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

ConditionDown Syndrome-Associated Alzheimer's Disease (DS-AD)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age40-55
SponsorAlnylam Pharmaceuticals

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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/1.73m² at Screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • Mivelsiran

    Drug

    Mivelsiran will be administered intrathecally

  • Placebo

    Drug

    Placebo will be administered intrathecally

Treatment groups

58 Participants
are divided into 3 treatment groups
Group A: Mivelsiran (Treatment Group A)Experimental treatment 1 intervention
Group B: Mivelsiran (Treatment Group B)Experimental treatment 1 intervention
Group C: Placebo (Treatment Group C)Placebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)

Time frame
Up to 24 months

Secondary outcomes

1

Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF)

Time frame
Up to 24 months
2

Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF

Time frame
Up to 24 months
3

Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma

Time frame
Up to 24 months
4

Double-Blind Period: Change from baseline on a cognitive scale

Time frame
Up to 24 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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