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

Completed · Phase 2 · Has a placebo group

Conditions studied: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia

In brief

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).

Key facts

Study ID

NCT04993755

Run by

Transposon Therapeutics, Inc.

People needed

42

Starts

2021-10-01

Expected to finish

2023-09-01

Last updated by the study team

2026-04-03

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify 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

Where it is running

• University of California San Diego — La Jolla, California, United States
• University of California Irvine - ALS & Neuromuscular Center — Orange, California, United States
• UCSF Neurosciences Clinical Research Unit (NCRU) — San Francisco, California, United States
• John Hopkins University — Baltimore, Maryland, United States
• Johns Hopkins Outpatient Center — Baltimore, Maryland, United States
• Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic — Boston, Massachusetts, United States
• Mayo Family Clinic Northwest — Rochester, Minnesota, United States
• Hospital for Special Surgery — New York, New York, United States
• Columbia University Medical Center - The Neurological Institute of New York — New York, New York, United States
• The University of North Carolina at Chapel Hill, Department of Neurology — Chapel Hill, North Carolina, United States
• VIB-KU Leuven Center for Brain & Disease Research — Leuven, Flemish Brabankt, Belgium
• CHU Lille - CMRR Hôpital Roger Salengro — Lille, France
• CHU Dupuytren, Limoges — Limoges, France
• Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux — Paris, France
• Universitaetsklinikum Ulm - Klinik fuer Neurologie — Ulm, Baden-Wurttemberg, Germany
• Complejo Hospitalario Universitario de Santiago (CHUS) — Santiago de Compostela, A Coruña, Spain
• Hospital de la Santa Creu i Sant Pau — Barcelona, Spain
• Hospital Universitario Vall d'Hebron — Barcelona, Spain
• Hospital Universitari I Politècnic La Fe — Valencia, Spain

Full record on ClinicalTrials.gov

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