NCT04993755

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

A Phase 2a Study of TPN-101 in Patients With Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated With Hexanucleotide Repeat Expansion in the C9orf72 Gene (C9ORF72 ALS/FTD)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: Transposon Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

Detailed description

This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with a long-term, open-label treatment phase in patients with C9ORF72 ALS and/or FTD. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open-label Treatment Period, and a Follow-up Visit 4 weeks post-treatment.

Conditions

• Amyotrophic Lateral Sclerosis
• Frontotemporal Dementia

Interventions

Type	Name	Description
DRUG	TPN-101, 400 mg/day	400 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
DRUG	Placebo	Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).

Timeline

• Start date: 2021-10-01
• Primary completion: 2023-08-17
• Completion: 2023-09-01
• First posted: 2021-08-06
• Last updated: 2026-04-03

Locations

19 sites across 5 countries: United States, Belgium, France, Germany, Spain

Regulatory

• FDA-regulated drug study