Alzheimer DiseasePhase 1/2NCT07221344
Study of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease
A Phase 1/2 study testing ARO-MAPT-SC in people with Alzheimer Disease and Alzheimer Disease, Early Onset, run by Arrowhead Pharmaceuticals at 4 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Alzheimer Disease, Alzheimer Disease, Early Onset
- What is being tested?
- ARO-MAPT-SC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 112 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 18 Nov 2025to Jun 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
- 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 non-disease-modifying AD medications, the doses must be stable for ≥weeks prior to Screening.
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- Participants with early AD are not required to be on AD medications.
- Participants previously treated or currently receiving anti-amyloid therapies, including lecanemab and donanemab, are not eligible.
- 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
Who cannot join
- 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.
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
Canada3 sites, 3 recruiting
- Recruiting
Research Site 3
East York, Ontario, M4G 3E8
- Recruiting
Research Site 4
Montreal, Quebec, H3G 1H9
- Recruiting
Research Site 2
Toronto, Ontario, M3B2S7
New Zealand1 site, 1 recruiting
- Recruiting
Research Site 1
Grafton, Auckland, 1010
About this study
Official 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
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.
- Conditions
- Alzheimer Disease, Alzheimer Disease, Early Onset
- Being tested
- ARO-MAPT-SC, Placebo
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-MAPT-SCDrug
- single or multiple doses of ARO-MAPT-SC by subcutaneous (SC) injection
PlaceboDrug
- calculated volume to match active treatment by SC administration
| Study group | Role | What they receive |
|---|---|---|
| ARO-MAPT-SC | Experimental | ARO-MAPT-SC injection Receives: Drug: ARO-MAPT-SC |
| Placebo | Placebo comparator | Sterile normal saline (0.9%) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Over Time
Measured: Through End of Study (EOS; Day 315)
Secondary outcomes (15)
PK of ARO-MAPT-SC: Maximum Observed Plasma Concentration (Cmax)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: Time to Maximum Plasma Concentration (Tmax)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: Area Under the Plasma Concentration (AUC) Versus Time Curve From Time Zero to 24 Hours (AUC0-24)
Measured: Through 24 hours postdose
PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to 48 Hours (AUC0-48)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to the Last Quantifiable Plasma Concentration (AUC0-t)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero Extrapolated to Infinity (AUC0-inf)
Measured: Through 48 hours post-dose
PK of ARO-MAPT-SC: Apparent Terminal Elimination Half-life (t1/2)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: Apparent Systemic Clearance (CL/F)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: Apparent Terminal-phase Volume of Distribution (Vz/F)
Measured: Through 48 hours postdose
PK of ARO-MAPT-SC: Amount Excreted (Ae) of Unchanged Drug in Urine From Time Zero to 24 Hours Postdose
Measured: Through 24 hours postdose
PK of ARO-MAPT-SC: Percentage of Administered Drug Recovered (Fe) in Urine From Time Zero to 24 Hours Postdose
Measured: Through 24 hours postdose
PK of ARO-MAPT-SC: Renal Clearance (CLR)
Measured: Through 24 hours postdose
Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time
Measured: Baseline through EOS (Day 315)
Change from Baseline in Glucose in CSF Over Time
Measured: Baseline through EOS (Day 315)
Change from Baseline in Cell Count in CSF Over Time
Measured: Baseline through EOS (Day 315)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2025
- Study start18 Nov 2025
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer DiseaseStudies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 112 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07221344
- Sponsor's protocol ID
- AROMAPT-SC-1001
- Status last verified
- Sep 2026
- First submitted
- 23 Oct 2025
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No