[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hereditary-angioedema\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hereditary-angioedema":22},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,42,60,76,103,133],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":4,"targetDuration":4,"studyType":19,"phases":4,"briefSummary":20,"conditions":21,"keywords":23,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":39,"locationsCount":4},"100651319","eap-for-deucrictibant-immediate-release-100651319",false,"NCT07759141","EAP for Deucrictibant Immediate-release","Expanded Access for Deucrictibant IR Capsule","Inclusion Criteria:\n\n* Provision of informed consent: Participants must provide written informed consent. Adolescents (≥12 to \\\u003C18 years old, or as per local law) require consent of parent or legally designated representative\u002Fguardian and must assent. If an adolescent reaches adulthood during the EAP, they must sign the adult informed consent form (ICF) to remain in the EAP.\n* Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent\u002Fassent.\n* Confirmed diagnosis of HAE.\n\n  1. for participants with hereditary angioedema with normal C1 inhibitor (HAE-nC1INH): documented genetic mutation associated with HAE-nC1INH\n\n     OR\n  2. if no documented mutation:\n\n     * clinical diagnosis with family history of HAE-nC1INH,\n     * attacks not responding to treatment with high-dose antihistamine (cetirizine 40 milligrams \\[mg\\]\u002Fday or equivalent high-dose second-generation antihistamine medication) and no clinical attack symptom relief if treated with corticosteroid, montelukast, and\u002For omalizumab.\n     * documented effective attack symptom relief with on-demand icatibant treatment\n* No satisfactory treatment options are available among currently approved HAE therapies for treatment of acute attacks ((i.e. Berinert (C1 esterase inhibitor \\[human\\]), Ekterly (sebetralstat), Firazyr (icatibant), Kalbitor (ecallantide), Ruconest (C1 esterase inhibitor \\[recombinant\\])) based on inadequate response, contraindication, safety\u002Ftolerability concerns, and\u002For other reasons, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation.\n* Not eligible for an ongoing clinical study or for whom participation in a clinical study is not possible or feasible for other reasons, including but not limited to clinical study eligibility criteria, geographic accessibility, study availability, timing considerations, participant decision to participate, or other participant-specific factors, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation.\n* Residence in the US or US territories.\n\nExclusion Criteria:\n\n* Pregnancy or nursing: any female participant who is pregnant, planning to become pregnant during the EAP, or currently breastfeeding.\n* Any diagnosis of angioedema other than HAE.\n* Significant comorbidity: any clinically significant comorbidity or systemic dysfunction (e.g., cardiovascular, gastrointestinal, renal, neurologic, respiratory) that, in the opinion of the treating physician, would interfere with the participant's safety or ability to participate in this EAP.\n* Severe hepatic impairment (Child-Pugh Class C).\n* Substance abuse: history of alcohol or drug abuse within the past year, or current evidence of substance dependence or abuse.\n* Prior treatment with deucrictibant IR resulting in discontinuation due to lack of efficacy, safety concerns, or tolerability issues.\n* Participation in another investigational drug study, or treatment with any investigational drug within 30 days or 5 half-lives (whichever is longer) prior to enrolment.\n* Prior gene therapy use for any indication at any time.