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TPN-101, 400 mg/day for Amyotrophic Lateral Sclerosis

Phase 2

Waitlist Available

Research Sponsored by Transposon Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 48 weeks

Awards & highlights

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

Amyotrophic Lateral Sclerosis
Frontotemporal Dementia

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 48 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~48 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD)

Secondary outcome measures

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)

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: TPN-101, 400 mg/dayExperimental Treatment1 Intervention

Group II: PlaceboPlacebo Group1 Intervention

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Who is running the clinical trial?

Transposon Therapeutics, Inc.Lead Sponsor

2 Previous Clinical Trials

56 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

How many trial subjects are engaging in this medical research?

"Correct. Clinicaltrials.gov 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."

Answered by AI

Are any openings available to participate in this experiment?

"The data located on clinicaltrials.gov 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."

Answered by AI

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."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

2021. "A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04993755.