[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07636811":3,"trial-entities:NCT07636811":111,"trial-summary:NCT07636811":116},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":22,"primary_purpose":23,"phases":24,"enrollment_info":26,"interventions":29,"primary_outcomes":44,"secondary_outcomes":48,"sex":60,"minimum_age":61,"maximum_age":62,"healthy_volunteers":63,"eligibility_criteria":64,"std_ages":79,"locations":81,"central_contacts":97,"overall_officials":105,"references":109,"see_also_links":110},"NCT07636811","ALN-APP-003","A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)","A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study, to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)","NOT_YET_RECRUITING","2031-07-23","2026-07","2026-07-15","2026-07-31","Alnylam Pharmaceuticals","INDUSTRY",true,"The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.",null,[19],"Down Syndrome-Associated Alzheimer's Disease (DS-AD)",[21],"Alzheimer's; DS-AD; Down Syndrome; ALZ","INTERVENTIONAL","TREATMENT",[25],"PHASE2",{"count":27,"type":28},58,"ESTIMATED",[30,39],{"type":31,"name":32,"description":33,"armGroupLabels":34,"otherNames":37},"DRUG","Mivelsiran","Mivelsiran will be administered intrathecally",[35,36],"Mivelsiran (Treatment Group A)","Mivelsiran (Treatment Group B)",[38],"ALN-APP",{"type":31,"name":40,"description":41,"armGroupLabels":42},"Placebo","Placebo will be administered intrathecally",[43],"Placebo (Treatment Group C)",[45],{"measure":46,"timeFrame":47},"Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)","Up to 24 months",[49,51,53,55,57],{"measure":50,"timeFrame":47},"Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF)",{"measure":52,"timeFrame":47},"Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF",{"measure":54,"timeFrame":47},"Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma",{"measure":56,"timeFrame":47},"Double-Blind Period: Change from baseline on a cognitive scale",{"measure":58,"timeFrame":59},"Double-blind Period and Open-label Extension (OLE) Period: Frequency of adverse events (AEs)","Up to 36 months","ALL","40 Years","55 Years",false,{"inclusion":65,"exclusion":70,"raw_text":78},[66,67,68,69],"Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21","Positive amyloid PET scan","Cognitively stable in the opinion of the investigator","Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening",[71,72,73,74,75,76,77],"Has severe intellectual disability (ID)","Has a history of DS regression disorder","Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2×upper limit of normal (ULN) at Screening","Has estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m\\^2 at Screening","Has recently received an investigational agent","Has had treatment with amyloid-targeting antibody","Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled","Inclusion Criteria:\n\n* Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21\n* Positive amyloid PET scan\n* Cognitively stable in the opinion of the investigator\n* Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening\n\nExclusion Criteria:\n\n* Has severe intellectual disability (ID)\n* Has a history of DS regression disorder\n* Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>2×upper limit of normal (ULN) at Screening\n* Has estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m\\^2 at Screening\n* Has recently received an investigational agent\n* Has had treatment with amyloid-targeting antibody\n* Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled\n\nNote: other protocol defined inclusion \u002F exclusion criteria apply",[80],"ADULT",[82,91],{"facility":83,"city":84,"state":85,"zip":86,"country":87,"geoPoint":88},"Clinical Study Site","Maitland","Florida","32751","United States",{"lat":89,"lon":90},28.62778,-81.36312,{"facility":83,"city":92,"state":85,"zip":93,"country":87,"geoPoint":94},"Naples","34105",{"lat":95,"lon":96},26.14234,-81.79596,[98,103],{"name":99,"role":100,"phone":101,"email":102},"Alnylam Clinical Trial Information Line","CONTACT","1-877-ALNYLAM","clinicaltrials@alnylam.com",{"name":99,"role":100,"phone":104,"email":102},"1-877-256-9526",[106],{"name":107,"affiliation":13,"role":108},"Medical Director","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":112,"biomarkers":114},[113],"Down Syndrome-Associated Alzheimer's Disease",[115],"Amyloid",{"nct_id":4,"found":63,"summary":17,"prompt_version":17}]