A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
1 other identifier
interventional
42
1 country
14
Brief Summary
This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2021
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 19, 2021
CompletedFirst Posted
Study publicly available on registry
August 6, 2021
CompletedStudy Start
First participant enrolled
December 12, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 24, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
March 24, 2024
CompletedApril 8, 2026
April 1, 2026
2.2 years
July 19, 2021
April 2, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)
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
48 weeks
Secondary Outcomes (3)
Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)
48 weeks
Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL)
48 weeks
Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)
48 weeks
Study Arms (4)
TPN-101, Dose A
EXPERIMENTALTPN-101, Dose B
EXPERIMENTALTPN-101, Dose C
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
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).
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).
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 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
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
Rocky Mountain Movement Disorders Center
Englewood, Colorado, 80113, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486, United States
UFHealth Fixel Institute for Neurological Diseases
Gainesville, Florida, 32608, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Quest Research Institute
Farmington Hills, Michigan, 48334, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Cleveland Clinic Lou Ruvo Center for Brain Health
Las Vegas, Nevada, 89106, United States
Irving Center for Clinical and Translational Research
New York, New York, 10032, United States
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Related Publications (1)
Nicodemus J, Liu CS, Ransom L, Tan V, Romanow W, Jimenez N, Chun J. Sequence Diversity and Encoded Enzymatic Differences of Monocistronic L1 ORF2 mRNA Variants in the Aged Normal and Alzheimer's Disease Brain. J Neurosci. 2025 Jun 18;45(25):e2298242025. doi: 10.1523/JNEUROSCI.2298-24.2025.
PMID: 40368603DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 19, 2021
First Posted
August 6, 2021
Study Start
December 12, 2021
Primary Completion
February 24, 2024
Study Completion
March 24, 2024
Last Updated
April 8, 2026
Record last verified: 2026-04