Clincosm LogoSign in Get a demo

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

Sponsor
Transposon Therapeutics, Inc. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04993768
Collaborator
(none)
40
Enrollment
14
Locations
4
Arms
21
Anticipated Duration (Months)
2.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Drug: TPN-101, 100 mg/day
  • Drug: TPN-101, 200 mg/day
  • Drug: TPN-101, 400 mg/day
  • Drug: Placebo
 Phase 2

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
40 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
Official Title:
A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
Actual Study Start Date :
Oct 15, 2021
Anticipated Primary Completion Date :
Jul 15, 2023
Anticipated Study Completion Date :
Jul 15, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: TPN-101, Dose A

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).
Experimental: TPN-101, Dose B

Drug: 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).
Experimental: TPN-101, Dose C

Drug: 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 Comparator: Placebo

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

Outcome Measures

Primary Outcome Measures

  1. Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP) [48 weeks]

    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

  1. Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF) [48 weeks]

  2. 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]

  3. Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS) [48 weeks]

    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.

Eligibility Criteria

Criteria

Ages Eligible for Study:
41 Years to 86 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 St. Joseph's Hospital and Medical Center, Barrow Neurological Institute Phoenix Arizona United States 85013
2 UC San Diego Altman Clinical And Translational Research Institute La Jolla California United States 92037
3 UCSF Neurosciences Clinical Research Unit (NCRU) San Francisco California United States 94158
4 Rocky Mountain Movement Disorders Center Englewood Colorado United States 80113
5 Parkinson's Disease and Movement Disorders Center of Boca Raton Boca Raton Florida United States 33486
6 UFHealth Fixel Institute for Neurological Diseases Gainesville Florida United States 32608
7 Johns Hopkins University School of Medicine Baltimore Maryland United States 21287
8 Massachusetts General Hospital Boston Massachusetts United States 02114
9 Quest Research Institute Farmington Hills Michigan United States 48334
10 Mayo Clinic Rochester Minnesota United States 55905
11 Cleveland Clinic Lou Ruvo Center for Brain Health Las Vegas Nevada United States 89106
12 Irving Center for Clinical and Translational Research New York New York United States 10032
13 Hospital of the University of Pennsylvania Philadelphia Pennsylvania United States 19104
14 University of Texas Southwestern Medical Center Dallas Texas United States 75390

Sponsors and Collaborators

  • Transposon Therapeutics, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Transposon Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT04993768
Other Study ID Numbers:
  • TPN-101-PSP-201
First Posted:
Aug 6, 2021
Last Update Posted:
Aug 11, 2022
Last Verified:
Aug 1, 2022
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Supranuclear Palsy, Progressive

Study Results

No Results Posted as of Aug 11, 2022