ARO-MAPT-SC in healthy adults and people 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
This trial will test whether ARO-MAPT-SC is safe, tolerable, and produces the expected drug levels and biomarker effects in healthy adults and people with early Alzheimer's disease.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 112 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Locations | 1 site (Grafton, Auckland) |
| Trial ID | NCT07221344 on ClinicalTrials.gov |
What this trial studies
This randomized, placebo-controlled Phase 1/2 interventional study gives subcutaneous ARO-MAPT-SC or placebo to adult healthy volunteers and participants with early Alzheimer's disease to characterize safety, tolerability, pharmacokinetics, and pharmacodynamics. The early AD cohort includes participants aged 50–80 with biomarker confirmation of AD and who are on stable AD medications. Key measures include adverse events, plasma/CSF drug concentrations, and downstream tau-related biomarkers; some cognitive and functional assessments are likely included for the AD cohort. Participants must meet BMI and reproductive health requirements and attend study visits at the listed site in Grafton, Auckland.
Who should consider this trial
Good fit: Adults aged 50 to 80 with biomarker-confirmed early AD (mild cognitive impairment due to AD or mild AD dementia), BMI 18–35 kg/m2, not pregnant or breastfeeding, and on stable AD medications are ideal candidates.
Not a fit: People with advanced Alzheimer's, without AD biomarker confirmation, outside the specified age or BMI ranges, or who are pregnant or breastfeeding are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, this approach could reduce harmful tau protein and potentially slow progression in people with early Alzheimer's disease.
How similar studies have performed: Tau-lowering strategies have shown mixed results in clinical research, and subcutaneous RNAi targeting MAPT like ARO-MAPT-SC is a relatively novel approach with limited prior human efficacy data.
Eligibility criteria
Show full inclusion / 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 80 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 * 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.
Where this trial is running
Grafton, Auckland
- Research Site 1 — Grafton, Auckland, New Zealand (Recruiting)
Study contacts
- Study coordinator: Medical Monitor
- Email: AROMAPT-SC-1001@arrowheadpharma.com
- Phone: 626-304-3400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.