Phase 3 Confirmatory Study (SD-007) of the Efficacy and Safety of SD-101 Cream
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This is a Phase 3, multi-center, randomized, double-blind, placebo controlled, study to assess the efficacy and safety of SD-101-6.0 cream vs. SD-101-0.0 (placebo) in the treatment of lesions in approximately 80 patients with Simplex, Recessive Dystrophic, or JEB-nH EB. Allantoin is the active ingredient contained within SD-101 skin cream, which is a formulation with previously demonstrated stability of allantoin for several years at concentrations up to 9%. SD-101 cream is currently being developed as a new topical therapy for the treatment of wounds and lesional skin in patients with EB across the entire skin surface. The same product is foreseen for adult and pediatric patients.
To enroll in the study, patients must meet the following inclusion criteria: they must have a signed informed consent form from a legal representative (with assent required, as all participants will be 12 years old or younger), and the patient or caretaker must be willing to follow all study requirements. Eligible participants must have a confirmed diagnosis of Simplex, Recessive Dystrophic, or JEB-nH EB; be between 28 days and 12 years old at study entry; have at least one target wound measuring 10–50 cm²; and present with a baseline wound burden of at least 5% body surface area. Location information will be provided when final site selection is completed. Sites planned to participate will include the US, Europe, South America, Mexico, Malaysia and Australia. |
EBShield Phase 2/3 Clinical Trial of AC-203 for Severe and Intermediate EBS
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TWi Biotechnology is currently recruiting patients into the EBShield study, a Phase 2/3 Clinical Trial of AC-203 (diacerein 1% ointment) for the treatment of severe and intermediate epidermolysis bullosa simplex (EBS). Participants will be randomized in a double-blind design to receive the once-daily ointment active drug or vehicle for 8 weeks, after which all participants continue to receive the active drug for 24 weeks.
Individuals 6 months of age or older with severe or intermediate EBS and the KRT5 or KRT14 gene mutation may qualify for this study (previous genetic testing is preferred but not required for eligibility). This trial is currently enrolling at various sites across the United States. For more information and to find the nearest enrollment center, please contact: [email protected] |
A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
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Castle Creek Biosciences is using its proprietary fibroblast technology platform to develop and evaluate D-Fi, an innovative personalized treatment that is compatible with a patient’s unique biology and delivers functional Type VII collagen (COL7) protein where it is needed – at the site of wounds. The COL7-corrected cells are delivered intradermally (by injection) at the wounds in an outpatient setting in three or more treatment sessions, 8 weeks apart.
The purpose of this Phase 3 Study is to determine whether administration of D-Fi in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Dystrophic Epidermolysis Bullosa. This study is currently recruiting patients at Mission Dermatology in Santa Margarita, CA, Children’s Hospital Colorado in Aurora, CO, and the University of Massachusetts in Worcester, MA, with plans to recruit from Stanford University and Thomas Jefferson University as well. |
Phase 1 Trial Repurposing Dupilumab for Management of Pruritic Genetic Inflammatory Skin Disorders
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Severe itch is a common symptom of many genetic skin disorders, such as EB, and leads to a negative impact on patient quality of life. These investigators hypothesize that: a) intervention with dupilumab will improve itch in patients with pruritic genetic inflammatory skin disorders; and b) the administration of dupilumab will be well-tolerated, regardless of underlying genetic skin disorder.
This trial is currently recruiting for patients older than 6 months at Ann & Robert H. Lurie Children's Hospital of Chicago. The total clinical study duration will be 26 months (104 Weeks). The treatment period will include a 16-week open-label phase and a 20-month long-term extension phase for those who qualify and wish to continue. |
Phase 3 Trial for Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)
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Due to their immunomodulatory and anti-inflammatory properties, ABCB5+ mesenchymal stem cells (ABCB5+ MSCs) may represent a new, promising therapeutic approach to EB. Since EB is a systemic, multi-organ disease, blistering and open wounds can occur anywhere in the body, including the inner mucous membranes (mouth, esophagus, or intestines).
Therefore, the stem cell therapy is not applied externally on the wounds but administered as an infusion. Because of the systemic effect via blood, stem cells can migrate to the injured tissue sites – externally as well as internally – nest in the wound (homing) and promote their healing. The completed phase 2a trial with ABCB5+ MSCs in 16 RDEB patients proved the general anti-inflammatory effect and showed a significant reduction in number and severity of wounds. In addition to the recorded wound healing potential, a significant reduction in itch and pain did improve the general disease burden in these patients4. Larger, pivotal Phase 3 studies are now important to further substantiate the benefit-risk profile in a larger patient population. Enrollment is open at the Masonic Cancer Center and Cedical Center Minneapolis for patients over 6 months old with RDEB. |
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The ocular manifestations of EB can be profound and painful, and if left unaddressed, they can lead to blindness. ELK-003 is an experimental biological drug currently in a clinical trial for the treatment of ocular manifestations of EB.
This study consists of two phases: an Observational Phase to evaluate the natural history of ocular manifestations in subjects with Dystrophic and Junctional Epidermolysis Bullosa, followed by a Treatment Phase to assess the effects of ELK-003 eye drops. Each subject will serve as their own control by comparing ocular manifestations documented during the Observational Phase to those recorded during the Treatment Phase. This study is currently enrolling in Chile for patients older than 2 years with any EB subtype and one or more corneal abrasions every 3 months. |
A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment
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Researchers at USC are studying Gentamicin as a therapy for JEB causes by nonsense mutations. Gentamicin is an aminoglycoside antibiotic which can override nonsense mutations in DNA sequences, allowing full length, functional proteins to form.
