TPN-101, 400 mg/day for Frontotemporal Dementia

Phase-Based Progress Estimates
UCSF Neurosciences Clinical Research Unit (NCRU), San Francisco, CAFrontotemporal Dementia+1 MoreTPN-101, 400 mg/day - Drug
All Sexes
What conditions do you have?

Study Summary

This trial will test a new drug, TPN-101, for safety and tolerability in people with ALS or FTD caused by a certain gene mutation.

Eligible Conditions
  • Frontotemporal Dementia
  • ALS (Amyotrophic Lateral Sclerosis)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 3 Secondary · Reporting Duration: 48 weeks

48 weeks
Assess the clinical effect of TPN-101 as measured by changes in score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
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 pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)
Assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

TPN-101, 400 mg/day
1 of 2
1 of 2

Experimental Treatment

Non-Treatment Group

42 Total Participants · 2 Treatment Groups

Primary Treatment: TPN-101, 400 mg/day · Has Placebo Group · Phase 2

TPN-101, 400 mg/day
Experimental Group · 1 Intervention: TPN-101, 400 mg/day · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 48 weeks

Who is running the clinical trial?

Transposon Therapeutics, Inc.Lead Sponsor
2 Previous Clinical Trials
56 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 9 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have experienced a slow and steady decline in your ability to think, speak, or move over time, which may be due to various conditions such as mild cognitive impairment or behavioral variant FTD.
You started feeling weak within the last 3 years before the screening.

Frequently Asked Questions

Has this scientific investigation been implemented in numerous locales within the state?

"This study is being conducted in a variety of sites, including the University of California, Irvine (Orange, CA), Massachusetts General Hospital (Boston MA), and Johns Hopkins Outpatient Center (Baltimore MD). There are an additional 12 locations that are accepting participants." - Anonymous Online Contributor

Unverified Answer

How many trial subjects are engaging in this medical research?

"Correct. notes that this trial, which was first published on October 1st 2021, is still actively recruiting participants. The study requires 40 volunteers to participate at 12 different healthcare facilities." - Anonymous Online Contributor

Unverified Answer

Are any openings available to participate in this experiment?

"The data located on indicates that this research endeavour is still in recruitment mode, with the initial posting being made October 1st of 2021 and most recent updates occurring November 5th 2022." - Anonymous Online Contributor

Unverified Answer

Has the FDA signified permission for TPN-101, 400 mg/day?

"Our team has rated TPN-101, 400 mg/day a 2 on the safety scale due to evidence of its security in earlier studies, yet no data indicating that it is an efficacious treatment." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.