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

Primary Purpose

Progressive Supranuclear Palsy

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
TPN-101, 100 mg/day
TPN-101, 200 mg/day
TPN-101, 400 mg/day
Placebo
Sponsored by
Transposon Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Progressive Supranuclear Palsy

Eligibility Criteria

41 Years - 86 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • St. Joseph's Hospital and Medical Center, Barrow Neurological Institute
  • UC San Diego Altman Clinical And Translational Research Institute
  • UCSF Neurosciences Clinical Research Unit (NCRU)
  • Rocky Mountain Movement Disorders Center
  • Parkinson's Disease and Movement Disorders Center of Boca Raton
  • UFHealth Fixel Institute for Neurological Diseases
  • Johns Hopkins University School of Medicine
  • Massachusetts General Hospital
  • Quest Research Institute
  • Mayo Clinic
  • Cleveland Clinic Lou Ruvo Center for Brain Health
  • Irving Center for Clinical and Translational Research
  • Hospital of the University of Pennsylvania
  • University of Texas Southwestern Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

TPN-101, Dose A

TPN-101, Dose B

TPN-101, Dose C

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
July 19, 2021
Last Updated
July 12, 2023
Sponsor
Transposon Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04993768
Brief Title
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

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 15, 2021 (Actual)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Transposon Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Supranuclear Palsy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
TPN-101, Dose A
Arm Type
Experimental
Arm Title
TPN-101, Dose B
Arm Type
Experimental
Arm Title
TPN-101, Dose C
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
TPN-101, 100 mg/day
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
TPN-101, 200 mg/day
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
TPN-101, 400 mg/day
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
Primary Outcome Measure Information:
Title
Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)
Description
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
Time Frame
48 weeks
Secondary Outcome Measure Information:
Title
Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)
Time Frame
48 weeks
Title
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
Title
Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)
Description
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.
Time Frame
48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
41 Years
Maximum Age & Unit of Time
86 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
St. Joseph's Hospital and Medical Center, Barrow Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
UC San Diego Altman Clinical And Translational Research Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
UCSF Neurosciences Clinical Research Unit (NCRU)
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Rocky Mountain Movement Disorders Center
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Parkinson's Disease and Movement Disorders Center of Boca Raton
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33486
Country
United States
Facility Name
UFHealth Fixel Institute for Neurological Diseases
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Johns Hopkins University School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Quest Research Institute
City
Farmington Hills
State/Province
Michigan
ZIP/Postal Code
48334
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Cleveland Clinic Lou Ruvo Center for Brain Health
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89106
Country
United States
Facility Name
Irving Center for Clinical and Translational Research
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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