[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"recessive-dystrophic-epidermolysis-bullosa\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:recessive-dystrophic-epidermolysis-bullosa":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,73,99,138,167],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":24,"conditions":25,"keywords":30,"overallStatus":60,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":62,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":72},"100593834","phase-1-targeting-collagen-vii-antibodies-in-bullous-diseases-using-efgartigimod-iv-vyvgart-100593834",false,"NCT07011589","Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)","Inclusion Criteria:\n\n* For DEB patients (aged 12 years or older): DEB confirmed with mutation analysis and correlated by phenotype, and treatment of at least 1 wound treated with topical gene therapy (VYJUVEK). Presence of C7 antibodies above normal cutoff on ELISA.\n* For (classic) EBA patients (aged 18 years or older): EBA confirmed with positive histopathology (DIF), C7 antibodies above normal cutoff on ELISA, and having at least 1 skin lesion.\n* The participant has a Karnofsky performance status of at least 60% at screening.\n* Contraceptive use by reproductive male and female patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:\n\n  1. Male participants:\n\n     \\- Must agree to use an acceptable method of contraception and not donate sperm from the time that the ICF is signed until they have received their last dose of IMP.\n  2. Female participants:\n\n     * Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at week 1 \u002F baseline before study intervention can be administered. Subsequent urine pregnancy tests are to be completed at week 6, week 11, week 16, week 21, and week 26 \u002F EoS.\n     * WOCBP must agree to use a highly effective or acceptable contraception method until at least 90 days after they receive their last dose of IMP.\n\nExclusion Criteria:\n\n* Linear IgA dermatosis-like EBA or other autoimmune blistering diseases (including but not limited to pemphigus vulgaris, bullous pemphigoid, mucous membrane pemphigoid).\n* Use of the following EBA treatments:\n\n  * sulfasalazine, IVIg, subcutaneous administration of immunoglobulin (SCIg), immunoadsorption or plasma exchange within 2 weeks of the screening visit, tetracyclines with or without nicotinamide at doses higher than the recommended daily allowance (RDA)\u002Fdietary reference intake (DRI) within 2 weeks of the screening visit.\n  * any monoclonal antibody (including rituximab or another anti-CD20 biologic) within 6 months of the screening visit.\n  * complementary therapies-such as traditional Chinese medicines, herbs, or procedures (e.g., acupuncture)-within 4 weeks (or 5 half-lives) of the screening visit, if the investigator determines that such therapies may interfere with the study's efficacy assessments and\u002For potentially risk the safety of the participant.\n  * The use of the following EBA treatments is permitted throughout the study: OCS, topical corticosteroids, conventional immunosuppressants (e.g., azathioprine, cyclophosphamide, methotrexate, mycophenolate, or mofetil), and dapsone.\n* Moderate to severe renal insufficiency.\n* Known contraindication to OCS therapy.\n* Clinically significant uncontrolled active or chronic, bacterial, viral, or fungal infection at screening\n* Medical instability limiting ability to travel to the Investigative Center\n* Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits\u002Fprocedures, as determined by the investigator.\n* Subjects actively receiving chemotherapy or immunotherapy at screening\n* Active drug or alcohol addiction as determined by the investigator.\n* Pregnant or nursing women\n* History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to screening:\n\n  1. Basal cell or squamous cell skin cancer\n  2. Carcinoma in situ of the cervix\n  3. Carcinoma in situ of the breast\n  4. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)","ALL","12 Years",{"count":18,"type":19},18,"ESTIMATED","INTERVENTIONAL",[22,23],"PHASE1","PHASE2","The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum.