[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Krystal Biotech, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":221},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,40,71,98,129,152,176,202],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100478043","phase-1-a-study-assessing-kb407-for-the-treatment-of-cystic-fibrosis-100478043",false,"NCT05504837","A Study Assessing KB407 for the Treatment of Cystic Fibrosis","KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis","Inclusion Criteria:\n\n1. The subject must have read, understood, and signed an Institutional Review Board\u002FEthics Committee (IRB\u002FIEC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions\n2. Subjects aged 18 years or older at the time of Informed Consent\n3. A confirmed diagnosis of CF as defined by clinical signs and symptoms of CF and at least one of the following:\n\n   * A historical sweat chloride value \\>60 mmol\u002FL\n   * Two copies of a disease causing mutation in the CFTR gene\n4. Clinically stable in the opinion of the Investigator\n5. Percent predicted FEV1 ≥40% and ≤90% of the predicted normal for age, gender, and height at Screening\n6. Resting oxygen saturation ≥92% on room air at Screening\n\nExclusion Criteria:\n\n1. Initiation of any new chronic therapy (eg, CFTR modulator, hypertonic saline, inhaled antibiotic) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to the first dose\n2. Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness 14 days prior to the first dose that, in the opinion of the Investigator, may confound study results\n3. Treatment for Mycobacterium abscessus within 3 months prior to the first dose or more than two pulmonary exacerbations with a history of Burkholderia cenocepacia infection within 6 months prior to the first dose\n4. Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to the first dose\n5. History of or listed for solid organ transplantation\n6. Any condition (including a history or current evidence of substance abuse or dependence, uncontrolled asthma, or is considered to be immunocompromised) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related procedures and\u002For poses an additional risk to the assessment of safety of KB407\n7. An active oral herpes infection 30 days prior to the first dose\n8. Women who are pregnant or nursing\n9. Subject who is unwilling to comply with contraception requirements per protocol\n10. Clinically significant abnormalities of hematology or chemistry testing at Screening that the Investigator believes may interfere with the assessment of safety and\u002For efficacy of the study treatment\n11. Subject has a known hypersensitivity to inhaled glycerol\n12. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator\n13. Bronchoscopy participants only: Unable to tolerate bronchoscopy procedure and airway sampling, in the opinion of the Investigator\n14. Cohort 4 participants only: Subjects who are ineligible for, do not tolerate, or do not benefit from modulator therapy","ALL","18 Years",{"count":19,"type":20},20,"ESTIMATED","INTERVENTIONAL",[23],"PHASE1","This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.",[26],"Cystic Fibrosis","RECRUITING","2026-05-01",{"date":30,"type":31},"2026-05-07","ACTUAL",{"date":33,"type":31},"2023-06-30",{"date":35,"type":20},"2027-03",{"name":37,"class":38},"Krystal Biotech, Inc.","INDUSTRY",6,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":46,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":4,"enrollmentInfo":49,"targetDuration":4,"studyType":21,"phases":51,"briefSummary":53,"conditions":54,"keywords":59,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":70},"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":50,"type":20},16,[52],"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).",[55,56,57,58],"Dystrophic Epidermolysis Bullosa","DEB - Dystrophic Epidermolysis Bullosa","Recessive Dystrophic Epidermolysis Bullosa","Dominant Dystrophic Epidermolysis Bullosa",[55,60,61],"DEB","Corneal