[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100574097":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":30,"centralContacts":39,"locations":45,"responsibleParty":294,"collaborators":30,"id":296,"slug":297,"hasResults":298,"nctId":299,"briefTitle":300,"officialTitle":301,"acronym":30,"eligibilityCriteria":302,"healthyVolunteers":298,"sex":303,"minAge":304,"maxAge":305,"enrollmentInfo":306,"targetDuration":30,"studyType":309,"phases":310,"briefSummary":313,"conditions":314,"keywords":319,"overallStatus":102,"whyStopped":30,"lastUpdateSubmitDate":326,"lastUpdatePostDateStruct":327,"startDateStruct":330,"completionDateStruct":332,"leadSponsor":334,"locationsCount":335},{"fullName":5,"class":6},"Hemab ApS","INDUSTRY",[8,14,19],{"label":9,"type":10,"description":11,"interventionNames":12},"Part A Single Ascending Dose Design","EXPERIMENTAL","A multicenter study to evaluate the safety, tolerability, PK, and PD effect of single dose HMB-002 in participants with Type 1 VWD.",[13],"Drug: HMB-002 (Part A)",{"label":15,"type":10,"description":16,"interventionNames":17},"Part B Multiple Dose Assessment","A multicenter study to evaluate the safety, tolerability, PK, and PD effect of repeat doses of HMB-002, as well as the preliminary prophylactic effects on bleeding events.",[18],"Drug: HMB-002 (Part B)",{"label":20,"type":10,"description":21,"interventionNames":22},"Part C HMB-002 with Concomitant Factor Concentrate","A multicenter study to evaluate the safety and tolerability of a single dose of HMB-002, administered to patients concurrently receiving regular factor concentrate as standard of care.",[23],"Drug: HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)",[25,31,35],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"DRUG","HMB-002 (Part A)","HMB-002 will be administered subcutaneously. Part A will utilize sentinel dosing. The planned duration of study participants in Part A is approximately 12 weeks.",[9],null,{"type":26,"name":32,"description":33,"armGroupLabels":34,"otherNames":30},"HMB-002 (Part B)","HMB-002 will be administered subcutaneously. Part B dosing intervals will be determined following evaluation of Part A results. The planned duration of study participants in Part B will be approximately 21 weeks.",[15],{"type":26,"name":36,"description":37,"armGroupLabels":38,"otherNames":30},"HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)","HMB-002 will be administered as a single dose with a concomitant single dose of factor concentrate. The planned duration of study participants in Part C will be approximately 17 weeks.",[20],[40],{"name":41,"role":42,"phone":43,"phoneExt":30,"email":44},"Clinical Trials","CONTACT","080 8304 6409","clinicaltrials@hemab.com",[46,60,70,80,90,100,111,121,131,141,151,161,171,181,193,203,213,225,235,244,253,262,271,280,288],{"facility":47,"status":48,"city":49,"state":50,"zip":51,"country":52,"countryCode":53,"cosmosGeoPoint":54,"geoPoint":59,"contacts":30},"Phoenix Children's Hospital","NOT_YET_RECRUITING","Phoenix","Arizona","85016","United States","US",{"type":55,"coordinates":56},"Point",[57,58],-112.07404,33.44838,{"lat":58,"lon":57},{"facility":61,"status":48,"city":62,"state":63,"zip":64,"country":52,"countryCode":53,"cosmosGeoPoint":65,"geoPoint":69,"contacts":30},"Arkansas Children's Hospital","Little Rock","Arkansas","72202",{"type":55,"coordinates":66},[67,68],-92.28959,34.74648,{"lat":68,"lon":67},{"facility":71,"status":48,"city":72,"state":73,"zip":74,"country":52,"countryCode":53,"cosmosGeoPoint":75,"geoPoint":79,"contacts":30},"Children's Hospital of Los Angeles","Los