for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
completion around



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).

Official Title

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)


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.


Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS, FTD, Dementia, Motor Neuron Disease, Primary Progressive Aphasia, Pick Disease of the Brain, Sclerosis, TPN-101, 400 mg/day


You can join if…

Open to people ages 18 years and up

  • Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene
  • Has a reliable caregiver/informant to accompany the patient to all study visits

    For patients with ALS (with or without FTD):

  • Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria
  • Onset of weakness within 3 years prior to Screening
  • Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position)
  • Able to perform reproducible pulmonary function tests.
  • ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 and score of 3 or 4 on Item #3 (swallowing) at Screening

    For patients with FTD:

  • A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome
  • CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening

You CAN'T join if...

  • Presence of other significant neurological or psychiatric disorders
  • History of clinically significant brain abnormality
  • Clinically significant medical illness
  • Tracheostomy or diaphragmatic pacing
  • Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable)
  • History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization


  • University of California Irvine - ALS & Neuromuscular Center
    Orange California 92868 United States
  • University of California San Diego
    La Jolla California 92037 United States


in progress, not accepting new patients
Start Date
Completion Date
Transposon Therapeutics, Inc.
Phase 2 research study
Study Type
About 42 people participating
Last Updated