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

Status Active, not recruiting

Clinical Trial 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).

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04993755
Study type	Interventional
Source	Transposon Therapeutics, Inc.
Contact
Status	Active, not recruiting
Phase	Phase 2
Start date	October 1, 2021
Completion date	September 1, 2023

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