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A Phase II, Closed Label, Placebo Controlled, Randomized, Double-Blinded Clinical Trial to Evaluate the Efficacy and Safety of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex (TAMES)
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and old…
A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa
The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 y…
A Randomized, Double-blind, Vehicle-controlled Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of Topically Applied TCP-25 in Patients With Epidermolysis Bullosa
This is a Phase 2, double-blind, randomized, vehicle-controlled study designed to evaluate efficacy, safety, and tolerability of topically applied TCP-25 gel i…
A Pilot Trial to Evaluate the Efficacy and Safety of Filsuvez in the Treatment of Moderate-to-Severe Epidermolysis Bullosa Simplex
This pilot study will look at whether the topical Birch Triterpenes (Filsuvez) gel is safe and helpful for adults and children 6 months of age and older who ha…
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystroph…
Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by genetic mutations in the gene for type VII collagen. Patients with RDEB develop large,…
Mesenchymal Stem Cell Extracellular Vesicles for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Wounds
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs). INDICATION AND …
A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or L…
Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves woun…
A Prospective, Observational, Long-Term Follow-Up (LTFU) Study to Evaluate the Long-Term Safety of the Krystal Biotech, Inc. Gene Therapy Backbone Products Using HSV-1 Backbone
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy product…
A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis B…
Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Ep…