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RecruitingPhase 1

A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)

Asset

Mivelsiran

Listed sites

8

Recruiting sites

6

Enrollment

60

estimated

Study population

Alzheimer’s disease

Key I/E criterion

•MMSE ≥20

Primary endpoints

•Part•Part B

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Secondary ID2023-508363-79-00EU CT Number
Org study IDALN-APP-001
NCT IDNCT05231785

Timeline

Milestones

Study start2022-02-04actual
Study first posted2022-02-09actual
Last update posted2026-07-15actual
Primary completion2029-04-20estimated
Study completion2029-04-20estimated

Assets

Drug assets

Mivelsiran

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age18 Years
SexAll
Healthy volunteersNot accepted

Inclusion criteria

•Has mild cognitive impairment or mild dementia due to EOAD
•Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) >20

Exclusion criteria

•Has Non-Alzheimer's disease dementia
•Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2×upper limit of normal (ULN)
•Has estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m^2 at Screening
•Has recently received an investigational agent
•Has recent treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Endpoints (7)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Safety / tolerability / PK
4
Amyloid biomarkers
1
Fluid / digital biomarkers
1
Other (unclassified)
1

Amyloid biomarkers

1 endpoint
Secondary/protocol endpoint

Part A and Part B: Change from Baseline in Levels of Cerebrospinal Fluid (CSF) Soluble Amyloid Precursor Protein Alpha (sAPPα) and Soluble Amyloid Precursor Protein Beta (sAPPβ)

Time frame:Part A up to 12 months; Part B up to 24 months

change from baseline, improvement

Fluid / digital biomarkers

1 endpoint
Secondary/protocol endpoint

Part A and Part B: Concentration of ALN-APP and Potential Metabolites at Time 't' (Ct) in Plasma and CSF

Time frame:Part A up to 12 months; Part B up to 24 months

concentration, descriptive

Safety / tolerability / PK

4 endpoints
Primary/protocol endpoint

Part A: Frequency of Adverse Events

Time frame:Up to 12 months

event count, event

Primary/protocol endpoint

Part B: Frequency of Adverse Events

Time frame:Up to 42 months

event count, event

Secondary/protocol endpoint

Part A and Part B: Area Under the Plasma Concentration-time Curve (AUC) of ALN-APP and of Potential Metabolites

Time frame:Part A up to 12 months; Part B up to 24 months

concentration, descriptive

Secondary/protocol endpoint

Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of ALN-APP and of Potential Metabolites

Time frame:Part A up to 12 months; Part B up to 24 months

concentration, descriptive

Other (unclassified)

1 endpoint
Secondary/protocol endpoint/low confidence

Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe)

Time frame:Up to 1 day

descriptive

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2
Snapshot dateJuly 21, 2026
Endpoint classificationDelfa ADRD endpoint taxonomy
Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.