Angeles","California","90027",{"type":55,"coordinates":76},[77,78],-118.24368,34.05223,{"lat":78,"lon":77},{"facility":81,"status":48,"city":82,"state":83,"zip":84,"country":52,"countryCode":53,"cosmosGeoPoint":85,"geoPoint":89,"contacts":30},"University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center","Miami","Florida","33136",{"type":55,"coordinates":86},[87,88],-80.19366,25.77427,{"lat":88,"lon":87},{"facility":91,"status":48,"city":92,"state":93,"zip":94,"country":52,"countryCode":53,"cosmosGeoPoint":95,"geoPoint":99,"contacts":30},"Emory Children's Center","Atlanta","Georgia","30329",{"type":55,"coordinates":96},[97,98],-84.38798,33.749,{"lat":98,"lon":97},{"facility":101,"status":102,"city":103,"state":104,"zip":105,"country":52,"countryCode":53,"cosmosGeoPoint":106,"geoPoint":110,"contacts":30},"Innovative Hematology, Inc.\u002FIndiana Hemophilia and Thrombosis Center","RECRUITING","Indianapolis","Indiana","46260",{"type":55,"coordinates":107},[108,109],-86.15804,39.76838,{"lat":109,"lon":108},{"facility":112,"status":48,"city":113,"state":114,"zip":115,"country":52,"countryCode":53,"cosmosGeoPoint":116,"geoPoint":120,"contacts":30},"Tulane University School of Medicine","New Orleans","Louisiana","70112",{"type":55,"coordinates":117},[118,119],-90.07507,29.95465,{"lat":119,"lon":118},{"facility":122,"status":48,"city":123,"state":124,"zip":125,"country":52,"countryCode":53,"cosmosGeoPoint":126,"geoPoint":130,"contacts":30},"University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders","Ann Arbor","Michigan","48109",{"type":55,"coordinates":127},[128,129],-83.74088,42.27756,{"lat":129,"lon":128},{"facility":132,"status":48,"city":133,"state":134,"zip":135,"country":52,"countryCode":53,"cosmosGeoPoint":136,"geoPoint":140,"contacts":30},"Mayo Clinic - Rochester","Rochester","Minnesota","55905",{"type":55,"coordinates":137},[138,139],-92.4699,44.02163,{"lat":139,"lon":138},{"facility":142,"status":48,"city":143,"state":144,"zip":145,"country":52,"countryCode":53,"cosmosGeoPoint":146,"geoPoint":150,"contacts":30},"Oregon Health & Science University","Portland","Oregon","97239",{"type":55,"coordinates":147},[148,149],-122.67621,45.52345,{"lat":149,"lon":148},{"facility":152,"status":48,"city":153,"state":154,"zip":155,"country":52,"countryCode":53,"cosmosGeoPoint":156,"geoPoint":160,"contacts":30},"Hemophilia Center of Western Pennsylvania","Pittsburgh","Pennsylvania","15213",{"type":55,"coordinates":157},[158,159],-79.99589,40.44062,{"lat":159,"lon":158},{"facility":162,"status":48,"city":163,"state":164,"zip":165,"country":52,"countryCode":53,"cosmosGeoPoint":166,"geoPoint":170,"contacts":30},"The University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"type":55,"coordinates":167},[168,169],-96.80667,32.78306,{"lat":169,"lon":168},{"facility":172,"status":48,"city":173,"state":174,"zip":175,"country":52,"countryCode":53,"cosmosGeoPoint":176,"geoPoint":180,"contacts":30},"Washington Institute For Coagulation (WIC)","Seattle","Washington","98101",{"type":55,"coordinates":177},[178,179],-122.33207,47.60621,{"lat":179,"lon":178},{"facility":182,"status":48,"city":183,"state":184,"zip":185,"country":186,"countryCode":187,"cosmosGeoPoint":188,"geoPoint":192,"contacts":30},"Fiona Stanley Hospital","Murdoch","Perth","WA 6150","Australia","AU",{"type":55,"coordinates":189},[190,191],115.83757,-32.06987,{"lat":191,"lon":190},{"facility":194,"status":102,"city":195,"state":196,"zip":197,"country":186,"countryCode":187,"cosmosGeoPoint":198,"geoPoint":202,"contacts":30},"Royal Prince Alfred Hospital","Camperdown","Sydney","NSW 2050",{"type":55,"coordinates":199},[200,201],151.17642,-33.88965,{"lat":201,"lon":200},{"facility":204,"status":102,"city":205,"state":206,"zip":207,"country":186,"countryCode":187,"cosmosGeoPoint":208,"geoPoint":212,"contacts":30},"The Alfred Hospital","Melbourne","Victoria","VIC 3004",{"type":55,"coordinates":209},[210,211],144.96332,-37.814,{"lat":211,"lon":210},{"facility":214,"status":102,"city":215,"state":216,"zip":217,"country":218,"countryCode":219,"cosmosGeoPoint":220,"geoPoint":224,"contacts":30},"Basingstoke