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Mivelsiran

Phase 2

Down Syndrome-Associated Alzheimer's Disease (DS-AD) | Small molecule | Neurology |Alnylam Pharmaceuticals, Inc.|Last Updated: Jun 9, 2026

Success Probability
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment58
FDA Designations
No designations recorded
Clinical trial landscape

Mivelsiran · 1 trial · 1 indication

Phase 2 1
NCT07636811A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)Down Syndrome-Associated Alzheimer's Disease (DS-AD)
NOT YET_RECRUITING58 Analytics
PHASE2NOT YET_RECRUITING
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Down Syndrome-Associated Alzheimer's Disease (DS-AD)
NCT0763681158
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Study Endpoints
Primary Endpoints
Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)
Up to 24 months
Secondary Endpoints
Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF)
Up to 24 months
Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF
Up to 24 months
Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma
Up to 24 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Mivelsiran (Treatment Group A)EXPERIMENTALParticipants will be administered multiple low doses of mivelsiran.
Mivelsiran (Treatment Group B)EXPERIMENTALParticipants will be administered multiple high doses of mivelsiran.
Placebo (Treatment Group C)PLACEBO_COMPARATORParticipants will be administered multiple doses of placebo.
Interventions
NameTypeDescription
MivelsiranDRUGMivelsiran will be administered intrathecally
PlaceboDRUGPlacebo will be administered intrathecally
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Eligibility Criteria
Age Range40 Years to 55 Years
SexALL
Healthy VolunteersNo
Study Sites2

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: * ...

Countries:United States
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Recent Changes (Last 90 Days)
LOWJun 9, 2026NCT07636811NEW_TRIAL: changed
LOWJun 9, 2026NCT07636811NEW_TRIAL: changed
LOWJun 9, 2026NCT07636811NEW_TRIAL: changed
LOWJun 9, 2026NCT07636811NEW_TRIAL: changed