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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
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
- 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
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
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