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

NCT ID: NCT04993768

Last Updated: 2023-07-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-15

Study Completion Date

2023-12-31

Brief Summary

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

Detailed Description

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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.

Conditions

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Progressive Supranuclear Palsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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TPN-101, Dose A

Group Type EXPERIMENTAL

TPN-101, 100 mg/day

Intervention Type DRUG

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, Dose B

Group Type EXPERIMENTAL

TPN-101, 200 mg/day

Intervention Type DRUG

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, Dose C

Group Type EXPERIMENTAL

TPN-101, 400 mg/day

Intervention Type DRUG

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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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).

Intervention Type 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).

Intervention Type 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).

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

41 Years

Maximum Eligible Age

86 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Transposon Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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St. Joseph's Hospital and Medical Center, Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

UC San Diego Altman Clinical And Translational Research Institute

La Jolla, California, United States

Site Status

UCSF Neurosciences Clinical Research Unit (NCRU)

San Francisco, California, United States

Site Status

Rocky Mountain Movement Disorders Center

Englewood, Colorado, United States

Site Status

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

Site Status

UFHealth Fixel Institute for Neurological Diseases

Gainesville, Florida, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Quest Research Institute

Farmington Hills, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Cleveland Clinic Lou Ruvo Center for Brain Health

Las Vegas, Nevada, United States

Site Status

Irving Center for Clinical and Translational Research

New York, New York, United States

Site Status

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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TPN-101-PSP-201

Identifier Type: -

Identifier Source: org_study_id

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