TPN-101 for Progressive Supranuclear Palsy

Recruiting at 16 trial locations

Overseen By Jay Soto

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Transposon Therapeutics, Inc.

Disqualifiers: Neurological, Psychiatric, Brain abnormality, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

40 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests a new drug, TPN-101, to see if it is safe and can be tolerated by patients with Progressive Supranuclear Palsy (PSP). PSP is a rare brain disorder that affects movement and balance. The study will check if TPN-101 can help with symptoms or slow down the disease.

Do I need to stop my current medications for the TPN-101 trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What makes TPN-101 unique for treating progressive supranuclear palsy?

TPN-101 is being tested for progressive supranuclear palsy (PSP), a condition with no effective treatments currently available. Unlike previous treatments like davunetide and tideglusib, which failed to show benefits, TPN-101 offers a new approach that could potentially address the lack of effective therapies for PSP. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for patients with Progressive Supranuclear Palsy (PSP) who've had symptoms for less than 5 years and have a probable PSP diagnosis. Participants need to score at least 18 on the Mini Mental State Exam, live outside of nursing facilities, and have a reliable caregiver. It's not for those with certain genetic mutations or significant neurological, psychiatric, or other serious health issues.

Inclusion Criteria

You have been experiencing symptoms of PSP for less than 5 years.

Has a reliable caregiver/informant to accompany the patient to all study visits

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

Your progranulin level in your blood is significantly lower than normal.

You have specific genetic mutations known to cause frontotemporal lobar degeneration, except those related to tau pathology.

You have other important brain or mental health conditions.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Double-blind Treatment

Participants receive either TPN-101 or placebo in a double-blind manner

24 weeks

Open-label Treatment

Participants receive TPN-101 in an open-label manner

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

Treatment Details

Interventions

• Placebo
• TPN-101

Trial Overview The study tests the safety and tolerability of TPN-101 at different doses (100 mg/day, 200 mg/day, and 400 mg/day) compared to a placebo in people with PSP. The goal is to find out how well patients handle the medication.

Participant Groups

Group I: TPN-101, Dose C Experimental Treatment 1 Intervention

Group II: TPN-101, Dose B Experimental Treatment 1 Intervention

Group III: TPN-101, Dose A Experimental Treatment 1 Intervention

Group IV: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Transposon Therapeutics, Inc.

Lead Sponsor

Trials

3

Recruited

100+

Findings from Research

A new quantitative MRI analysis method was developed to measure disease progression in progressive supranuclear palsy (PSP), using data from 99 patients in two clinical trials, which can help in designing future studies.

The study identified that changes in the volumes of the third ventricle, midbrain, and frontal lobe are effective indicators of disease progression, requiring fewer patients to detect treatment efficacy compared to traditional clinical scales.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Longitudinal magnetic resonance imaging in progressive supranuclear palsy: A new combined score for clinical trials. Höglinger, GU., Schöpe, J., Stamelou, M., et al.[2018]

In a study involving 187 patients with progressive supranuclear palsy (PSP), the PSP-Rating Scale was identified as the most efficient measure for detecting significant changes in disease progression, requiring only 51 patients per group for a 50% change over one year.

The study found no detectable placebo effect on the PSP-Rating Scale or the Schwab and England Activities of Daily Living, suggesting that these scales can reliably assess treatment efficacy in future clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. Stamelou, M., Schöpe, J., Wagenpfeil, S., et al.[2021]

There is currently no effective treatment for progressive supranuclear palsy (PSP), as most studies have shown negative outcomes and there are no randomized controlled trials to establish therapeutic standards.

The review highlights the ongoing challenges in developing effective therapies for PSP and suggests that future strategies may be needed to address this unmet medical need.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Toward future therapies in progressive supranuclear palsy. Burn, DJ., Warren, NM.[2005]

References

1. United States pubmed.ncbi.nlm.nih.gov

Longitudinal magnetic resonance imaging in progressive supranuclear palsy: A new combined score for clinical trials. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Interventions in progressive supranuclear palsy. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Toward future therapies in progressive supranuclear palsy. [2005]

5. England pubmed.ncbi.nlm.nih.gov

Progressive supranuclear palsy: Advances in diagnosis and management. [2023]