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NCT Number: NCT04993768

A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)

This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.

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Key information

Conditions

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

Age range

41 year–86 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St. Joseph's Hospital and Medical Center, Barrow Neurological Institute, Phoenix, Arizona, United States

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About this study

This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 4-arm study with an open-label treatment phase in patients with PSP. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open label Treatment Period, and a Follow-up Visit 4 weeks post treatment.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Clinical diagnosis of probable progressive supranuclear palsy (PSP)
  • Presence of PSP symptoms for less than 5 years
  • Has a reliable caregiver/informant to accompany the patient to all study visits.
  • Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
  • 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:

  • Presence of other significant neurological or psychiatric disorders
  • History of clinically significant brain abnormality
  • Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa
  • Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean
  • 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
  • History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease

Treatment and study plan

TPN-101, 100 mg/day

Drug

100 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

TPN-101, 200 mg/day

Drug

200 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

TPN-101, 400 mg/day

Drug

400 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

Placebo

Drug

Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

Primary outcomes

  1. Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)

    Time frame: 48 weeks

    Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) associated with TPN-101 v. placebo administered for up to 48 weeks in patients with PSP

Secondary outcomes

  1. Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)

    Time frame: 48 weeks

  2. Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL)

    Time frame: 48 weeks

  3. Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)

    Time frame: 48 weeks

    The PSPRS is comprised of 28 items in six categories: daily activities (by history), behavior, bulbar, ocular motor, limb motor and gait/midline. Scores range from 0 to 100, each item is graded 0-2 (six items) or 0-4 (22 items), with lower scores indicating better clinical and functional status.

Sponsors and collaborators

Lead sponsor

Transposon Therapeutics, Inc.

Industry

Registry information

Important dates

Study start
2021
Primary completion
2024
Study completion
2024
First posted
Aug 6, 2021
Registry last updated
Apr 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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