[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100492574":3},{"organization":4,"outcomesModule":7,"designInfo":32,"detailedDescription":11,"studyPopulation":11,"armGroups":38,"interventions":60,"overallOfficials":66,"centralContacts":70,"locations":75,"responsibleParty":88,"collaborators":90,"id":93,"slug":11,"hasResults":94,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":11,"eligibilityCriteria":98,"healthyVolunteers":94,"sex":99,"minAge":100,"maxAge":11,"enrollmentInfo":101,"targetDuration":11,"studyType":104,"phases":105,"briefSummary":107,"conditions":108,"keywords":11,"overallStatus":78,"whyStopped":11,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":120},{"fullName":5,"class":6},"Pharmacosmos A\u002FS","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":16,"otherOutcomes":11},[9,13],{"measure":10,"description":11,"timeFrame":12},"To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia",null,"24 weeks",{"measure":14,"description":11,"timeFrame":15},"To assess the safety and tolerability of ascending doses of SP-420 after 12 weeks treatment of subjects with transfusion-dependent low-risk myelodysplastic syndrome","12 weeks",[17,20,24,28],{"measure":18,"description":19,"timeFrame":12},"To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia","Change in liver iron concentration (LIC) measured by R2-magnetic resonance imaging (MRI) from baseline to week 24",{"measure":21,"description":22,"timeFrame":23},"To assess the efficacy of SP-420 in clearing iron from the liver after 12 and 48 weeks treatment of subjects with transfusion-dependent β-thalassemia","Change in LIC measured by R2-MRI from baseline to week 12 and week 48","12 and 48 weeks",{"measure":25,"description":26,"timeFrame":27},"To assess the efficacy of SP-420 on serum (s-) ferritin","Change in s-ferritin from baseline to weeks 1, 2, 3, 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48","up to 48 weeks",{"measure":29,"description":30,"timeFrame":31},"To assess the safety and tolerability of ascending doses of SP-420","Type and incidence of adverse events (AEs)","48 weeks",{"allocation":33,"interventionModel":34,"interventionModelDescription":11,"primaryPurpose":35,"observationalModel":11,"timePerspective":11,"maskingInfo":36},"RANDOMIZED","SEQUENTIAL","TREATMENT",{"masking":37,"maskingDescription":11,"whoMasked":11},"NONE",[39,45,48,51,54,57],{"label":40,"type":41,"description":42,"interventionNames":43},"Cohort 1a-1b","EXPERIMENTAL","SP-420 for 48 weeks",[44],"Drug: SP-420",{"label":46,"type":41,"description":42,"interventionNames":47},"Cohort 2a-2b",[44],{"label":49,"type":41,"description":42,"interventionNames":50},"Cohort 3a-3b",[44],{"label":52,"type":41,"description":42,"interventionNames":53},"1c-1d",[44],{"label":55,"type":41,"description":42,"interventionNames":56},"2c-2d",[44],{"label":58,"type":41,"description":42,"interventionNames":59},"3c-3d",[44],[61],{"type":62,"name":63,"description":64,"armGroupLabels":65,"otherNames":11},"DRUG","SP-420","Capsules for oral intake",[52,55,58,40,46,49],[67],{"name":68,"affiliation":5,"role":69},"Pharmacosmos Clinical and non-clinical Department","STUDY_DIRECTOR",[71],{"name":68,"role":72,"phone":73,"phoneExt":11,"email":74},"CONTACT","+45 5948 5959","info@pharmacosmos.com",[76],{"facility":77,"status":78,"city":79,"state":11,"zip":11,"country":80,"countryCode":81,"cosmosGeoPoint":82,"geoPoint":87,"contacts":11},"Pharmacosmos Investigational Site","RECRUITING","Copenhagen","Denmark","DK",{"type":83,"coordinates":84},"Point",[85,86],12.56553,55.67594,{"lat":86,"lon":85},{"type":89,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[91],{"name":92,"class":6},"ICON plc","100492574",false,"NCT05693909","A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes","An Open-label, Dose-escalation, Dose-finding, and Proof-of-concept Trial of SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes","Thalassemia cohorts:\n\nInclusion criteria:\n\n* Women and men aged 18 years or older\n* Transfusion-dependent β-thalassemia including HbE\u002Fβ-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and\u002For multiplication of α-globin is allowed)\n* On a stable dose of iron chelation for at least 4 weeks prior to screening\n* Weight ≥ 35kg at screening\n* Transfusion iron overload\n* Treated and followed for at least the past 6 months in a specialized centre\n\nExclusion criteria:\n\n* β-thalassemia with the structural Hb variants HbS and HbC\n* Current MDS\n* Current biliary disorder\n* Historic or ongoing clinically significant kidney disease\n* Unable to undergo trial assessments including MRI e.g. due to claustrophobia in MRI scanner\n* Pregnant or nursing women\n* Men who do not agree to practice effective barrier contraception during the entire period\n\nMyelodysplastic Syndromes Cohorts:\n\nInclusion criteria:\n\n* Women and men aged 18 years or older\n* Very low, low, or intermediate risk Myelodysplastic Syndrome according to IPSS-R\n* Weight ≥ 35kg at screening\n* Transfusion iron overload\n* Treated and followed for at least the past 6 months at medical facilities experienced with MDS\n\nExclusion criteria:\n\n* Therapy-related MDS or MDS with a known bone marrow fibrosis\n* Diagnosis of decompensated liver cirrhosis\n* Clinically significant kidney disease, either historic or ongoing\n* Uncontrolled ischemic heart disease or uncontrolled arrythmia\n* Uncontrolled hypertension\n* Uncontrolled dyslipidaemia\n* Uncontrolled Diabetes\n* Major surgery within 8 weeks prior to screening\n* Pregnant or nursing women\n* Men who do not agree to practice effective barrier contraception during the entire period","ALL","18 Years",{"count":102,"type":103},90,"ESTIMATED","INTERVENTIONAL",[106],"PHASE2","The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.\n\nThe main questions it aims to answer are:\n\n* How efficient is SP-420 in cleaning iron from the liver?\n* How is the safety and tolerability of ascending doses of SP-420?\n\nParticipants will:\n\n* Take medication three times weekly\n* Attend up to 20 site visits\n* Undergo MRI scans",[109,110],"Beta Thalassemia Major Anemia","Myelodysplastic Syndrome","2025-09-19",{"date":113,"type":114},"2025-09-24","ACTUAL",{"date":116,"type":114},"2023-09-04",{"date":118,"type":103},"2028-05",{"name":5,"class":6},1]