Abrasions","2026-03-11",{"date":64,"type":31},"2026-03-12",{"date":66,"type":31},"2025-06-20",{"date":68,"type":20},"2026-12",{"name":37,"class":38},5,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":77,"eligibilityCriteria":78,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":79,"targetDuration":4,"studyType":21,"phases":81,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":97},"100592922","phase-1-a-study-comparing-kb801-verse-placebo-in-patients-with-stage-2-or-3-neurotrophic-keratitis-100592922","NCT06999733","A Study Comparing KB801 Verse Placebo in Patients With Stage 2 or 3 Neurotrophic Keratitis","A Phase 1\u002F2, Multicenter, Double-Masked, Placebo-Controlled Study of KB801 in Subjects With Stage 2 or 3 Neurotrophic Keratitis","EMERALD-1","Inclusion Criteria:\n\n1. The subject must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions.\n2. Aged ≥ 18 years at the time of informed consent.\n3. Diagnosis of Stage 2 or Stage 3 NK, as defined by the Mackie criteria.\n4. Persistent corneal epithelial defect (PCED) between Screening 1 and Screening 2 without clinical improvement during that time. PCED is defined as a focal loss of the cornea's outermost cell layer and can include involvement of the deeper stromal layers (i.e., corneal ulcer).\n5. PCED at Screening 1 must have a minimum of 1 mm of any linear measurement of defect.\n6. A negative pregnancy test at Screening 1 and Day 1 for women of child-bearing potential.\n\nExclusion Criteria:\n\n1. An active ocular infection (bacterial, viral, fungal or protozoal) or active ocular inflammation not related to NK in the study eye as assessed by Investigator at Screening 1 or 2.\n2. Use of any prohibited therapies at Screening or anticipated need for topical treatments other than the investigational product or products allowed per protocol in the study eye from Screening through the study Treatment Period\n\n   a. Sufficient washout of prohibited therapies must occur prior to meeting eligibility criteria.\n3. Anticipated use of therapeutic contact lenses or contact lens wear for refractive correction during the study Treatment Period in the study eye.\n4. Evidence of corneal ulceration involving the posterior third of the corneal stroma, corneal melting, descemetocele, impending or perforation in the study eye at the time of Screening 1 or 2.\n5. PCED at Screening 1 or 2 of \\> 8 mm maximum linear measurement in any eye.\n6. The central 3 mm of corneas contain PCED bilaterally at Screening 1 or 2.\n7. Anticipated need for punctal occlusion of the study eye during the Treatment Period.\n8. Presence or history of any ocular or systemic disorder or condition that might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the Investigator to be incompatible with the study visit schedule or conduct (e.g., progressive or degenerative corneal or retinal conditions, uveitis, optic neuritis, poorly controlled diabetes, autoimmune disease, systemic infection, neoplastic diseases).\n9. Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to Day 1 (participation in a non-interventional observational study is permitted).\n10. Intraocular surgery on the eligible eye(s) within 6 months prior to Screening 1, and any ophthalmic condition that may require surgery on the study eye during the Treatment Period. An exception to the preceding statement is allowed if, in the opinion of the Investigator, the ocular surgery is deemed the cause of the NK.\n11. Corneal transplantation (i.e., keratoplasty) of the study eye within the past 2 years or a history of multiple keratoplasty operations to the study eye regardless of timing. This includes full- or partial-thickness, or endothelial keratoplasty.\n12. Subject who is pregnant or nursing.\n13. Subject who is unwilling to comply with contraception requirements per-protocol (for subjects of childbearing potential).