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Down Syndrome Associated Alzheimer's Disease (DS AD) / Mivelsiran · Phase 2 Trial
ClinicalTrials.gov · August 12, 2026
Early or partial results. Treat as a signal, not a conclusion.
This is a Phase 2 trial registration for mivelsiran, an investigational agent, in adults with early-stage Down syndrome-associated Alzheimer's disease. The study is recruiting and has not yet reported results; the primary endpoint is change in brain amyloid burden over 24 months. No evidence of efficacy or safety is available from this registry record alone.
Phase 2, Interventional, Randomized, Parallel, Quadruple masking, Treatment purpose. Down Syndrome-Associated Alzheimer's Disease (DS-AD); age from 40 Years; to 55 Years. Intervention: Mivelsiran (Treatment Group A); Mivelsiran (Treatment Group B). Compared with: Placebo (Treatment Group C) — Placebo Comparator. n = 58. 2 sites: United States.
This is a Phase 2 trial registration for mivelsiran, an investigational agent, in adults with early-stage Down syndrome-associated Alzheimer's disease. The study is recruiting and has not yet reported results; the primary endpoint is change in brain amyloid burden over 24 months. No evidence of efficacy or safety is available from this registry record alone.
This is a registry record with no results posted; no efficacy, safety, or tolerability data are reported here.
This trial is in an early phase and has not reported results. Clinicians should not yet draw conclusions about mivelsiran's efficacy, safety, or role in DS-AD management. Results, when available, will inform whether this approach merits further development.
This is a Phase 2 trial registration with no results reported; the study is actively recruiting and designed to evaluate safety, tolerability, and a surrogate biomarker endpoint in a rare population.
As stated by the source record.
Quoted from the source exactly as published.
This trial is in an early phase and has not reported results. Clinicians should not yet draw conclusions about mivelsiran's efficacy, safety, or role in DS-AD management. Results, when available, will inform whether this approach merits further development.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
What is missing. This record has no key findings. That is a gap in the analysis, not a judgement about the study.
Registry record from ClinicalTrials.gov (NCT07636811). This is a study registration, not published results. Lead sponsor: Alnylam Pharmaceuticals. Recruitment status: RECRUITING. Phase: PHASE2. Study type: INTERVENTIONAL. Enrollment: 58 participants (ESTIMATED). Conditions: Down Syndrome-Associated Alzheimer's Disease (DS-AD). Interventions: DRUG: Mivelsiran; DRUG: Placebo. Primary outcome measures: Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) , Up to 24 months. Brief summary: 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.
Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.