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

Progressive Supranuclear Palsy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04993768
• Other study ID #: TPN-101-PSP-201
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: October 15, 2021
• Est. completion date: December 31, 2023

Study information

• Verified date: July 2023
• Source: Transposon Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Progressive Supranuclear Palsy
• Supranuclear Palsy, Progressive

Intervention

Drug:
• TPN-101, 100 mg/day
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
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
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
Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Transposon Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

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