Progressive Supranuclear Palsy Clinical Trial
Official title:
A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
Verified date | July 2023 |
Source | Transposon Therapeutics, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.
Status | Active, not recruiting |
Enrollment | 40 |
Est. completion date | December 31, 2023 |
Est. primary completion date | December 31, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 41 Years to 86 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Johns Hopkins University School of Medicine | Baltimore | Maryland |
United States | Parkinson's Disease and Movement Disorders Center of Boca Raton | Boca Raton | Florida |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | University of Texas Southwestern Medical Center | Dallas | Texas |
United States | Rocky Mountain Movement Disorders Center | Englewood | Colorado |
United States | Quest Research Institute | Farmington Hills | Michigan |
United States | UFHealth Fixel Institute for Neurological Diseases | Gainesville | Florida |
United States | UC San Diego Altman Clinical And Translational Research Institute | La Jolla | California |
United States | Cleveland Clinic Lou Ruvo Center for Brain Health | Las Vegas | Nevada |
United States | Irving Center for Clinical and Translational Research | New York | New York |
United States | Hospital of the University of Pennsylvania | Philadelphia | Pennsylvania |
United States | St. Joseph's Hospital and Medical Center, Barrow Neurological Institute | Phoenix | Arizona |
United States | Mayo Clinic | Rochester | Minnesota |
United States | UCSF Neurosciences Clinical Research Unit (NCRU) | San Francisco | California |
Lead Sponsor | Collaborator |
---|---|
Transposon Therapeutics, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF) | 48 weeks | ||
Secondary | 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 | ||
Secondary | Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS) | 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. | 48 weeks |
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