\n\nFewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.",[26,27,28,29],"Epidermolysis Bullosa (EB)","Epidermolysis Bullosa Acquisita","Recessive Dystrophic Epidermolysis Bullosa","Dystrophic Epidermolysis Bullosa",[31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51,52,53,54,55,56,57,58,59],"epidermolysis bullosa","epidermolysis bullosa acquisita","dystrophic epidermolysis bullosa","recessive dystrophic epidermolysis bullosa","recessive","dystrophic","epidermolysis","bullosa","acquisita","collagven 7","c7","Col7","EB","EBA","DEB","RDEB","antibodies","Collagen VII","VII","IV IgG","IgG","IV","IVIgG","C7 antibodies","Collagen 7 antibodies","Collagen VII Antibodies","Vyjuvek","B-VEC","Efgartigimod","NOT_YET_RECRUITING","2026-04-29",{"date":63,"type":64},"2026-05-05","ACTUAL",{"date":66,"type":19},"2026-07",{"date":68,"type":19},"2028-01",{"name":70,"class":71},"M. Peter Marinkovich","OTHER",1,{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":77,"acronym":50,"eligibilityCriteria":78,"healthyVolunteers":11,"sex":15,"minAge":79,"maxAge":4,"enrollmentInfo":80,"targetDuration":4,"studyType":20,"phases":82,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":93,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":72},"100580187","phase-1-targeting-collagen-vii-antibodies-with-iv-igg-in-dystrophic-epidermolysis-bullosa-100580187","NCT06834035","Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa","Inclusion Criteria:\n\n1. Diagnosis of generalized Recessive dystrophic epidermolysis bullosa (RDEB) demonstrated by COL7A1 mutations.\n2. Diagnosis of EBA demonstrated by the presence of levels of serum C7 antibodies above the normal ELISA range\n3. Baseline skin blistering greater than 5% total body surface area\n4. 1 wound at least 20 cm\\^2 able to be entirely treated with Vyjuvek weekly\n5. 1 wound at least 20 cm\\^2 that has never been treated with Vyjuvek\n6. Ongoing VYJUVEK treatment.\n\nExclusion Criteria:\n\n1. History of thrombotic event(s)\n2. History of cardiac failure\n3. History of renal failure\n4. IgA deficiency","6 Years",{"count":81,"type":19},8,[22,23],"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.\n\nFewer wounds, more rapidly healing wounds, and decreased C7 and HSV-1 antibodies could improve quality of life.",[85,27,29,28],"Epidermolysis Bullosa",[85,27,29,28,87,88,42,43,44,45,46,89,48,50,51,53,90,91,56,57,58],"Collagen 7","C7","Antibodies","C7 Antibodies","Collagen 7 Antibodies","RECRUITING",{"date":63,"type":64},{"date":95,"type":64},"2025-08-04",{"date":97,"type":19},"2027-03",{"name":70,"class":71},{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":4,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":15,"minAge":106,"maxAge":4,"enrollmentInfo":107,"targetDuration":4,"studyType":20,"phases":109,"briefSummary":110,"conditions":111,"keywords":113,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":129,"lastUpdatePostDateStruct":130,"startDateStruct":132,"completionDateStruct":134,"leadSponsor":136,"locationsCount":72},"100607790","phase-1-gmeb-sass-a-gene-modified-skin-substitute-for-rdeb-treatment-100607790","NCT07193134","GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment","Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) to Treat Patients Suffering From Recessive Dystrophic Epidermolysis Bullosa (RDEB)","Inclusion Criteria:\n\n* Age\n\nLearning phase:\n\n* Subjects 1 to 3: Eighteen (18) years old or older.\n* Subjects 4 to 6: Twelve (12) years old or older.\n* Subjects 7 to 9: Seven (7) years old or older.\n\nOther Inclusion Criteria:\n\n* Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene.\n* Candidates - or their parents\u002Fcaregivers if the candidates have limited comprehension, who are able to understand the study and to comply with the study procedures.\n* On the day of grafting, one or more blistered and\u002For erosive skin areas on the trunk and\u002For extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts.\n* Ability to undergo anesthesia.\n\nExclusion Criteria:\n\n* Medical instability limiting the ability to travel to the investigative center.\n* Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment.\n* Evidence of systemic infection.\n* Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted.\n* Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that could compromise participant safety, as per the investigator's judgment.\n* History of or known allergy to bovine proteins.\n* Active drug or alcohol addiction.\n* Female candidate who are pregnant or breast-feeding.\n* Candidate who has received immunotherapy, including oral corticosteroids (Prednisolone \\> 1 mg\u002Fkg), for more than one week, within 2 weeks prior to the study intervention (initial biopsy and GMEB-SASS grafting) (intranasal and topical preparations are permitted).\n* Candidate who has received chemotherapy within 60 days prior to the study intervention (initial biopsy and GMEB-SASS grafting).\n* Candidate who has received, in the last 6 months prior to the study intervention (initial biopsy and GMEB-SASS grafting), any gene therapy, chemical or biological product modifying collagen 7 expression.","7 Years",{"count":108,"type":19},9,[22,23],"This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa).\n\nThe GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts. It is made in a laboratory using a small sample of the patient's own skin.