\n14. Subject who is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.",{"count":80,"type":20},60,[23,82],"PHASE2","The Sponsor is developing KB801, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human nerve growth factor (NGF) to the corneas of people with Stage 2 or 3 neurotrophic keratitis (NK) via topical application. This is a Phase 1\u002F2, multicenter, double-masked, placebo-controlled study to evaluate the safety, tolerability, and preliminary efficacy of KB801 in these subjects.",[85],"Neurotrophic Keratitis",[85,87,88],"Persistent Corneal Epithelial Defect","Corneal Epithelial Defect","2026-02-25",{"date":91,"type":31},"2026-02-27",{"date":93,"type":31},"2025-07-07",{"date":95,"type":20},"2027-12",{"name":37,"class":38},9,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":21,"phases":108,"briefSummary":109,"conditions":110,"keywords":116,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":128},"100533631","phase-1-a-study-assessing-kb707-for-the-treatment-of-advanced-solid-tumor-malignancies-affecting-the-lungs-100533631","NCT06228326","A Study Assessing KB707 for the Treatment of Advanced Solid Tumor Malignancies Affecting the Lungs","KB707-02: A Phase 1\u002F2 Study of Inhaled KB707 in Patients With Advanced Solid Tumor Malignancies Affecting the Lungs","KYANITE-1","Key Inclusion Criteria:\n\n* Age 18 years or older at the time of informed consent\n* Life expectancy \\>12 weeks\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1\n* Have at least one measurable lung lesion per RECIST v1.1 at Screening\n* Cohorts 1 through 4 only: Histologically confirmed diagnosis of advanced solid tumor malignancy affecting the lungs and the individual has progressed on standard of care therapy, cannot tolerate standard of care therapy, refused standard of care therapy, or has no standard of care therapy.\n* Cohorts 5, 6, and 7 only: (1) Histologically or cytologically confirmed diagnosis of stage 3 or 4 NSCLC, as per American Joint Committee on Cancer (AJCC) staging system (8th edition) and (2) Subject must meet the following criteria of prior lines of therapy:\n\n  1. Subject has previously received no more than one line of prior immune checkpoint inhibitor (ICI) with or without platinum-based chemotherapy, or no more than two prior lines of therapy when given the ICI and platinum-based chemotherapy sequentially as two separate lines.\n  2. Subjects with an actionable mutation (e.g., EGFR, KRAS, ALK, or ROS1 genomic alteration), are permitted to have received one additional line of approved targeted therapy.\n\nKey Exclusion Criteria:\n\n* Not fully recovered from prior surgery or radiotherapy, including all radiation-related toxicities\n* The subject is pregnant, nursing, or plans to become pregnant during study treatment and through three months after the last dose of KB707\n* Have known history of positive human immunodeficiency virus (HIV 1\u002F2)\n* Cohorts 5, 6, and 7 only:\n\n  1. Subject has a known additional malignancy that is progressing or requires active treatment\n  2. Subject has active brain metastases or leptomeningeal metastases\n  3. Prior anti-PD-1\u002FPD-L1 therapy was intolerable and required discontinuation of treatment\n  4. Subject has active, known, or suspected autoimmune disease requiring systemic treatment\n  5. Subject has known acute or chronic hepatitis\n  6. Subject has active pneumonitis or history of ICI-induced pneumonitis that required steroids",{"count":107,"type":20},250,[23,82],"The Sponsor is developing KB707, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector designed to stimulate an anti-tumor immune response through the production of cytokines within the local tumor microenvironment in the lungs. KB707 is administered via nebulization, delivering the therapy directly through the airways to the lungs of subjects with advanced solid tumor malignancies.\n\nThis is a Phase 1\u002F2, open-label, multicenter, dose escalation and expansion study designed to evaluate the safety, tolerability, preliminary efficacy, and immunologic effect of KB707.\n\nMonotherapy KB707 dose escalation and expansion cohorts enrolled adults with advanced solid tumor malignancies affecting the lungs who had progressed on standard of care therapy, cannot tolerate standard of care therapy, or refused standard of care therapy. The dose escalation phase (Cohorts 1 and 2) evaluated KB707 monotherapy using a standard 3+3 design, followed by a dose expansion phase (Cohort 4) to further evaluate the selected dose. Subjects received inhaled KB707 weekly for three weeks, then every three weeks. The dose escalation portion of the study is now complete, and the selected dose is being evaluated in the expansion phase.