Clinical trial NCT04993768: A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
Official title: A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Progressive Supranuclear Palsy
Lead sponsor: Transposon Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2021-08-06
Enrollment: 40
Sex: ALL
Minimum age (years): 41
Maximum age (years): 86
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)
timeFrame: 48 weeks
Secondary outcomes:
measure: Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)
timeFrame: 48 weeks
measure: Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL)
timeFrame: 48 weeks
measure: Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center, Barrow Neurological Institute
state: Arizona
city: La Jolla
country: United States
facility: UC San Diego Altman Clinical And Translational Research Institute
state: California
city: San Francisco
country: United States
facility: UCSF Neurosciences Clinical Research Unit (NCRU)
state: California
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center
state: Colorado
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Gainesville
country: United States
facility: UFHealth Fixel Institute for Neurological Diseases
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Las Vegas
country: United States
facility: Cleveland Clinic Lou Ruvo Center for Brain Health
state: Nevada
city: New York
country: United States
facility: Irving Center for Clinical and Translational Research
state: New York
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
Study officials:
References: