For more information, contact:
Kunju Sridhar kunju@stanford.edu
or
Jim Cimino cimino@stanford.edu
Clinical Trials for Epidermolysis Bullosa (EB)
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)
The aim of this trial is to investigate the safety and efficacy of allogeneic dermal ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs) administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. These cells suppress inflammation and produce proteins that are important for wound healing.
Recruitment Status: Recruting
Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
This clinical study aims to investigate the safety and efficacy of ALLO-ASC-SHEET used in the form of a dressing applied to wounds and surrounding areas. The type of stem cell being used in this research study are adipose-derived stem cells (ASCs) obtained from adipose (fat) tissue. They release growth factor proteins that promote the formation of new blood vessels which may aid in the healing of the wound and can enhance regeneration of new tissue.
Recruitment Status: Recruiting
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
- This study investigates the safety and the effectiveness of an experimental drug called PTW-002 given to patients who have confirmed DDEB or RDEB with at least one mutation in exon 73 of the COL7A1 gene.
- More
- Recruitment Status: Recruiting
A Phase III Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
This study will look at an experimental therapy called FCX-007. FCX-007 is a genetically modified (changed) version of your own cells. FCX-007 is designed to deliver a functioning version of the collagen VII protein that is missing in RDEB patients into the skin. The study is designed to see how well FCX-007 works for treating RDEB wounds and to look at its safety.
Recruitment Status: On-hold for recruitment
Open Label Treatment of Beremagene Geperpavec (B-VEC)
The primary objective of this stdudy will be to assess long term safety and tolerability of the weekly dosed topical B-VEC gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.
Recruitment Status: Closed
Clinical Trials for Bullous Pemphigoid (BP)
A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
The objective of this study is to investigate the efficacy and safety of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP.
Recruitment Status: Recruiting
Clinical Trials for Pemphigus
A phase 1, open-label, safety and dosing study of autologous desmoglein 3 chimeric autoantibody receptor T cells (DSG3-CAART) in subjects with active, anti-DSG3, mucosal-dominant pemphigus vulgaris
Mucosal-dominant pemphigus vulgaris (mPV) is a B-cell mediated autoimmune disorder in which painful blisters are formed on the mucosal membrane, including the mouth, nose, throat, eyelids, anus, and genitals. This study is being conducted to find the maximum tolerated dose and optimal fractionated infusion schedule of an investigational cell therapy, DSG3-CAART, that can be given to patients with mPV who are inadequately managed by standard therapies. DSG3-CAART may potentially lead to complete and durable remission of disease.
Recruitment Status: Recruiting
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus
This is an open label extension (OLE) trial on the efficacy and safety of efgartigimod PH20 SC in adult pemphigus vulgaris (PV)
or pemphigus foliaceus (PF) participants, who participated in antecedent trial ARGX-113-1904.
Recrutiment Status: Closed