ARO-MAPT-SC in healthy adults and people with early Alzheimer's disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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

Inclusion Criteria (Early AD):

* 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 ≥8 weeks prior to Screening.

  1. Participants with early AD are not required to be on AD medications.
  2. Participants previously treated or currently receiving anti-amyloid therapies, including lecanemab and donanemab, are not eligible.

Inclusion Criteria (MAPT-FTD)

* Adults ≥18 years of age with Clinical Dementia Rating plus National Alzheimer's Coordinating Center Frontotemporal Lobar Degeneration-Neuropsychiatric and Motor domains Sum of Boxes Global Score of 0.5 or 1 at Screening
* Frontotemporal Lobar Degeneration (F-causative MAPT mutation, based on source verifiable medical records.
* If the participant is on medications for symptomatic management of MAPT-FTD, the doses must be stable for ≥8 weeks prior to Screening.

Inclusion Criteria (PSP-RS)

* Adults ≥41 to ≤86 years of age with clinical diagnosis of probable PSP-RS phenotype as defined by the 2017 Movement Disorder Society criteria (Höglinger 2017) with symptom onset ≥40 to ≤85 years of age
* Presence of PSP symptoms ≤3 years at Screening (occurring newly and subsequently progressing during the clinical course in the absence of another identifiable cause) as determined by the Investigator
* Mini-Mental Status Examination (MMSE) score of 20 to 30 (inclusive) at Screening
* Able to walk at least 10 steps with minimal assistance (eg, one arm for safety, but not postural support)
* Reside at home or in the community (assisted living is acceptable); outside of a skilled nursing facility or dementia care facility at the time of screening, with no planned admissions to such a facility at time of Screening.
* If the participant is on medications for symptomatic management of PSP-RS, the doses must be stable for ≥8 weeks prior to Screening.

Inclusion Criteria (AD and primary tauopathies)

* Have a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to trial 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 trial in order to provide accurate and reliable information relevant to trial 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 it's running

• Research Site 3
  East York, Ontario, Canada Recruiting
• Research Site 2
  Toronto, Ontario, Canada Recruiting
• Research Site 4
  Montreal, Quebec, Canada Recruiting
• Research Site 1
  Grafton, Auckland, New Zealand Recruiting

Study contacts

• Study coordinator: Medical Monitor
• Email: AROMAPT-SC-1001@arrowheadpharma.com
• Phone: 626-304-3400

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.