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

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.

Interventions

• Mivelsiran · DRUG

  Mivelsiran will be administered intrathecally

• Placebo · DRUG

  Placebo will be administered intrathecally

Published eligibility criteria

Verbatim from the registry. Only the site can decide how these apply to a specific patient.

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) \<45 mL/min/1.73m\^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

Note: other protocol defined inclusion / exclusion criteria apply

Sites (2)

• Clinical Study Site, Maitland, Florida · RECRUITING
• Clinical Study Site, Naples, Florida · RECRUITING