The Progressive Supranuclear Palsy Clinical Trial Platform

NCT07173803 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ATRI-015R01AG085029
• Study Type: interventional
• Target: 440
• Locations: 0 countries
• Sites: N/A

Brief Summary

The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of PSP.

Conditions

Progressive Supranuclear Palsy(PSP)

Keywords

PSP; Placebo-Controlled; Double-Blind; Master Protocol

Outcome Measures

Primary Outcomes (1)

• Disease progression

  Change in disease severity as measured by the 15-item modified Progressive Supranuclear Palsy Rating Scale (mPSPRS-15) in which the minimum score is 0 and the maximum score is 52, with higher scores indicating a worse outcome.

  52 weeks

Secondary Outcomes (9)

• Disease progression

  52 weeks

• Disease progression

  52 weeks

• Experiences of daily living

  52 weeks

• Activities of daily living

  52 weeks

• Disease severity

  52 weeks

Study Arms (2)

Regimen A: AADvac1

EXPERIMENTAL

Participants are randomized to receive either active AADvac1 or matching placebo.

Biological: AADvac1

Regimen B: LM11A-31

EXPERIMENTAL

Participants are randomized to receive either active LM11A-31 or matching placebo.

Drug: LM11A-31

Interventions

AADvac1 BIOLOGICAL

Active immunotherapy

Regimen A: AADvac1

LM11A-31 DRUG

Small molecule

Regimen B: LM11A-31

Eligibility Criteria

• Age: 41 Years - 86 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of possible or probable PSP Richardson's Syndrome as defined by the 2017 Movement Disorder Society (MDS) criteria.
• Presence of PSP symptoms for ≤5 years at screening (based on the best judgment of the site PI).
• Mini-Mental State Examination (MMSE) score at screening of ≥25.
• Able to walk at least 10 steps with minimal assistance (e.g., one arm for safety, but not postural support).
• Stable doses of permitted medications as described per protocol for 30 days prior to screening.
• Resides at home or in the community (assisted living is acceptable).
• As assessed by the site PI, participant is likely to be able to comply with the protocol for the duration of the study, and has adequate vision, hearing (hearing aid permitted), and literacy (English or Spanish) sufficient for compliance with the required testing procedures.

You may not qualify if:

• Females who are breastfeeding or pregnant (as documented by a urine pregnancy test) during screening, or plan to become pregnant during the study.
• Females of childbearing potential who did not use a highly effective method of contraception within 28 days of screening and/or are not willing to use a highly effective method of contraception for the duration of their participation in the study.
• Lacks good venous access such that multiple blood draws would be precluded.
• Weighs less than 40kg, or more than 136kg at screening.
• Blood transfusion within 4 weeks of screening.
• Contraindications to MRI studies, including metal (ferromagnetic) implants, a cardiac pacemaker that is not compatible with MRI, and/or severe claustrophobia.
• Screening MRI scan showing structural evidence of alternative pathology not consistent with PSP that could explain a substantial portion of the participant's symptoms as indicated by the central MRI read.
• Any unstable and/or clinically significant medical condition likely to hamper the evaluation of safety and/or efficacy of study drug (e.g., clinically significant reduction in serum B12 or folate levels, clinically significant abnormalities of thyroid function, stroke, or other cerebrovascular or cardiovascular conditions), as per the site PI's judgment.
• History of severe allergic reaction (e.g., anaphylaxis) including but not limited to: severe allergic reaction to previous vaccines, foods, and/or medications.
• Hospitalization within 30 days prior to screening or baseline.
• Infections or major surgical procedures within 3 months prior to screening, judged to be clinically significant by the site PI.
• Myocardial infarction within 1 year prior to baseline, unstable angina pectoris, symptomatic congestive heart failure.
• History of cancer within the past 5 years other than treated skin squamous cell carcinoma, basal cell carcinoma, and melanoma in-situ, localized prostate cancer not requiring treatment, or prostate or breast cancer, which have been fully removed and are considered cured.
• History or presence of immunological or inflammatory conditions, including neurological disorders, meningitis or meningoencephalitis.
• History or presence of epilepsy requiring ongoing use of antiepileptic medications. Antiepileptic medications are permitted for pain or psychiatric use per the protocol.

Sponsors & Collaborators

• Adam Boxer: lead
• National Institute on Aging (NIA): collaborator
• Alzheimer's Therapeutic Research Institute: collaborator
• University of Southern California: collaborator
• Massachusetts General Hospital: collaborator
• University of California, San Diego: collaborator
• Alzheimer's Clinical Trials Consortium: collaborator

MeSH Terms

Conditions

Supranuclear Palsy, Progressive

Interventions

AADvac1; LM11A-31

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Adam Boxer, MD, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

• Irene Litvan, MD

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

• Julio Rojas-Martinez, MD, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

• Anne-Marie Wills, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

ATRI Recruitment and Retention (RER) Unit

CONTACT

213-821-0569; psp-participate@usc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Endowed Professor in Memory and Aging

Study Record Dates

• First Submitted: September 3, 2025
• First Posted: September 15, 2025
• Study Start (Estimated): June 30, 2026
• Primary Completion (Estimated): September 28, 2030
• Study Completion (Estimated): September 28, 2030
• Last Updated: April 27, 2026

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL