Summary

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

Description

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).

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)

Details

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.

Keywords

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

Eligibility

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

Locations

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

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Transposon Therapeutics, Inc.
ID
NCT04993755
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 40 study participants
Last Updated