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

Arrowhead Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Alzheimer Disease; Alzheimer Disease, Early Onset

Treatments: Drug: Placebo; Drug: ARO-MAPT-SC

Study type: Interventional

Funder types: Industry

Identifiers

NCT07221344

AROMAPT-SC-1001

Details and patient eligibility

About

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.

Enrollment: 112 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria (All Participants):

• Body mass index between 18.0 and 35.0 kg/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 75 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis
• On stable doses of AD-related medications for at least 8 weeks prior to Screening Visit and throughout the Screening period until Day 1

Exclusion Criteria (All Participants):

• Uncontrolled hypertension
• 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

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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112 participants in 2 patient groups, including a placebo group

ARO-MAPT-SC

Experimental group

Description:

ARO-MAPT-SC injection

Treatment:

Drug: ARO-MAPT-SC

Placebo

Placebo Comparator group

Description:

Sterile normal saline (0.9%)

Treatment:

Drug: Placebo