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NCT Number: NCT07221344

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

Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia.

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Key information

Conditions

Alzheimer Disease Alzheimer Disease, Early Onset Brain Diseases Central Nervous System Diseases Dementia Mental Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Tauopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site 3, East York, Ontario, Canada

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ARO-MAPT-SC

Drug
  • single or multiple doses of ARO-MAPT-SC by subcutaneous (SC) injection

Placebo

Drug
  • calculated volume to match active treatment by SC administration

Primary outcomes

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

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

Secondary outcomes

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

    Time frame: Through 48 hours postdose

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

    Time frame: Through 48 hours postdose

  3. 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

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

    Time frame: Through 48 hours postdose

  5. 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

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

    Time frame: Through 48 hours post-dose

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

    Time frame: Through 48 hours postdose

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

    Time frame: Through 48 hours postdose

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

    Time frame: Through 48 hours postdose

  10. 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

  11. 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

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

    Time frame: Through 24 hours postdose

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

    Time frame: Baseline through EOS, Day 270

  14. Change from Baseline in Glucose in CSF Over Time

    Time frame: Baseline through EOS, Day 270

  15. Change from Baseline in Cell Count in CSF Over Time

    Time frame: Baseline through EOS, Day 270

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Monitor

CONTACT

[email protected]

626-304-3400

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Oct 27, 2025
Registry last updated
Jun 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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