\n\nTo help the patient's skin cells produce a missing protein called type VII collagen, scientists grow the patient's cells in the lab and use a virus-like tool (called a retroviral vector) to give the cells the correct instructions. This allows the cells to make the normal protein that is missing in people with RDEB.\n\nThe graft is designed to be permanent, and the goal is to improve wound healing by replacing damaged skin cells with healthy ones.",[46,28,112],"Epidermolysis Bullosa Dystrophica, Recessive",[114,115,85,116,117,118,119,120,121,122,123,124,125,126,127,128],"Skin Abnormalities","Congenital Abnormalities","Skin Diseases, Genetic","Genetic Diseases, Inborn","Collagen Diseases","Connective Tissue Diseases","Skin and Connective Tissue Diseases","Skin Diseases","Skin Diseases, Vesiculobullous","Epidermolysis Bullosa Dystrophica","Genetic therapy","Autologous Skin Graft","SIN Retroviral Vector","Type VII Collagen","COL7A1","2026-03-17",{"date":131,"type":64},"2026-03-18",{"date":133,"type":64},"2026-01-07",{"date":135,"type":19},"2035-12",{"name":137,"class":71},"CHU de Quebec-Universite Laval",{"id":139,"slug":140,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":143,"acronym":144,"eligibilityCriteria":145,"healthyVolunteers":11,"sex":15,"minAge":146,"maxAge":4,"enrollmentInfo":147,"targetDuration":4,"studyType":20,"phases":149,"briefSummary":151,"conditions":152,"keywords":155,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":157,"lastUpdatePostDateStruct":158,"startDateStruct":160,"completionDateStruct":162,"leadSponsor":164,"locationsCount":5},"100594231","phase-3-a-study-comparing-kb803-and-matched-placebo-in-patients-with-dystrophic-epidermolysis-bullosa-100594231","NCT07016750","A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa","A Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa","IOLITE","Inclusion Criteria:\n\n1. The subject and\u002For their parent\u002Flegal guardian must provide informed consent\u002Fassent and must be able to and willing to follow study procedures and instructions.\n2. Age 6 months or older at time of informed consent\u002Fassent.\n3. Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.\n4. Meets minimum corneal abrasion symptom frequency in the NHS study.\n\nExclusion Criteria:\n\n1. Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears\u002Flubricants associated with standard of care treatment of corneal abrasions.\n2. Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).\n3. Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and\u002For poses an additional risk to the subject.\n4. Women who are pregnant or nursing.\n5. Subject who is unwilling to comply with contraception requirements per protocol.\n6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.","6 Months",{"count":148,"type":19},16,[150],"PHASE3","KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).",[29,153,28,154],"DEB - Dystrophic Epidermolysis Bullosa","Dominant Dystrophic Epidermolysis Bullosa",[29,45,156],"Corneal Abrasions","2026-03-11",{"date":159,"type":64},"2026-03-12",{"date":161,"type":64},"2025-06-20",{"date":163,"type":19},"2026-12",{"name":165,"class":166},"Krystal Biotech, Inc.","INDUSTRY",{"id":168,"slug":169,"hasResults":11,"nctId":170,"briefTitle":171,"officialTitle":172,"acronym":4,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":15,"minAge":146,"maxAge":4,"enrollmentInfo":174,"targetDuration":4,"studyType":176,"phases":4,"briefSummary":177,"conditions":178,"keywords":4,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":179,"lastUpdatePostDateStruct":180,"startDateStruct":182,"completionDateStruct":184,"leadSponsor":186,"locationsCount":187},"100432959","long-term-follow-up-protocol-100432959","NCT04917887","Long-Term Follow-up Protocol","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","Inclusion Criteria:\n\n* All adult and pediatric participants who received at least one gene therapy treatment in a previous Krystal Biotech, Inc. sponsored study, and have discontinued or completed the parent treatment protocol (which do not have the LTFU embedded into the parent protocol), as applicable.\n* Consent\u002FAssent must be obtained by the participants (and, parental\u002Flegal representative, when applicable) prior to any study-related data being collected.\n* Participant is willing and able to adhere to the protocol requirements.\n\nExclusion Criteria:\n\n* Participants who enroll into a non-Krystal Biotech, Inc. interventional gene therapy clinical trial will be excluded from the study.",{"count":175,"type":19},50,"OBSERVATIONAL","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 products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).",[29,28,154],"2021-06-07",{"date":181,"type":64},"2021-06-08",{"date":183,"type":64},"2021-05-25",{"date":185,"type":19},"2028-05-25",{"name":165,"class":166},3]