\n* Current use of medications with systemic absorption that are strong CYP3A4 inhibitors (e.g., clarithromycin, itraconazole, ketoconazole, ritonavir) or strong CYP3A4 inducers (e.g., carbamazepine, phenytoin) within 30 days (or 5 half-lives, whichever longer) prior to enrolment.","ALL","12 Years","75 Years","EXPANDED_ACCESS","This is an expanded access program (EAP) designed to allow access to deucrictibant immediate-release (IR) capsule for eligible participants with hereditary angioedema (HAE) in the United States (US) and US territories who lack satisfactory alternative treatment options for on-demand treatment of HAE attacks, as determined by their treating physician.",[22],"Hereditary Angioedema",[24,22,25,26,27,28,29,30,31,32,33],"Angioedema","HAE","Deucrictibant","Deucrictibant immediate-release","Bradykinin","Bradykinin B2 receptor","Hereditary angioedema with C1 inhibitor deficiency\u002Fdysfunction","HAE-C1INH","Hereditary angioedema with normal C1 inhibitor","HAE-nC1INH","AVAILABLE","2026-08-05",{"date":37,"type":38},"2026-08-12","ACTUAL",{"name":40,"class":41},"Pharvaris Netherlands B.V.","INDUSTRY",{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":19,"phases":4,"briefSummary":50,"conditions":51,"keywords":52,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":56,"lastUpdatePostDateStruct":57,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":58,"locationsCount":4},"100548012","donidalorsen-expanded-access-program-for-patients-with-hereditary-angioedema-100548012","NCT06415448","Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema","Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)","Inclusion Criteria:\n\n* Patients in the ISIS 721744-CS3 (Open-Label Extension \\[OLE\\] Study) who have completed 205 weeks of treatment.\n* Female patients of childbearing potential, and male patients with partners of childbearing potential must be willing to use acceptable contraception, or refrain from sexual activity.\n\nExclusion Criteria:\n\n* Any patient who is pregnant or plans to become pregnant.\n* Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of treatment.","18 Years","The purpose of the Expanded Access Program is to provide pre-approval access of donidalorsen to eligible patients with Hereditary Angioedema (HAE) who complete the ISIS 721744-CS3 clinical trial.",[22],[25,53,54,55],"IONIS PKK-LRx","Donidalorsen","ISIS 721744","2026-08-03",{"date":35,"type":38},{"name":59,"class":41},"Ionis Pharmaceuticals, Inc.",{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":65,"acronym":4,"eligibilityCriteria":66,"healthyVolunteers":11,"sex":16,"minAge":67,"maxAge":68,"enrollmentInfo":4,"targetDuration":4,"studyType":19,"phases":4,"briefSummary":69,"conditions":70,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":74,"locationsCount":5},"100609576","treatment-of-angioedema-attacks-in-pediatric-ages-2-11-post-trial-and-naive-patients-with-hae-with-sebetralstat-100609576","NCT07216378","Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat","On Demand Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With Hereditary Angioedema (HAE) With Sebetralstat","Inclusion Criteria:\n\n* Male or female patients 2 to 11 years of age.\n* Parent or LAR provides signed informed consent and patient provides assent (when applicable).\n* Confirmed diagnosis of HAE.\n\nExclusion Criteria:\n\n* Confirmed diagnosis of HAE with nC1-INH or acquired angioedema\n* Patient weighs \\\u003C9.5 kg\n* Patient participated in the KVD900-303 trial and withdrew prior to trial completion per the protocol or trial closure\n* Any clinically significant medical condition or medical history that, in the opinion of the Treating Physician, would interfere with the patient's safety.\n* Known hypersensitivity to sebetralstat or its excipients.\n* Patient with a medical history or known to have severe hepatic impairment (Child Pugh C).