and North Hampshire Hospital","Basingstoke","Hampshire","RG24 9NA","United Kingdom","UK",{"type":55,"coordinates":221},[222,223],-1.08708,51.26249,{"lat":223,"lon":222},{"facility":226,"status":48,"city":227,"state":228,"zip":229,"country":218,"countryCode":219,"cosmosGeoPoint":230,"geoPoint":234,"contacts":30},"St George's Hospital","Tooting","London","SW17 0QT",{"type":55,"coordinates":231},[232,233],-0.16394,51.42524,{"lat":233,"lon":232},{"facility":236,"status":48,"city":237,"state":228,"zip":238,"country":218,"countryCode":219,"cosmosGeoPoint":239,"geoPoint":243,"contacts":30},"Royal London Hospital","Whitechapel","E1 1FR",{"type":55,"coordinates":240},[241,242],-0.06583,51.51382,{"lat":242,"lon":241},{"facility":245,"status":48,"city":246,"state":30,"zip":247,"country":218,"countryCode":219,"cosmosGeoPoint":248,"geoPoint":252,"contacts":30},"University Hospitals Birmingham NHS Foundation Trust","Birmingham","B15 2TH",{"type":55,"coordinates":249},[250,251],-1.89983,52.48142,{"lat":251,"lon":250},{"facility":254,"status":102,"city":255,"state":30,"zip":256,"country":218,"countryCode":219,"cosmosGeoPoint":257,"geoPoint":261,"contacts":30},"University Hospital of Wales","Cardiff","CF14 4XW",{"type":55,"coordinates":258},[259,260],-3.18,51.48,{"lat":260,"lon":259},{"facility":263,"status":48,"city":264,"state":30,"zip":265,"country":218,"countryCode":219,"cosmosGeoPoint":266,"geoPoint":270,"contacts":30},"St James's University Hospital, Leeds Haemophilia Centre","Leeds","LS9 7TF",{"type":55,"coordinates":267},[268,269],-1.54785,53.79648,{"lat":269,"lon":268},{"facility":272,"status":48,"city":273,"state":30,"zip":274,"country":218,"countryCode":219,"cosmosGeoPoint":275,"geoPoint":279,"contacts":30},"Royal Liverpool and Broadgreen University Hospitals NHS TRUST, The Roald Dahl Haemostasis and Thrombosis Centre","Liverpool","L7 8XP",{"type":55,"coordinates":276},[277,278],-2.97794,53.41058,{"lat":278,"lon":277},{"facility":281,"status":102,"city":228,"state":30,"zip":282,"country":218,"countryCode":219,"cosmosGeoPoint":283,"geoPoint":287,"contacts":30},"Richmond Pharmacology","SE1 1YR",{"type":55,"coordinates":284},[285,286],-0.12574,51.50853,{"lat":286,"lon":285},{"facility":289,"status":48,"city":228,"state":30,"zip":290,"country":218,"countryCode":219,"cosmosGeoPoint":291,"geoPoint":293,"contacts":30},"St Thomas' Hospital","SE1 7EH",{"type":55,"coordinates":292},[285,286],{"lat":286,"lon":285},{"type":295,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100574097","phase-1-a-study-assessing-hmb-002-in-participants-with-von-willebrand-disease-100574097",false,"NCT06754852","A Study Assessing HMB-002 in Participants With Von Willebrand Disease","A Phase 1\u002F2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants With Von Willebrand Disease (Velora Pioneer)","Key Inclusion Criteria:\n\n1. Weight 50 to 120 kg, inclusive.\n2. Documented diagnosis of Congenital VWD, confirmed by laboratory testing consistent with ISTH\u002FASH) diagnostic guidelines).\n3. Vital signs are within normal ranges at Screening.\n4. Participants must meet the following baseline organ function, indicated by laboratory criteria as Screening:\n\n   1. Renal: Estimated glomerular filtration rate (eGFR) of ≥45 mL\u002Fmin\u002F1.73m\\^2.\n   2. Hepatic: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and total bilirubin ≤1.5 upper limit of normal (ULN) at Screening. For participants with a history of Gilbert's Syndrome, total bilirubin ≤2 × ULN.\n   3. Hematology \\>85 g\u002FL and platelet count \\>120 x 10\\^9\u002FL.\n\n   Part A Only:\n5. Age: ≥18 and \\\u003C70 years of age at the time of informed consent.\n6. VWD Subtype Eligibility:\n\n   * Cohorts A1 and A2: Participants with Type 1 VWD, only.