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RecruitingNCT07221344Updated Nov 10, 2026

Trial of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease or Primary Tauopathies

A Phase 1/2 interventional study of ARO-MAPT-SC and Placebo in Alzheimer Disease, Tauopathies and Frontotemporal Dementia, sponsored by Arrowhead Pharmaceuticals. Recruiting at 4 sites in 2 countries. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-11-10.

Sponsored by Arrowhead Pharmaceuticals · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Nov 2025; still recruiting 11 months later.
Updated Nov 10, 2026Enrollment updatedEligibility revised+2 moreGo to Updates ↓
Phase
Phase 1/2
Study type
Interventional
Enrollment
160
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

Trial to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) 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, as well as primary tauopathies, including microtubule associated protein tau -related frontotemporal dementia (MAPT-FTD) and progressive supranuclear palsy- Richardson syndrome (PSP-RS).

02

Conditions studied

  • Alzheimer Disease
  • Tauopathies
  • Frontotemporal Dementia
  • Progressive Supranuclear Palsy

Keywords

  • microtubule associated protein tau-related frontotemporal dementia
  • MAPT-FTD
  • progressive supranuclear palsy- Richardson syndrome
  • PSP-RS
03

In context

Alzheimer Disease

3,965 studies on the registry are indexed under Alzheimer Disease; 926 are open to participants now.

This study's planned enrollment of 160 is above the median of 70 across 3,019 interventional studies indexed under Alzheimer Disease.

Browse Alzheimer Disease studies →

Lead sponsor

Arrowhead Pharmaceuticals is the lead sponsor of 50 studies on the registry; 9 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 12 (50%) have results posted.

Browse Arrowhead Pharmaceuticals studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Nov 10, 2026. See what changed

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
Yes

Eligibility criteria

Inclusion Criteria (All Participants):

  • 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 prior to the performance of any trial specific procedures
  • Participants of must agree to use highly effective 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 () 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
  • (HIV) infection (seropositive at Screening)
  • Seropositive for (HBV) or (HCV) at Screening
  • Intellectual disability or significant behavioral neuropsychiatric manifestation
  • cardiac, liver, or renal disease
  • Any to lumbar puncture
  • Known allergy or possible allergy to either ARO-MAPT-SC or to its

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

Terms in these criteria, in plain language(11)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

BMI (body mass index)

Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.

What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

MMSE and MoCA

Short tests of memory and thinking, each scored out of 30. Lower scores suggest more difficulty; criteria give a range, either to include people with memory problems or to leave them out.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
160 participants (estimated)

Study arms

  • Experimental
    ARO-MAPT-SC

    ARO-MAPT-SC injection

    Drug: ARO-MAPT-SC

  • Placebo comparator
    Placebo

    Sterile normal saline (0.9%)

    Drug: Placebo

Interventions

  • DrugARO-MAPT-SC

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

  • DrugPlacebo

    • calculated volume to match active treatment by SC administration

06

What researchers measure

Primary outcomes

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

    Time frame: Through End of Trial (EOT; up to a maximum of Day 375)

Secondary outcomes

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

    Time frame: Through a maximum of 192 hours postdose

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

    Time frame: Through a maximum of 192 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 a maximum of 192 hours postdose

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

    Time frame: Through a maximum of 192 hours post-dose

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

    Time frame: Through a maximum of 192 hours postdose

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

    Time frame: Through a maximum of 192 hours postdose

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

    Time frame: Through a maximum of 192 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 EOT (through to maximum of Day 375)

  14. Change from Baseline in Glucose in CSF Over Time

    Time frame: Baseline through EOT (through to maximum of Day 375)

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

    Time frame: Baseline through EOT (through to maximum of Day 375)

07

Study locations

4 of 4 sites recruiting
  • Research Site 3
    East York, Ontario M4G 3E8, Canada
    Recruiting
  • Research Site 2
    Toronto, Ontario M3B2S7, Canada
    Recruiting
  • Research Site 4
    Montreal, Quebec H3G 1H9, Canada
    Recruiting
  • Research Site 1
    Grafton, Auckland 1010, New Zealand
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

