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

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

Summary

This trial tests a new drug, TPN-101, for safety and tolerability in patients with ALS and/or FTD who have a specific genetic mutation. The goal is to see if it can help manage symptoms or slow disease progression.

Eligible Conditions
  • Frontotemporal Dementia
  • Amyotrophic Lateral Sclerosis

Timeline

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD)
Secondary study objectives
Assess the clinical effect of TPN-101 as measured by changes in score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

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
~10 spots leftby Dec 2025
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