\n\nCombination regimens with a selected (fixed) dose of KB707 are being evaluated in subjects with advanced or metastatic non-small cell lung cancer (NSCLC). Subjects in Cohorts 5 and 6 are receiving inhaled KB707 once every 2 weeks (q2w), delivered in combination with Keytruda once every 6 weeks. Subjects in Cohort 7 are receiving inhaled KB707 in combination with docetaxel once every 3 weeks.\n\nAll subjects will be treated until tumor progression, death, unacceptable toxicity, symptomatic deterioration, achievement of maximal response, subject choice, Investigator decision to discontinue treatment, or the Sponsor determines to terminate the study.",[111,112,113,114,115],"Lung Cancer, Non-small Cell","Lung Cancer Metastatic","Solid Tumor, Adult","Advanced Cancer","Lung Cancer (NSCLC)",[117,118,119],"cancer","Krystal Biotech","lung cancer","2025-10-22",{"date":122,"type":31},"2025-10-24",{"date":124,"type":31},"2024-04-17",{"date":126,"type":20},"2028-07",{"name":37,"class":38},14,{"id":130,"slug":131,"hasResults":11,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":135,"eligibilityCriteria":136,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":137,"enrollmentInfo":138,"targetDuration":4,"studyType":21,"phases":140,"briefSummary":141,"conditions":142,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":150,"locationsCount":151},"100519863","phase-1-a-study-of-kb408-for-the-treatment-of-alpha-1-antitrypsin-deficiency-100519863","NCT06049082","A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","Serpentine-1","Inclusion Criteria:\n\n1. The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions.\n2. Subject is aged ≥18 to ≤70 years, at the time of informed consent.\n3. Subject has a genetically confirmed diagnosis of AATD with a PI\\*ZZ or PI\\*ZNull genotype.\n4. Cohort 2b and Cohort 3: Subjects receiving AAT augmentation therapy must be willing to washout for at least 10 days prior to Screening and be willing to remain off augmentation therapy for the duration of the study.\n5. Cohort 2b and Cohort 3: Serum AAT level \\\u003C11 μM at Screening.\n6. Willing to remain on a stable regimen of treatment during the study.\n7. Resting oxygen saturation ≥92% on room air at Screening.\n8. Clinically stable and in good general health, except for AATD, as determined by the Investigator.\n\nExclusion Criteria:\n\n1. Pulmonary function test with percent predicted forced expired volume in 1 second (ppFEV1) after inhalation of a bronchodilator is \\\u003C40% at Screening.\n2. Diffusing capacity of the lungs for carbon monoxide (DLCO) \\\u003C30 percent predicted (historical DLCO within 2 years prior to Screening without any intervening change in clinical status since the measurement was taken, or as measured at Screening).\n3. Known ongoing or history of clinically significant pulmonary impairment other than AATD.\n4. A pulmonary exacerbation within six weeks (42 days) of first dose.\n5. Initiation of any new chronic therapy or any change in ongoing therapy routine within 28 days of first dose.\n6. Participation in another interventional clinical study or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, of first dose. Previous treatment with a genetic therapy for AATD, where the investigational product was demonstrated to be non-efficacious, is not exclusionary.\n7. History of or listed for solid organ transplantation or has undergone major lung surgery (e.g., lobectomy) within 6 months of first dose.\n8. Any clinical condition or illness (including a history or current evidence of substance abuse or dependence) that, in the opinion of the Investigator, would impact a subject's ability to complete all study-related procedures and\u002For poses an additional risk to the assessment of safety of KB408.\n9. An active oral herpes infection 30 days prior to the first dose.\n10. Clinically significant hepatic dysfunction defined as any one of the following:\n\n    1. AST and ALT ≥3× upper limit of normal (ULN) at Screening\n    2. Total bilirubin ≥2× ULN at Screening (unless associated with Gilbert's syndrome)\n    3. Evidence of liver cirrhosis with clinical manifestations of portal hypertension (e.g., ascites, encephalopathy, variceal hemorrhage)\n11. History of cigarette smoking or any other tobacco use, or use of e-cigarettes or other recreational inhalant, within 6 months of Screening.