\n* Patients who require sustained use of strong cytochrome P450 3A4 inhibitors or inducers.","2 Years","11 Years","The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve patients for the on-demand treatment of angioedema attacks where the treating Physician determines they might benefit from this treatment.",[22],"2026-07-22",{"date":73,"type":38},"2026-07-23",{"name":75,"class":41},"KalVista Pharmaceuticals, Ltd.",{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":83,"targetDuration":4,"studyType":86,"phases":87,"briefSummary":89,"conditions":90,"keywords":91,"overallStatus":93,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":102},"100641514","phase-4-safety-and-effectiveness-of-sebetralstat-kvd900-for-short-term-prophylaxis-before-procedures-in-people-with-hereditary-angioedema-kontrol-100641514","NCT07654829","Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)","A Phase 4, Open-Label Trial to Evaluate the Safety and Effectiveness of Sebetralstat When Used as Preprocedural Short-term Prophylaxis in Patients With Hereditary Angioedema","Inclusion Criteria:\n\n1. Male or female patients ≥12 years of age at time of Screening\n2. Confirmed diagnosis of HAE Type I or II based on patient report\n3. Patient has a qualifying procedure that is scheduled or planned to be scheduled to occur within 8 weeks of Screening\n4. Patient has access to a commercial available on-demand treatment for HAE\n5. Female patients of childbearing potential, including post-menarchal adolescents, must meet contraception requirements per Section 11.3. (Note: male patients do not require contraception)\n6. Patients must be able to swallow trial tablets whole\n7. Patients, as assessed by the Investigator, must be able to appropriately receive and store IMP, and be able to read, understand, and complete the questionnaire\n8. Investigator believes that the patient is willing and able to adhere to all protocol requirements through the duration of the trial\n9. Patient provides informed consent or assent (when applicable). A parent or legally authorized representative (LAR) must also provide signed informed consent when required\n\nExclusion Criteria:\n\n1. A clinically significant history of poor response to bradykinin receptor 2 blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator\n2. Use of angiotensin-converting enzyme (ACE) inhibitors within 7 days prior to the Screening Visit\n3. Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit\n4. Patients who are taking strong cytochrome P450 (CYP)3A4 inhibitors or inducers or moderate CYP3A4 inducers within 2 weeks of the procedure\n5. Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial\n6. History of substance abuse or dependence that would interfere with the completion of the trial, as determined by the Investigator\n7. Known hypersensitivity to sebetralstat or to any of the excipients\n8. Participation in any gene therapy treatment or trial for HAE\n9. Any pregnant or breastfeeding patient",{"count":84,"type":85},100,"ESTIMATED","INTERVENTIONAL",[88],"PHASE4","This is a Phase 4, prospective, open-label trial to evaluate the safety and effectiveness of sebetralstat when used for STP for a qualifying procedures in patients 12 years of age or older with hereditary angioedema (HAE).",[22],[92],"KVD900","NOT_YET_RECRUITING","2026-07-14",{"date":96,"type":38},"2026-07-16",{"date":98,"type":85},"2026-07-31",{"date":100,"type":85},"2027-12-06",{"name":75,"class":41},1,{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":4,"enrollmentInfo":111,"targetDuration":4,"studyType":86,"phases":113,"briefSummary":115,"conditions":116,"keywords":119,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":126,"completionDateStruct":128,"leadSponsor":130,"locationsCount":132},"100589884","phase-3-stop-hae-a-phase-3-study-of-adx-324-in-hae-100589884","NCT06960213","STOP-HAE: A Phase 3 Study of ADX-324 in HAE","STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema","STOP-HAE","Key Inclusion Criteria:\n\n* Age ≥18 years at the time of signing informed consent.\n* Have a documented diagnosis of HAE-1\u002FHAE-2 (Type I or II)\n* Experience ≥1 Investigator-confirmed HAE attack in the first 4 weeks of Screening or ≥2 Investigator-confirmed HAE attacks in 8 weeks of Screening\n* Able to use at least one acute therapy to treat HAE attacks (such as a plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist)\n\nKey Exclusion Criteria:\n\n* Concurrent diagnosis of another form of recurrent angioedema (such as acquired angioedema, HAE with normal C1-INH (previously known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria).