A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)

Sex: All

Ages: 40 Years – 55 Years

Healthy volunteers: No

Phase: PHASE2

Enrollment: 58

Sponsor: Alnylam Pharmaceuticals

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.

Eligibility Criteria

Inclusion Criteria: * 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 Exclusion Criteria: * 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) \

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View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07636811). StuddyBuddy aggregates publicly available trial information.