\n12. Unwilling to refrain from smoking, e-cigarette use, or vaping throughout the duration of the study.\n13. A positive urine cotinine result that is consistent with active smoking at Screening. (A positive cotinine test due to nicotine replacement therapy for the purpose of smoking cessation, as attested by the Investigator, is allowed.)\n14. Abnormal hematology or chemistry testing at Screening as defined below, or any other clinically significant abnormalities that the Investigator believes may interfere with the assessment of safety of the study treatment.\n\n    * Platelet count \\\u003C100×10\\^9\u002FL\n    * Hemoglobin \\\u003C9 g\u002FdL\n    * White blood cell count \\\u003C3 or \\>15×10\\^9\u002FL\n    * Sodium \\\u003C130 or \\>150 mmol\u002FL\n    * Potassium \\\u003C3 or \\>5.5 mmol\u002FL\n    * Carbon dioxide \\\u003C16 mmol\u002FL\n    * Creatinine \\>2 mg\u002FdL\n15. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator.\n16. Females who are pregnant or nursing.\n17. Subject who is unwilling to comply with contraception requirements per protocol","70 Years",{"count":139,"type":20},15,[23],"The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\\*ZZ or PI\\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.",[143],"Alpha 1-Antitrypsin Deficiency","2025-07-17",{"date":146,"type":31},"2025-07-22",{"date":148,"type":31},"2024-02-15",{"date":68,"type":20},{"name":37,"class":38},3,{"id":153,"slug":154,"hasResults":11,"nctId":155,"briefTitle":156,"officialTitle":156,"acronym":4,"eligibilityCriteria":157,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":4,"enrollmentInfo":158,"targetDuration":4,"studyType":160,"phases":4,"briefSummary":161,"conditions":162,"keywords":166,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":167,"lastUpdatePostDateStruct":168,"startDateStruct":170,"completionDateStruct":172,"leadSponsor":174,"locationsCount":175},"100559383","a-natural-history-study-of-corneal-abrasions-in-patients-with-dystrophic-epidermolysis-bullosa-deb-100559383","NCT06563414","A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)","Inclusion Criteria:\n\n1. The subject or their respective legal guardian must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent\u002FAssent Form 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. Prior genetic diagnosis of DEB due to mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.",{"count":159,"type":20},40,"OBSERVATIONAL","This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).",[163,164,165],"Epidermolysis Bullosa Dystrophica","Epidermolysis Bullosa Dystrophica, Recessive","Epidermolysis Bullosa Dystrophica Dominans",[55,60,61],"2025-07-01",{"date":169,"type":31},"2025-07-02",{"date":171,"type":31},"2024-08-02",{"date":173,"type":20},"2025-12",{"name":37,"class":38},1,{"id":177,"slug":178,"hasResults":11,"nctId":179,"briefTitle":180,"officialTitle":181,"acronym":4,"eligibilityCriteria":182,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":183,"targetDuration":4,"studyType":21,"phases":185,"briefSummary":186,"conditions":187,"keywords":192,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":194,"lastUpdatePostDateStruct":195,"startDateStruct":197,"completionDateStruct":199,"leadSponsor":201,"locationsCount":139},"100513824","phase-1-a-study-assessing-kb707-for-the-treatment-of-locally-advanced-or-metastatic-solid-tumors-100513824","NCT05970497","A Study Assessing KB707 for the Treatment of Locally Advanced or Metastatic Solid Tumors","A Phase 1\u002F2, Open-Label, Multi-Center, Dose Escalation and Expansion Study of KB707 in Subjects With Locally Advanced or Metastatic Solid Tumor Malignancies","Key Inclusion Criteria:\n\n* Life expectancy \\>12 weeks\n* ECOG performance status of 0 or 1\n* Have measurable disease per RECIST v1.1 at Screening\n* Cohorts 1-4 only: Histologically confirmed diagnosis of a locally advanced or metastatic solid tumor and the individual has progressed on standard of care therapy, cannot tolerate standard of care therapy, refused standard of care therapy, or there is no standard of care therapy.