\n   * Cohorts A3 and A4: Participants with Type 1 VWD (including Type 1C) and Type 2A VWD\n7. Residual VWF activity of ≤ 50 IU\u002FdL and FVIII activity ≤ 70 IU\u002FdL during screening.\n\n   Part B Only:\n8. Age: ≥16 and \\\u003C70 years of age at the time of informed consent.\n9. VWD Subtype Eligibility: Participants with Type 1 VWD (including Type 1C) and Type 2A.\n10. Residual VWF activity of ≤50 IU\u002FdL and FVIII activity ≤70 IU\u002FdL during screening.\n11. Symptomatic Disease: Participants must be symptomatic, typically reporting bleeding events on a monthly basis.\n12. Bleeding History (must meet one of the following):\n\n    1. Prior Observational Study Participation:\n\n       The participant must have participated in the observational study HMB-002-101\\_SCR (VELORA Discover), have a minimum annualized treated bleeding event (ATBR) of 3; OR\n    2. Medical Record-Documented Bleeding History:\n\n    The Investigator confirms that ≥3 treated bleeding events have been documented in the participant's medical record within the preceding 12 months.\n\n    Part C Only:\n13. Age: ≥18 and \\\u003C70 years of age at the time of informed consent.\n14. Participants with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII activity levels (VWF activity \\\u003C5 IU\u002FdL and FVIII activity \\\u003C10 IU\u002FdL).\n15. Receives regular VWF concentrate (at least 1\u002Fweek) as part of their routine care (usual dose ≤50 IU\u002Fkg).\n\nKey Exclusion Criteria:\n\n1. Personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial venous thrombosis.\n2. High risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL\u002FProthrombin gene mutation, Antithrombin deficiency with activity \\\u003C50%. Congenital Protein C and Protein S deficiency with levels \\\u003C50%.\n3. Body mass index (BMI) \\>35 kg\u002Fm\\^2 (obese, adjusted for ethnicity).\n4. Presence of other conditions that substantially increase risk of thrombosis either individually (for participants \\>65 years of age) or in combination (for participants ≤65 years of age), at the discretion of the Investigator or Medical Monitor.\n5. Clinically significant cardiovascular disease.\n6. Other known severe bleeding disorder(s) other than VWD.\n7. Requirement for concomitant medications that affect hemostasis (including, but not limited to anticoagulation, antiplatelet agents, certain non-steroidal anti-inflammatory drugs) and cannot refrain from use for 14 days prior to the first dose of study drug and throughout the study.\n\n   Exclusion Criteria for Part A and Part B Only\n8. Requirement for ongoing hemostatic treatment to prevent bleeding (bleed prophylaxis). Prophylaxis administered intermittently for procedures or surgery to reduce bleeding risk is permitted.","ALL","16 Years","69 Years",{"count":307,"type":308},108,"ESTIMATED","INTERVENTIONAL",[311,312],"PHASE1","PHASE2","This is a first-in-human (FIH), Phase 1\u002F2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) regimen design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy. Part C will evaluate the safety, PK, and PD of a single concomitant dose of HMB-002 and factor concentrate with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII who use factor concentrate as prophylaxis.",[315,316,317,318],"Von Willebrand Disease (VWD)","Von Willebrand Disease (VWD), Type 1","Von Willebrand Disease (VWD), Type 2","Von Willebrand Disease (VWD), Type 3",[320,321,322,323,324,325],"Von Willebrand Disease (VMD)","Type 1 VWD","Type 1 with low residual VWF and FVIII who use factor concentrate as prophylaxis","Type 2 VWD","Type 3 VWD","Von Willebrand Factor (VWF)","2026-06-26",{"date":328,"type":329},"2026-06-30","ACTUAL",{"date":331,"type":329},"2025-02-06",{"date":333,"type":308},"2027-07",{"name":5,"class":6},25]