Clinical Trial Search

Primary: Number of Wounds by Healing Category Per Investigator Visual Assessment — 35; 5; 2; 28 wounds

Primary: Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence — 31.8; 28.4; 65.6 C7 Expression (percent of normal skin)

Primary: Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. — 5; 5; 4; 4 Fluorescence Intensity (MFI) for C7

Primary: Percentage of Wound Surface Area Change From Baseline to Week 8 — -63; -8.1 percentage of wound surface area

Primary: Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment — 8; 6; 7; 7 Participants — p==0.3699

Primary: Number of Wounds With Greater Than 90% Wound Closure at 4 Months — 0; 3 Wounds — p=0.21

Primary: Foot Health Status Questionnaire, Foot Function Domain Score — 61.7; 49.3; 60.2; 56.3 score on a scale

Primary: Percentage of Participants With Event-free Survival — 67; 80; 50; 29 Percentage of participants

Primary: Proportion of Patients With First Complete Closure of the EB Target Wound Within 45 Days of Treatment — 45; 33 Participants — p=0.013

Primary: Event-free Survival — 4; 2; 1 Count of Participants

Primary: Intra-individual Difference in Reepithelialization of Wound (Halves) at Day 14 in 'Recent Wounds' or Day 28 in 'Chronic Wounds' — 8; 0 Wounds — p==0.008

Primary: Percent Change of Active Blisters and in Total Blister/Erosion Counts — -29.6 percent change — p=0.82

Primary: Percentage of Grafts Successfully Treated — 78 Percentage of grafts

Primary: Treatment-emergent Adverse Events — 40 Participants

Primary: Blister/Erosion Reduction Based on Change in Body Surface Area (BSA) Coverage — 57.25; 39.40; 27.25; 25.45 percentage of BSA involvement

Primary: Time To Complete Target Wound Closure Within 3 Months — 41.6; 53.6 days — p=0.985

Primary: Number of Subjects Reported at Least One Adverse Event, Safety Population — 9 Participants

Primary: Heterozygous for Filaggrin (FLG) Null Mutations — 1; 4 participants

Primary: Detectable Plasma Concentrations of Diacerein and Rhein — 3; 2; 0; 0 Participants

Primary: Mortality — 5; 3; 5 Participants

Primary: Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period — -2; -1 score/week comparative change

Primary: Plasma Follistatin

Primary: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks — 0.55; 0.86 AU — p=0.0434

Primary: The Hospital Anxiety and Depression Scale (HADS) — 4.6; 1.9 score on a scale

Primary: Number of Patients Who Achieve at Least a 3-point Reduction in AI-NRS. — 1; 3 Participants — p=0.59

Primary: Change From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756

Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30

Primary: Change in Investigator Global Assessment (IGA) Score — 41.4; 44.9 percentage of responders — p=0.39

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants

Primary: Number of Participants With Partial Response to Dabrafenib and Trametinib — 4 Participants