Progressive Supranuclear Palsy Clinical Trial Platform

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 is a Phase 2, double-blind, placebo-controlled platform trial for progressive supranuclear palsy. It uses a single Master Protocol to evaluate multiple regimens, adding new ones over time. Participants are randomized to active drug or placebo within a regimen, and each regimen has its own specific outcome measures. The trial emphasizes multi-center participation and flexible enrollment as new regimens become available.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Clinical diagnosis of possible or probable PSP Richardson's Syndrome as defined by the 2017 Movement Disorder Society (MDS) criteria.
2. Presence of PSP symptoms for ≤5 years at screening (based on the best judgment of the site PI).
3. Mini-Mental State Examination (MMSE) score at screening of ≥25.
4. Able to walk at least 10 steps with minimal assistance (e.g., one arm for safety, but not postural support).
5. Stable doses of permitted medications as described per protocol for 30 days prior to screening.
6. Resides at home or in the community (assisted living is acceptable).
7. 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.

Exclusion Criteria:

1. Females who are breastfeeding or pregnant (as documented by a urine pregnancy test) during screening, or plan to become pregnant during the study.
2. 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.
3. Lacks good venous access such that multiple blood draws would be precluded.
4. Weighs less than 40kg, or more than 136kg at screening.
5. Blood transfusion within 4 weeks of screening.
6. Contraindications to MRI studies, including metal (ferromagnetic) implants, a cardiac pacemaker that is not compatible with MRI, and/or severe claustrophobia.
7. 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.
8. 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.
9. History of severe allergic reaction (e.g., anaphylaxis) including but not limited to: severe allergic reaction to previous vaccines, foods, and/or medications.
10. Hospitalization within 30 days prior to screening or baseline.
11. Infections or major surgical procedures within 3 months prior to screening, judged to be clinically significant by the site PI.
12. Myocardial infarction within 1 year prior to baseline, unstable angina pectoris, symptomatic congestive heart failure.
13. 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.
14. History or presence of immunological or inflammatory conditions, including neurological disorders, meningitis or meningoencephalitis.
15. History or presence of epilepsy requiring ongoing use of antiepileptic medications. Antiepileptic medications are permitted for pain or psychiatric use per the protocol.
16. DSM-5 criteria for drug or alcohol abuse or dependence currently met within the past 5 years.
17. Clinically significant abnormal vital signs including sustained sitting blood pressure \>160/100 mm Hg.
18. Diabetes mellitus with hemoglobin A1c (HbA1c) levels of ≥8.0%.
19. Known history of human immunodeficiency virus (HIV-1 or 2).
20. Known history of acute/chronic hepatitis B or C unless treated curatively.

Where it's running

• University of California, San Francisco
  San Francisco, California, United States Recruiting
• Massachusetts General Hospital
  Boston, Massachusetts, United States Recruiting
• Butler Hospital
  Providence, Rhode Island, United States Recruiting
• Houston Methodist Neurological Institute
  Houston, Texas, United States Recruiting
• University of Washington, Memory and Brain Wellness Center
  Seattle, Washington, United States Recruiting
• Banner Sun Health Research Institute
  Sun City, Arizona, United States Not yet recruiting

Study contacts

• Principal investigator: Adam Boxer, MD, PhD — University of California, San Francisco
• Study coordinator: PSP Trial Platform Team
• Email: psptrialplatform@ucsf.edu
• Phone: 415-514-9420

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Tributyrin for PSP: a virtual, early-phase study

  This study tests whether tributyrin is safe and tolerable in people with Progressive Supranuclear Palsy who are 45 or older, using virtual visits.

  Recruiting Phase 1/2 University of Michigan · Ann Arbor, Michigan · NCT07863180

• Biomarker Repository for Age-Related and Inherited Neurodegenerative Diseases

  This study collects blood and genetic samples from people with neurodegenerative diseases and their families to build a shared resource for studying disease risk and biology.

  Recruiting University of Pennsylvania · Philadelphia, Pennsylvania · NCT07798700

• Using AI to find facial and speech signs of Parkinson's and related disorders

  This project uses AI to analyze facial expressions and speech to try to find digital markers that help diagnose and tell apart Parkinson's disease, PSP, MSA, and healthy people.

  Recruiting Beijing Tiantan Hospital · Beijing, Beijing Municipality · NCT07392411

• Using AI to improve the levodopa challenge test for Parkinson's disease diagnosis

  This study is testing a new way to diagnose Parkinson's disease using artificial intelligence and video recordings to see if it can improve the accuracy of the levodopa challenge test.

  Recruiting Beijing Tiantan Hospital · Beijing · NCT06949865

• Genetic program for Progressive Supranuclear Palsy and related conditions

  This study is collecting DNA from adults with Progressive Supranuclear Palsy and similar conditions to see how genetics might help improve diagnosis and care for patients.

  Recruiting Massachusetts General Hospital · Boston, Massachusetts · NCT06647641

• Creating a repository for neurodegenerative disease data

  This study is creating a large collection of information about neurodegenerative diseases to help researchers learn more and develop better treatments for people affected by these conditions.

  Recruiting University of Pennsylvania · Philadelphia, Pennsylvania · NCT04715399

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.