\n* Any clinically significant renal disease\n* Any clinically significant hepatic disease\n* Have used any of the following for long-term prevention of HAE attacks:\n\n  1. C1-INH agent (CINRYZE, HAEGARDA, RUCONEST) within 2 weeks prior to Screening.\n  2. Berotralstat (ORLADEYO) within 3 weeks prior to Screening.\n  3. Lanadelumab (TAKHZYRO) within 8 weeks prior to the Screening.\n  4. Androgen use within 12 weeks prior to Screening.\n* Received prior treatment with any RNA\u002FDNA-based therapy for HAE or intolerant to any prior RNA\u002FDNA-based therapy for any condition, excluding vaccines.",{"count":112,"type":85},90,[114],"PHASE3","This study will evaluate the efficacy and safety of ADX-324 in participants with Type 1 or Type 2 hereditary angioedema. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life measures.",[22,25,117,118],"Hereditary Angioedema - Type 1","Hereditary Angioedema - Type 2",[25,120,121],"Hereditary angioedema","onvuzosiran","RECRUITING","2026-06-12",{"date":125,"type":38},"2026-06-16",{"date":127,"type":38},"2025-08-28",{"date":129,"type":85},"2027-12-31",{"name":131,"class":41},"ADARx Pharmaceuticals, Inc.",51,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":140,"targetDuration":142,"studyType":143,"phases":4,"briefSummary":144,"conditions":145,"keywords":171,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":177,"lastUpdatePostDateStruct":178,"startDateStruct":180,"completionDateStruct":182,"leadSponsor":184,"locationsCount":102},"100560175","institutional-registry-of-rare-diseases-100560175","NCT06573723","Institutional Registry of Rare Diseases","Institutional Registries of Rare Diseases at Hospital Italiano de Buenos Aires (HIBA)","Inclusion Criteria:\n\n* Clinical and\u002For molecular diagnosis of any of the following rare diseases: Amyloidosis, Sarcoidosis, Phacomatosis, Pheochromocytoma, Paraganglioma, Von Hippel-Lindau Disease, Immunoglobulin G4-Related Disease, Demyelinating Diseases, Inborn Errors of Metabolism, Eosinophilic Gastrointestinal Disorders, Hypertrophic Cardiomyopathy, Gaucher Disease, Congenital Adrenal Hyperplasia, Hereditary Angioedema, Pulmonary Hypertension, Wilson Disease, Vascular Anomalies, Mastocytosis, Multiple Endocrine Neoplasia, Inflammatory Bowel Diseases, Prader-Willi Syndrome, Hirschsprung Disease, or Cushing Syndrome.\n* Must be followed at Hospital Italiano de Buenos Aires.\n\nExclusion Criteria:\n\n\\- Refusal to participate in the study or in the informed consent process.",{"count":141,"type":85},380,"10 Years","OBSERVATIONAL","The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).\n\nMoreover, the specific goals are to generate an alert system for possible cases of RD with data from the electronic medical record, to describe the occurrence of RD in the evaluated population, to characterize the population, to describe patterns of diagnosis and treatment of RD present at the time, and to explore patient-reported outcomes.",[146,147,148,149,150,151,152,153,154,155,156,157,158,159,22,160,161,162,163,164,165,166,167,168,169,170],"Rare Diseases","Amyloidosis","Sarcoidosis","Phacomatosis","Pheochromocytoma","Paraganglioma","Von Hippel-Lindau Disease","Immunoglobulin G4-Related Disease","Demyelinating Diseases","Inborn Errors of Metabolism","Eosinophilic Gastrointestinal Disorders","Hypertrophic Cardiomyopathy","Gaucher Disease","Congenital Adrenal Hyperplasia","Pulmonary Hypertension","Wilson Disease","Vascular Anomalies","Mastocytosis","Multiple Endocrine Neoplasia","Inflammatory Bowel Diseases","Prader-Willi Syndrome","Hirschsprung Disease","Cushing Syndrome","HHT","Hemorrhagic Hereditary Telangiectasia",[172,173,174,149,175,176,152,153,154,155,156,157,158,159,22,160,161,162,163,164,165,166,167,168,170],"rare diseases","amyloidosis","sarcoidosis","pheochromocytoma","paraganglioma","2026-01-12",{"date":179,"type":38},"2026-01-14",{"date":181,"type":38},"2024-07-01",{"date":183,"type":85},"2034-12-31",{"name":185,"class":186},"Hospital Italiano de Buenos Aires","OTHER"]