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New drug tested for rare brain disease PSP

NCT ID NCT04993768

Disease control Sponsor: Transposon Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called TPN-101 in 42 people with progressive supranuclear palsy (PSP), a rare brain disease that affects movement and thinking. The main goal was to see if the drug is safe and tolerable over 48 weeks. Researchers also looked at how the drug moves in the body and its effects on brain health.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

42 people

The number who actually took part.

Started

Dec 2021

Finished

Mar 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

41 to 86 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Clinical diagnosis of probable progressive supranuclear palsy (PSP) 2. Presence of PSP symptoms for less than 5 years 3. Has a reliable caregiver/informant to accompany the patient to all study visits. 4. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening 5. Patient must reside outside a skilled nursing facility or dementia care facility at the time of Screening, and admission to such a facility must not be planned. Residence in an assisted living facility is allowed Exclusion Criteria: Patients must not meet any of the following criteria: 1. Presence of other significant neurological or psychiatric disorders 2. History of clinically significant brain abnormality 3. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa 4. Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean 5. Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or any other frontotemporal lobar degeneration causative genes not associated with underlying tau pathology 6. History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease

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Conditions

The condition(s) this trial relates to.

progressive supranuclear palsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Progressive supranuclear palsy

Contacts and locations

Locations

  • Cleveland Clinic Lou Ruvo Center for Brain Health

    Las Vegas, Nevada, 89106, United States

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • Irving Center for Clinical and Translational Research

    New York, New York, 10032, United States

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21287, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Parkinson's Disease and Movement Disorders Center of Boca Raton

    Boca Raton, Florida, 33486, United States

  • Quest Research Institute

    Farmington Hills, Michigan, 48334, United States

  • Rocky Mountain Movement Disorders Center

    Englewood, Colorado, 80113, United States

  • St. Joseph's Hospital and Medical Center, Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • UC San Diego Altman Clinical And Translational Research Institute

    La Jolla, California, 92037, United States

  • UCSF Neurosciences Clinical Research Unit (NCRU)

    San Francisco, California, 94158, United States

  • UFHealth Fixel Institute for Neurological Diseases

    Gainesville, Florida, 32608, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

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