The investigators recently demonstrated that gentamicin readily induced nonsense mutation readthrough and produced full-length laminin beta3 in several nonsense mutations tested. Importantly, the gentamicin-induced laminin beta3 restored laminin 332 assembly, secretion, and deposition into the dermal-epidermal junction (DEJ). Newly induced laminin 332 reversed abnormal H-JEB cellular phenotypes. Now, the investigators propose the first clinical trial of gentamicin (by topical and intravenous administration) in JEB patients with nonsense mutations. Recruiting is now open at the University of Southern California for JEB patients with nonsense mutations in the LAMB3 gene in either one or two alleles. |
A Phase 3 Trial Assessing KB803 Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa (IOLITE)
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This phase 3 trial aims to evaluate KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients. KB803 is designed to deliver two copies of the COL7A1 transgene to the corneal epithelium and enable local type VII collagen production in the front of the eye.
IOLITE is an intra-patient, double-blind, placebo-controlled, multicenter Phase 3 study with a crossover design to evaluate KB803, administered as an eye drop, for the treatment and prevention of corneal abrasions in DEB patients, 6 months of age or older. Patients seeking to participate in IOLITE must first enroll in the ongoing natural history study and complete a 12-week run-in period, during which they report the number of days that they experience symptoms of corneal abrasions. Subjects meeting the inclusion criteria following the 12-week run-in are eligible to participate in the IOLITE trial. Enrollment is open at multiple sites across the US, including Mission Dermatology Center and Texas Dermatology and Laser Specialists, with more sites to come including UMass Memorial Medical Center and Cincinnati Children's Hospital Medical Center. |
Phase 2 Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
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ALLO-ASC-SHEET is a hydrogel sheet containing allogeneic adipose-derived mesenchymal stem cells (ASCs) being investigated for the treatment of Dystrophic Epidermolysis Bullosa (DEB). It's being studied for its potential to promote wound healing and tissue regeneration in DEB patients.
After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which lesion to apply which IP treatment will be determined randomly at the time of enrollment using pre-designed block randomization scheme. This trial is currently enrolling at University of Miami Dermatology for DEB patients aged 4-60 years old. |
A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex (TAMES)
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BioMendics is investigating a topical gel called TolaSure to see if it can help with intermediate and severe EB Simplex blistering. Participants may receive TolaSure Gel or a Placebo Gel (without the active ingredient) during the first part of the study which consists of daily application of treatment for 2months. During the second part of the study, all participants will receive TolaSure gel and treat their blistering skin daily for an additional 2 months. A follow-up visit will take place 2 more months after treatment for a total of a 6-month time commitment.
The BioMendics’ TAMES-02 trial is studying is actively recruiting patients 4 years and older at Northwestern University and Stanford University. Patients must have a documented diagnosis of generalize intermediate (previously Kӧbner) to severe (previously EBS-DM) autosomal dominant epidermolysis bullosa simplex (EBS) and/or genetic mutation in either the K14 or K5 genes consistent with generalized intermediate to severe EBS. |
Phase 1/2A, Randomized, Multi-center Study to Assess the Effectiveness and Safety of AGLE-102 on Lesions in Subjects with EB.
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AGLE-102 is an allogeneic extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs). AGLE-102 contains COL7 protein as well as COL7A1 mRNA, which in preclinical models stimulates recessive DEB cells (cannot make any COL7) to produce COL7. AGLE-102 has the potential to induce functional regeneration and organization of complex tissue structures in DEB and EB patients that can potentially accelerate and enhance healing, reduce scarring, and improve overall cosmesis. In addition, AGLE-102 is non-immunogenic and carries the immunomodulating and anti-inflammatory properties of MSCs.
Aegle’s Phase 1/2 clinical trial involving AGLE-102 in individuals living with Recessive Dystrophic Epidermolysis Bullosa (RDEB) is now open for enrollment. Clinical Trial sites include Phoenix Children's Hospital, University of Southern California, and Children's Hospital of Philadelphia. |
A Phase 2 Trial Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
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The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in serum. Fewer wounds, more rapidly healing wounds, and decreased C7 and HSV-1 antibodies could improve quality of life for those living with EB.
Enrollment is now open at Stanford University for patients aged 6 years or older with a diagnosis of generalized Recessive dystrophic epidermolysis bullosa (RDEB) demonstrated by COL7A1 mutations or a diagnosis of EBA demonstrated by the presence of levels of serum C7 antibodies above the normal ELISA range. |
A Phase II, Open Study to Assess Efficacy and Safety of Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced/Metastatic Squamous Cell Carcinoma
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Eighty percent of all patients suffering from recessive dystrophic EB (RDEB), a subtype originating from mutations in the COL7A1 gene, develop squamous cell carcinoma (SCC). In light of scarce data on the efficacy and safety of systemic treatment regimens for advanced SCC, the investigators propose to perform a small, "first in EB " trial of an experimental drug called rigosertib for the treatment of EB cancer.
The trial will be conducted in two study centres, in London and Salzburg, and will last approximately 2.5 years with each patient recruited being in the study for 1 year. The drug is a polo-like kinase inhibitor interfering with different molecular pathways that are essential for cancer cell growth. The investigators have identified that rigosertib most selectively kills EB cancer cells in vitro while leaving normal EB skin cells unaffected. Rigosertib was developed by Onconova Therapeutics and is currently tested in several clinical trials for a number of other cancers including myelodysplastic syndrome (a cancer of the blood). Enrollment is currently open at the EB Haus in Salzburg, Austria for patients 18-79 years of age with a diagnosis of unresectable, locally advanced or metastatic SCC confirmed prior to the Screening Visit. |
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