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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04993768
Recruitment Status : Active, not recruiting
First Posted : August 6, 2021
Last Update Posted : July 13, 2023
Sponsor:
Information provided by (Responsible Party):
Transposon Therapeutics, Inc.

Study Description
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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
Progressive Supranuclear Palsy 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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : October 15, 2021
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : December 31, 2023

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Arms and Interventions
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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
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Primary Outcome Measures :
  1. Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 48 weeks ]
  3. Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS) [ Time Frame: 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
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Ages Eligible for Study:   41 Years to 86 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04993768


Locations
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United States, Arizona
St. Joseph's Hospital and Medical Center, Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
United States, California
UC San Diego Altman Clinical And Translational Research Institute
La Jolla, California, United States, 92037
UCSF Neurosciences Clinical Research Unit (NCRU)
San Francisco, California, United States, 94158
United States, Colorado
Rocky Mountain Movement Disorders Center
Englewood, Colorado, United States, 80113
United States, Florida
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, United States, 33486
UFHealth Fixel Institute for Neurological Diseases
Gainesville, Florida, United States, 32608
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Michigan
Quest Research Institute
Farmington Hills, Michigan, United States, 48334
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Nevada
Cleveland Clinic Lou Ruvo Center for Brain Health
Las Vegas, Nevada, United States, 89106
United States, New York
Irving Center for Clinical and Translational Research
New York, New York, United States, 10032
United States, Pennsylvania
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Transposon Therapeutics, Inc.
More Information
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Responsible Party: Transposon Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04993768    
Other Study ID Numbers: TPN-101-PSP-201
First Posted: August 6, 2021    Key Record Dates
Last Update Posted: July 13, 2023
Last Verified: July 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Supranuclear Palsy, Progressive
Paralysis
Neurologic Manifestations
Nervous System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
 Movement Disorders
Ophthalmoplegia
Ocular Motility Disorders
Cranial Nerve Diseases
Tauopathies
Neurodegenerative Diseases
Eye Diseases