Ignite Creation Date:
2024-05-06 @ 9:34 AM
Last Modification Date:
2024-10-26 @ 12:16 PM
Study NCT ID:
NCT03016715
Status:
UNKNOWN
Last Update Posted:
2017-10-24
First Post:
2017-01-09
Brief Title:
Using Topical Sirolimus 2 for Patients With Epidermolysis Bullous Simplex EBS Study
Sponsor:
Premier Specialists Australia
Organization:
Premier Specialists Australia
Study Overview
Official Title:
A Prospective Double-Blind Cross-Over Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2 in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex EBS
Status:
UNKNOWN
Status Verified Date:
2017-10
Last Known Status:
RECRUITING
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:
Epidermolysis bullosa EB simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin Patients with EB simplex develop lifelong painful thick soles on their feet and current standard of care is supportive This pilot study will target the dominant mutant keratin proteins in the skin to ameliorate the severity of EB simplex The purpose is to improve the function of EB simplex feet with an application of topical sirolimus 2 The investigators plan on inhibiting the mTOR pathway to down regulate the translation of defective keratin proteins and work through anti proliferative pathways
Detailed Description:
The proposed 40 week pilot study being conducted is a prospective double-blind randomized placebo-controlled crossover study Participants will be assigned to treat both feet with either topical sirolimus 2 cream daily or placebo vehicle-control for 12 weeks followed by a 4 week washout period then re-treatment to both feet will occur by the cross-over intervention
These studies will exploit the naturally occurring transcriptional regulation of keratin sequences the known gene aberration causing EB simplex and assess the potential for mTOR pathway inhibition in treatment of the patients plantar lesions The objective of this study is to assess 1 the safety of topical rapamycin for plantar lesions for the treatment of EB simplex and 2 test if topical rapamycin to improves the clinical severity of lesional skin including pain and itch in subjects with EB simplex at the end of treatment versus baseline and compared to an intrasubject placebo treated control Wound size measurement quality of life evaluation will be assessed using epidermolysis bullosa QOLEB and EB disease activity and Scarring Index EBDASI With the results of this pilot study physicians would be able to transition from supportive care the current state of the art for EB simplex to targeted molecular therapeutics leading to improved mobility and quality of life for patients with EB simplex
These studies will exploit the naturally occurring transcriptional regulation of keratin sequences the known gene aberration causing EB simplex and assess the potential for mTOR pathway inhibition in treatment of the patients plantar lesions The objective of this study is to assess 1 the safety of topical rapamycin for plantar lesions for the treatment of EB simplex and 2 test if topical rapamycin to improves the clinical severity of lesional skin including pain and itch in subjects with EB simplex at the end of treatment versus baseline and compared to an intrasubject placebo treated control Wound size measurement quality of life evaluation will be assessed using epidermolysis bullosa QOLEB and EB disease activity and Scarring Index EBDASI With the results of this pilot study physicians would be able to transition from supportive care the current state of the art for EB simplex to targeted molecular therapeutics leading to improved mobility and quality of life for patients with EB simplex
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