A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD
a study on Amyotropic Lateral Sclerosis (ALS) Dementia Frontotemporal Dementia Primary Progressive Aphasia
Summary
- Eligibility
- for people ages 18 years and up (full criteria)
- Location
- at La Jolla, California and other locations
- Dates
- study startedestimated 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, Primary Progressive Aphasia, 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 San Diego
accepting new patients
La Jolla California 92037 United States - University of California Irvine - ALS & Neuromuscular Center
accepting new patients
Orange California 92868 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
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