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

NCT04993755 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 42

Last updated 2026-04-03

No results posted yet for this study

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).

Conditions

Interventions

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).

Sponsors & Collaborators

  • Transposon Therapeutics, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-01
Primary Completion
2023-08-17
Completion
2023-09-01
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • France
  • Germany
  • Spain

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04993755 on ClinicalTrials.gov