RecruitingPhase 1 / PHASE2

Study of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease

A Phase 1/2a Placebo-Controlled Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-MAPT-SC in Healthy Subjects and Subjects With Early Alzheimer's Disease

Lead sponsor

Arrowhead Pharmaceuticals

Asset

ARO-MAPT-SC

Listed sites

Recruiting sites

Enrollment

112

estimated

Study population

Alzheimer’s disease

Key I/E criteria

•Alzheimer's disease•Study partner/caregiver required

Primary endpoint

•Treatment-Emergent Adverse Events (TEAEs) Over Time

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

Org study IDAROMAPT-SC-1001

NCT IDNCT07221344

Timeline

Milestones

Study first posted2025-10-27actual

Study start2025-11-18actual

Last update posted2026-06-23actual

Primary completion2027-06estimated (month precision)

Study completion2027-06estimated (month precision)

Assets

Drug assets

ARO-MAPT-SC

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age18 Years

Maximum age80 Years

SexAll

Healthy volunteersAccepted

Inclusion criteria

(All Participants):

•Body mass index between 18.0 and 35.0 kilograms (kg)/square meter (m^2) at Screening

•Not pregnant or breast-feeding

•Able and willing to provide written informed consent prior to the performance of any study specific procedures

•Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later; participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later

Inclusion Criteria (Alzheimer's Disease):

•Adults aged 50 to 80 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis

•If participant is on AD medications, the doses must be stable for ≥weeks prior to Screening.

a. Participants with early AD are not required to be on AD medications.

•Have a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to study visits involving informant-based assessments, to be available to site staff by telephone as needed, and in the opinion of the Investigator, be sufficiently familiar with the participant throughout the study in order to provide accurate and reliable information relevant to study outcome measures

Exclusion criteria

(All Participants):

•Blood pressure outside of specified range in the protocol

•Human immunodeficiency virus (HIV) infection (seropositive at Screening)

•Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening

•Intellectual disability or significant behavioral neuropsychiatric manifestation

•Clinically significant cardiac, liver, or renal disease

•Any contraindications to lumbar puncture

•Known allergy or possible allergy to either ARO-MAPT-SC or to its excipients

Note: Additional inclusion/exclusion criteria may apply per protocol.

Endpoints (16)

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

Other (unclassified)

Fluid / digital biomarkers

3 endpoints

Secondary/protocol endpoint

Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time

Time frame:Baseline through EOS, Day 270

change from baseline, improvement

Secondary/protocol endpoint

Change from Baseline in Glucose in CSF Over Time

Time frame:Baseline through EOS, Day 270

change from baseline, improvement

Secondary/protocol endpoint

Change from Baseline in Cell Count in CSF Over Time

Time frame:Baseline through EOS, Day 270

change from baseline, improvement

Safety / tolerability / PK

8 endpoints

Primary/protocol endpoint

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Over Time

Time frame:Through End of Study (EOS), Day 270

event count, event

Secondary/protocol endpoint

PK of ARO-MAPT-SC: Maximum Observed Plasma Concentration (Cmax)

Time frame:Through 48 hours postdose

concentration, descriptive

Secondary/protocol endpoint

PK of ARO-MAPT-SC: Time to Maximum Plasma Concentration (Tmax)

Time frame:Through 48 hours postdose

time to event, event

Secondary/protocol endpoint

PK of ARO-MAPT-SC: Area Under the Plasma Concentration (AUC) Versus Time Curve From Time Zero to 24 Hours (AUC0-24)

Time frame:Through 24 hours postdose

concentration, descriptive

Secondary/protocol endpoint

PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to 48 Hours (AUC0-48)

Time frame:Through 48 hours postdose

concentration, descriptive

Secondary/protocol endpoint

PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to the Last Quantifiable Plasma Concentration (AUC0-t)

Time frame:Through 48 hours postdose

concentration, descriptive

Secondary/protocol endpoint

PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero Extrapolated to Infinity (AUC0-inf)

Time frame:Through 48 hours post-dose

concentration, descriptive

Secondary/protocol endpoint

PK of ARO-MAPT-SC: Apparent Terminal Elimination Half-life (t1/2)

Time frame:Through 48 hours postdose

concentration, descriptive

Other (unclassified)

5 endpoints

Secondary/protocol endpoint/low confidence

PK of ARO-MAPT-SC: Apparent Systemic Clearance (CL/F)

Time frame:Through 48 hours postdose

descriptive

Secondary/protocol endpoint/low confidence

PK of ARO-MAPT-SC: Apparent Terminal-phase Volume of Distribution (Vz/F)

Time frame:Through 48 hours postdose

descriptive

Secondary/protocol endpoint/low confidence

PK of ARO-MAPT-SC: Amount Excreted (Ae) of Unchanged Drug in Urine From Time Zero to 24 Hours Postdose

Time frame:Through 24 hours postdose

descriptive

Secondary/protocol endpoint/low confidence

PK of ARO-MAPT-SC: Percentage of Administered Drug Recovered (Fe) in Urine From Time Zero to 24 Hours Postdose

Time frame:Through 24 hours postdose

threshold achievement, improvement

Secondary/protocol endpoint/low confidence

PK of ARO-MAPT-SC: Renal Clearance (CLR)

Time frame:Through 24 hours postdose

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.