study treatment patients are followed periodically for at least 5 years
PROJECTED ACCRUAL 30 patients
Primary
Estimate the incidence of detectable donor-derived collagen type VII at day 100 in patients with epidermolysis bullosa by donor
Secondary
Determine the incidence of transplant-related mortality at day 180
Determine the incidence of blood chimerism at days 21 100 180 365 and 730
Determine the incidence of neutrophil recovery at day 42 and platelet recovery at day 180
Determine the incidence of acute graft-versus-host disease GVHD grade II-IV and grade III-IV at day 100
Determine the incidence of chronic GVHD at 1 year
Determine the probability of survival at 1 and 2 years
Determine the incidence of donor derived cells in the skin
Determine resistance to blister formation OUTLINE This is an open-label pilot study
Conditioning regimen Busulfan intravenously IV over 2 hours every 6 hours on days -9 to -4 fludarabine phosphate IV over 1 hour on days -5 to -3 and high-dose cyclophosphamide IV over 1 hour on days -5 to -2
Stem cell transplantation on day 0
After completion of study treatment patients are followed periodically for at least 5 years
PROJECTED ACCRUAL 30 patients
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 |
|---|---|---|---|
| UMN-0702M01504 | OTHER | IRB University of Minnesota | None |
| CDR0000546620 | OTHER | None | None |