1 registry update since Sep 25, 2026
Enrollment
112→160
Nov 10, 2026
Eligibility
Maximum age 80 Years→none; 6 criteria edited, 12 added
What changed in the criteria
  • EditedAble and willing to provide written informed consent prior to the performance of any studytrial specific procedures
  • EditedParticipants of childbearing potential must agree to use highly effective contraception in addition to a condom during the studytrial and for at least 90 days following the end of the studytrial or last dose of studytrial drug, whichever is later; participants must not donate sperm or eggs during the studytrial and for at least 90 days following the end of the studytrial or last dose of studytrial drug whichever is later
  • EditedInclusion Criteria (Alzheimer's DiseaseEarly AD):
  • EditedAdults aged 50 to ≤80 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis
  • EditedIf participant is on non-disease-modifying AD medications, the doses must be stable for ≥8 weeks prior to Screening.
  • AddedInclusion Criteria (MAPT-FTD)
  • AddedAdults ≥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
  • AddedFrontotemporal Lobar Degeneration (F-causative MAPT mutation, based on source verifiable medical records.
  • AddedIf the participant is on medications for symptomatic management of MAPT-FTD, the doses must be stable for ≥8 weeks prior to Screening.
  • AddedInclusion Criteria (PSP-RS)
  • AddedAdults ≥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
  • AddedPresence 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
  • AddedMini-Mental Status Examination (MMSE) score of 20 to 30 (inclusive) at Screening
  • AddedAble to walk at least 10 steps with minimal assistance (eg, one arm for safety, but not postural support)
  • AddedReside 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.
  • AddedIf the participant is on medications for symptomatic management of PSP-RS, the doses must be stable for ≥8 weeks prior to Screening.
  • AddedInclusion Criteria (AD and primary tauopathies)
  • EditedHave a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to studytrial 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 studytrial in order to provide accurate and reliable information relevant to studytrial outcome measures
Nov 10, 2026
Primary outcomes
1 edited
What changed in the outcomes
  • EditedNumber of Participants with Treatment-Emergentemergent Adverse Events (TEAEs) Over TimeTime frame: Through End of Study (EOS; Day 315) → Through End of Trial (EOT; up to a maximum of Day 375)
Nov 10, 2026
Secondary outcomes
10 time frame changed
What changed in the outcomes
  • Time framePK of ARO-MAPT-SC: Maximum Observed Plasma Concentration (Cmax)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time framePK of ARO-MAPT-SC: Time to Maximum Plasma Concentration (Tmax)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time framePK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to the Last Quantifiable Plasma Concentration (AUC0-t)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time framePK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero Extrapolated to Infinity (AUC0-inf)Through 48 hours post-dose → Through a maximum of 192 hours post-dose
  • Time framePK of ARO-MAPT-SC: Apparent Terminal Elimination Half-life (t1/2)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time framePK of ARO-MAPT-SC: Apparent Systemic Clearance (CL/F)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time framePK of ARO-MAPT-SC: Apparent Terminal-phase Volume of Distribution (Vz/F)Through 48 hours postdose → Through a maximum of 192 hours postdose
  • Time frameChange from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
  • Time frameChange from Baseline in Glucose in CSF Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
  • Time frameChange from Baseline in Cell Count in CSF Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
Nov 10, 2026
Title
StudyTrial of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease or Primary Tauopathies
Nov 10, 2026
Show all 1 update
  1. Nov 10, 2026
    Enrollment 112→160
    Eligibility Maximum age 80 Years→none; 6 criteria edited, 12 added
    What changed in the criteria
    • EditedAble and willing to provide written informed consent prior to the performance of any studytrial specific procedures
    • EditedParticipants of childbearing potential must agree to use highly effective contraception in addition to a condom during the studytrial and for at least 90 days following the end of the studytrial or last dose of studytrial drug, whichever is later; participants must not donate sperm or eggs during the studytrial and for at least 90 days following the end of the studytrial or last dose of studytrial drug whichever is later
    • EditedInclusion Criteria (Alzheimer's DiseaseEarly AD):
    • EditedAdults aged 50 to ≤80 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis
    • EditedIf participant is on non-disease-modifying AD medications, the doses must be stable for ≥8 weeks prior to Screening.
    • AddedInclusion Criteria (MAPT-FTD)
    • AddedAdults ≥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
    • AddedFrontotemporal Lobar Degeneration (F-causative MAPT mutation, based on source verifiable medical records.
    • AddedIf the participant is on medications for symptomatic management of MAPT-FTD, the doses must be stable for ≥8 weeks prior to Screening.
    • AddedInclusion Criteria (PSP-RS)
    • AddedAdults ≥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
    • AddedPresence 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
    • AddedMini-Mental Status Examination (MMSE) score of 20 to 30 (inclusive) at Screening
    • AddedAble to walk at least 10 steps with minimal assistance (eg, one arm for safety, but not postural support)
    • AddedReside 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.
    • AddedIf the participant is on medications for symptomatic management of PSP-RS, the doses must be stable for ≥8 weeks prior to Screening.
    • AddedInclusion Criteria (AD and primary tauopathies)
    • EditedHave a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to studytrial 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 studytrial in order to provide accurate and reliable information relevant to studytrial outcome measures
    Primary outcomes 1 edited
    What changed in the outcomes
    • EditedNumber of Participants with Treatment-Emergentemergent Adverse Events (TEAEs) Over TimeTime frame: Through End of Study (EOS; Day 315) → Through End of Trial (EOT; up to a maximum of Day 375)
    Secondary outcomes 10 time frame changed
    What changed in the outcomes
    • Time framePK of ARO-MAPT-SC: Maximum Observed Plasma Concentration (Cmax)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time framePK of ARO-MAPT-SC: Time to Maximum Plasma Concentration (Tmax)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time framePK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero to the Last Quantifiable Plasma Concentration (AUC0-t)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time framePK of ARO-MAPT-SC: AUC Versus Time Curve From Time Zero Extrapolated to Infinity (AUC0-inf)Through 48 hours post-dose → Through a maximum of 192 hours post-dose
    • Time framePK of ARO-MAPT-SC: Apparent Terminal Elimination Half-life (t1/2)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time framePK of ARO-MAPT-SC: Apparent Systemic Clearance (CL/F)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time framePK of ARO-MAPT-SC: Apparent Terminal-phase Volume of Distribution (Vz/F)Through 48 hours postdose → Through a maximum of 192 hours postdose
    • Time frameChange from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
    • Time frameChange from Baseline in Glucose in CSF Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
    • Time frameChange from Baseline in Cell Count in CSF Over TimeBaseline through EOS (Day 315) → Baseline through EOT (through to maximum of Day 375)
    Title StudyTrial of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease or Primary Tauopathies
    + 5 other changes: index terms, verification date, description, conditions and site details

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07221344
Lead sponsor
Arrowhead Pharmaceuticals
Responsible party
Sponsor
First posted
Oct 27, 2025
Start date
Nov 18, 2025
Primary completion
Jun 2027 (estimated)
Completion
Jun 2027 (estimated)
Last update
Nov 10, 2026

Study contacts

Medical Monitor
Contact
AROMAPT-SC-1001@arrowheadpharma.com
626-304-3400

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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