New drug shows promise for rare genetic brain diseases
NCT ID NCT04993755
First seen Apr 06, 2026 · Last updated Apr 23, 2026 · Updated 1 time
Summary
This study tested a new medicine called TPN-101 in 42 people who have ALS or frontotemporal dementia caused by a specific gene change (C9orf72). The main goal was to see if the drug is safe and tolerable. Researchers also looked at how the drug moves in the body and whether it affects markers of nerve damage.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FRONTOTEMPORAL DEMENTIA are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
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
Conditions
Explore the condition pages connected to this study.