New drug TPN-101 tested in ALS and dementia patients with C9orf72 gene mutation
NCT ID NCT04993755
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHU Dupuytren, Limoges
Limoges, 87042, France
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CHU Lille - CMRR Hôpital Roger Salengro
Lille, 59037, France
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Columbia University Medical Center - The Neurological Institute of New York
New York, New York, 10032, United States
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Complejo Hospitalario Universitario de Santiago (CHUS)
Santiago de Compostela, A Coruña, 15706ES, Spain
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Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux
Paris, 75013, France
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Hospital Universitari I Politècnic La Fe
Valencia, 46026, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035ES, Spain
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Hospital de la Santa Creu i Sant Pau
Barcelona, 08025, Spain
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Hospital for Special Surgery
New York, New York, 10021, United States
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John Hopkins University
Baltimore, Maryland, 21287, United States
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Johns Hopkins Outpatient Center
Baltimore, Maryland, 21287, United States
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Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic
Boston, Massachusetts, 02114, United States
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Mayo Family Clinic Northwest
Rochester, Minnesota, 55905, United States
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The University of North Carolina at Chapel Hill, Department of Neurology
Chapel Hill, North Carolina, 27599, United States
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UCSF Neurosciences Clinical Research Unit (NCRU)
San Francisco, California, 94158, United States
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Universitaetsklinikum Ulm - Klinik fuer Neurologie
Ulm, Baden-Wurttemberg, 89081, Germany
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University of California Irvine - ALS & Neuromuscular Center
Orange, California, 92868, United States
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University of California San Diego
La Jolla, California, 92037, United States
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VIB-KU Leuven Center for Brain & Disease Research
Leuven, Flemish Brabankt, 3000, Belgium
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