Mivelsiran: Mivelsiran will be administered intrathecally
Placebo: Placebo will be administered intrathecally
Study 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
Sex
ALL
Min age
40 Years
Max age
55 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) — Up to 24 months
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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