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New drug TPN-101 tested in ALS and dementia patients with C9orf72 gene mutation

NCT ID NCT04993755

Disease control Sponsor: Transposon Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called TPN-101 in 42 people with ALS or frontotemporal dementia caused by a specific gene change (C9orf72). The main goal was to check if the drug is safe and tolerable over 48 weeks. Researchers also looked at how the drug moves in the body and whether it affects markers of nerve damage.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

42 people

The number who actually took part.

Started

Oct 2021

Finished

Sep 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 Exclusion Criteria: * 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

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis frontotemporal dementia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyotrophic lateral sclerosis Frontotemporal dementia

Contacts and locations

Locations

  • CHU Dupuytren, Limoges

    Limoges, 87042, France

  • CHU Lille - CMRR Hôpital Roger Salengro

    Lille, 59037, France

  • Columbia University Medical Center - The Neurological Institute of New York

    New York, New York, 10032, United States

  • Complejo Hospitalario Universitario de Santiago (CHUS)

    Santiago de Compostela, A Coruña, 15706ES, Spain

  • Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux

    Paris, 75013, France

  • Hospital Universitari I Politècnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035ES, Spain

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08025, Spain

  • Hospital for Special Surgery

    New York, New York, 10021, United States

  • John Hopkins University

    Baltimore, Maryland, 21287, United States

  • Johns Hopkins Outpatient Center

    Baltimore, Maryland, 21287, United States

  • Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic

    Boston, Massachusetts, 02114, United States

  • Mayo Family Clinic Northwest

    Rochester, Minnesota, 55905, United States

  • The University of North Carolina at Chapel Hill, Department of Neurology

    Chapel Hill, North Carolina, 27599, United States

  • UCSF Neurosciences Clinical Research Unit (NCRU)

    San Francisco, California, 94158, United States

  • Universitaetsklinikum Ulm - Klinik fuer Neurologie

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University of California Irvine - ALS & Neuromuscular Center

    Orange, California, 92868, United States

  • University of California San Diego

    La Jolla, California, 92037, United States

  • VIB-KU Leuven Center for Brain & Disease Research

    Leuven, Flemish Brabankt, 3000, Belgium

More trials for these conditions

Other studies related to the condition(s) this trial covers.