\n* Cohorts 5 and 6 only: Histologically confirmed stage III (unresectable) or stage IV melanoma, as per American Joint Committee on Cancer (AJCC) staging system (8th edition; AJCC 2017) and\n\n  1. Subject has previously failed one prior anti-PD-1\u002FPD-L1 treatment (as monotherapy or in combination with other checkpoint inhibitors such as anti-LAG-3 or anti-CTLA-4); and\n  2. If proto-oncogene B-Raf (BRAF) V600 mutation-positive, subject previously failed a BRAF inhibitor or BRAF inhibitor in combination with mitogen-activated extracellular signal-regulated kinase (MEK) inhibitor\n* Cohort 5 only: Age 12 years or older at the time of informed consent\n* Cohort 6 only: Age 18 years or older at the time of informed consent\n\nKey Exclusion Criteria:\n\n* Prior surgery or radiation therapy must be fully recovered, including all radiation -related toxicities and subject does not require systemic corticosteroids\n* The subject is pregnant, nursing, or plans to become pregnant during study treatment and through three months after the last dose of KB707\n* Have known history of positive human immunodeficiency virus (HIV 1\u002F2)\n* Cohorts 5 and 6 only:\n\n  1. Subject has a known additional malignancy that is progressing or requires active treatment.\n  2. Subject has uveal\u002Focular melanoma.\n  3. The subject has active brain metastases or leptomeningeal metastases\n  4. Subject has received more than 2 lines of systemic therapy for unresectable or metastatic melanoma\n  5. Prior anti-LAG-3 and\u002For anti-PD-1 therapy was intolerable and required discontinuation of treatment",{"count":184,"type":20},240,[23,82],"KB707-01 is a Phase 1\u002F2, open-label, multicenter, dose escalation and expansion study. The study will evaluate the safety and tolerability of KB707 in adults with locally advanced or metastatic solid tumors who have progressed on standard of care therapy, cannot tolerate standard of care therapy, refused standard of care therapy, or for whom there is no standard of care therapy as well as the safety, tolerability, preliminary efficacy, and immunologic effect of KB707 administered in combination with Opdualag to subjects with unresectable or metastatic melanoma.\n\nSubjects in dose escalation (Cohorts 1 through 3) and dose expansion (Cohort 4) will receive intratumoral injections of KB707 approximately every three weeks. Cohorts 1 through 4 are closed to new enrollment. Dose expansion Cohort 5 and Cohort 6 will evaluate subjects with advanced melanoma. Subjects in Cohort 5 will receive intratumoral injections of KB707 biweekly (q2w), delivered in combination with Opdualag (dosed every q4w per prescribing information). Subjects in Cohort 6 will receive intratumoral injections of KB707 biweekly (q2w), delivered in combination with Keytruda (dosed every q6w per prescribing information). All subjects will be treated until disease progression, death, unacceptable toxicity, symptomatic deterioration, achievement of maximal response, subject choice, Investigator decision to discontinue treatment, or the Sponsor determines to terminate the study.",[188,189,190,191],"Cancer","Melanoma Stage III","Melanoma Stage IV","Cutaneous Melanoma",[118,117,193],"melanoma","2025-05-14",{"date":196,"type":31},"2025-05-18",{"date":198,"type":31},"2023-10-31",{"date":200,"type":20},"2027-07",{"name":37,"class":38},{"id":203,"slug":204,"hasResults":11,"nctId":205,"briefTitle":206,"officialTitle":207,"acronym":4,"eligibilityCriteria":208,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":4,"enrollmentInfo":209,"targetDuration":4,"studyType":160,"phases":4,"briefSummary":211,"conditions":212,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":216,"completionDateStruct":218,"leadSponsor":220,"locationsCount":151},"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":210,"type":20},50,"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).",[55,57,58],"2021-06-07",{"date":215,"type":31},"2021-06-08",{"date":217,"type":31},"2021-05-25",{"date":219,"type":20},"2028-05-25",{"name":37,"class":38},""]