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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 40 Years and 55 Years
Eligible Sex
All
Accepts 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Mivelsiran (Treatment Group A) 		Participants will be administered multiple low doses of mivelsiran.
  • Drug: Mivelsiran
    Mivelsiran will be administered intrathecally
    Other names:
    • ALN-APP
Experimental
Mivelsiran (Treatment Group B) 		Participants will be administered multiple high doses of mivelsiran.
  • Drug: Mivelsiran
    Mivelsiran will be administered intrathecally
    Other names:
    • ALN-APP
Placebo Comparator
Placebo (Treatment Group C) 		Participants will be administered multiple doses of placebo.
  • Drug: Placebo
    Placebo will be administered intrathecally

Recruiting Locations

Clinical Study Site
Maitland, Florida 32751

Clinical Study Site
Naples, Florida 34105

More Details

Status
Recruiting
Sponsor
Alnylam Pharmaceuticals

Study Contact

Alnylam Clinical Trial Information Line
1-877-ALNYLAM
clinicaltrials@alnylam.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.