Ignite Creation Date:
2024-05-06 @ 7:37 PM
Last Modification Date:
2024-10-26 @ 3:10 PM
Study NCT ID:
NCT06073132
Status:
RECRUITING
Last Update Posted:
2024-07-11
First Post:
2023-09-12
Brief Title:
An International Multicenter Randomized Double-Blind Parallel Group Vehicle-Controlled Phase 23 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1 Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex EBS
Sponsor:
TWi Biotechnology Inc
Organization:
TWi Biotechnology Inc
Study Overview
Official Title:
An International Multicenter Randomized Double-Blind Parallel Group Vehicle-Controlled Phase 23 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1 Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex EBS EBShield Study
Status:
RECRUITING
Status Verified Date:
2024-10
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:
EBShield
Brief Summary:
The proposed Phase 23 trial with double-blind and open-label extension phases is an international multicenter study designed to assess the efficacy and safety of diacerein 1 ointment in patients with generalized EBS
Detailed Description:
Epidermolysis bullosa simplex EBS is a genetic skin disorder characterized by skin fragility and recurrent blister formation primarily caused by mutations in keratins 5 and 14 EBS has 3 common subtypes based on clinical severity and manifestations localized EBS intermediate EBS and severe EBS Severe EBS and intermediate EBS collectively are also known as generalized EBS due to widespread blistering
Disruption of the keratin 514 filament network in basal keratinocytes is a key factor in EBS pathogenesis compromising skin integrity The severity of EBS is linked to the extent of keratin mutations disrupting this network particularly resulting in keratin aggregates in severe cases Recent studies suggest that mutated keratin proteins can trigger inflammation exacerbating EBS Elevated proinflammatory cytokines like IL-1β and IL-6 are observed in EBS patients and IFN-γ may mediate inflammation promoting keratin aggregations As a result targeting inflammation is considered a potential therapeutic approach in EBS
AC-203 diacerein 1 ointment is a topical formulation of diacerein well-known for its ability to inhibit IL-1β and other proinflammatory cytokines Moreover diacerein and its active metabolite rhein have demonstrated ability in reducing keratin aggregates in keratinocytes derived from severe EBS Taken together with its anti-inflammatory property and ability to diminish keratin aggregation AC-203 shows promise in reducing the clinical severity of EBS
Disruption of the keratin 514 filament network in basal keratinocytes is a key factor in EBS pathogenesis compromising skin integrity The severity of EBS is linked to the extent of keratin mutations disrupting this network particularly resulting in keratin aggregates in severe cases Recent studies suggest that mutated keratin proteins can trigger inflammation exacerbating EBS Elevated proinflammatory cytokines like IL-1β and IL-6 are observed in EBS patients and IFN-γ may mediate inflammation promoting keratin aggregations As a result targeting inflammation is considered a potential therapeutic approach in EBS
AC-203 diacerein 1 ointment is a topical formulation of diacerein well-known for its ability to inhibit IL-1β and other proinflammatory cytokines Moreover diacerein and its active metabolite rhein have demonstrated ability in reducing keratin aggregates in keratinocytes derived from severe EBS Taken together with its anti-inflammatory property and ability to diminish keratin aggregation AC-203 shows promise in reducing the clinical severity of EBS
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